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  • Writer's pictureJohn Q Leonard

37 New Drug Approvals from the FDA in 2022

Updated: Jan 18, 2023

37 New approvals from the FDA in 2022 is a bit on the high side compared to the average (see the graphic at the end of the post) This is impressive industry output given the damper that the coronavirus pandemic put on recruiting for phase 3 clinical trials in 2020 and 2021.



Active ingredient: Daridorexant Disease: Insomnia Peak sales estimate: $1.05 billion Approved: Jan. 7 Company: Idorsia

Summary: Over two decades, the husband-and-wife team of Jean-Paul and Martine Clozel considered—by their estimate—25,000 compounds before they hit on the molecule they thought could be a game changer in an untapped market. There are roughly 25 million people in the U.S. who have insomnia, and only 30% have been diagnosed. According to Allied Research, the market for insomnia will reach $6.3 billion by 2030. Quviviq became the third approved drug from the dual orexin receptor class. The others—Merck’s Belsomra and Eisai’s Dayvigo—have had limited success, with sales of $318 million and $67 million, respectively, in 2021. Idorsia is promoting Quviviq as having the unique ability to keep patients alert during the day. Upon its launch in May—which was delayed because Quviviq is classified as a controlled substance—Idorsia came with a multimedia blitz featuring actress Jennifer Aniston. It also hired 500 sales reps in U.S. Quviviq is priced at roughly $490 for a month’s supply of 30 tablets, compared to Belsomra’s price tag of $434. Quviviq is forecast to reach peak sales of $1.05 billion in 2026, according to Jefferies.



Active ingredient: Abrocitinib Disease: Refractory atopic dermatitis Peak sales estimate: $3 billion Approved: Jan. 14 Company: Pfizer

Summary: Pfizer began touting Cibinqo—its oral daily JAK1 inhibitor for moderate to severe atopic dermatitis that hasn’t responded to other treatments—as a potential blockbuster for the company well before it even secured FDA approval, with the company's own biopharma chief Angela Hwang suggesting in 2021 that the drug could ultimately reach an estimated $3 billion in sales. But that forecast may be overly optimistic: A survey of global physicians published shortly after the approval showed that many are still wary of prescribing oral and topical JAK meds for eczema—worries that had previously caused the FDA to pause its reviews of all pending drugs in the category in 2021 while it investigated a potentially heightened risk of cardiovascular conditions, death and cancer for those taking JAK inhibitors. Despite that uncertainty, Pfizer is full steam ahead on Cibinqo’s U.S. launch. The drug is already the star of two TV ads, the first of which was a relatively simple, text-only commercial launched at the end of May, followed by a more traditional spot that rolled out in November and features actors astral-projecting their eczema onto a separate, ghostly copy of themselves.



Active ingredient: Tebentafusp-tebn Disease: Uveal melanoma Peak sales estimate: $219 million in 2030 (Jefferies) Approved: Jan. 25 Company: Immunocore Summary: Immunocore double-dipped in its early 2022 approval for Kimmtrak, notching the first approval for rare eye cancer uveal melanoma and also the first drug of its class. Evidence of the regulatory support for the drug, it was approved a month before a response was due. In the first nine months of 2022, the drug raked in $83 million in revenue and is growing through sales in the U.S., France and Germany. Almost half of that came in the third quarter, and revenue jumped 20% compared to the second quarter. Those numbers are leagues ahead of projections, which estimated Kimmtrak would bring in a combined $62 million between 2022 and 2023. The company noted in its third-quarter earnings report that German public health officials gave the treatment a “considerable added benefit” rating, the second-highest possible and only the second time such a rating was issued for an orphan oncology medication. With that said, the cost of Kimmtrak is anything but cheap, with treatment costing roughly $400,000 on average.



Active ingredient: Faricimab-svoa Disease: Macular degeneration, diabetic macular edema Peak sales estimate: $3.6 billion Approved: Jan. 28 Company: Roche

Summary: Vabysmo's FDA endorsement was the first ever for an injectable to cover macular degeneration and diabetic macular edema simultaneously. But the biggest reason that Vabysmo will be an immediate threat to the dominant drug in the space—Regeneron and Bayer’s Eylea—is its staying power. While Eylea requires injection directly into the eye every two months, Vabysmo can last for up to four months. Uptake has been strong: Roche reported sales of 173 Swiss francs ($184 million) in the third quarter. But Eylea—which amassed sales of $8.9 billion last year—is battling to keep its market share. In September, Regeneron reported stunning results for its four-month formulation, indicating Eylea may be suitable for more patients than Vabysmo. Regeneron will use a priority voucher to expedite review of its four-month version, eyeing potential approval in August 2023. While a single shot of Vabysmo is more expensive than Eylea—approximately $2,300 versus $1,950—it becomes cheaper if it is dosed less frequently. The drug is anticipated to hit peak sales of $3.6 billion in 2028, according to Evaluate.



Active ingredient: Elasomeran Disease: COVID-19 Peak sales estimate: $19 billion for 2022 Approved: Jan. 31 Company: Moderna

Summary: Though Moderna’s Spikevax has been around under emergency authorization since December 2020, the COVID-19 shot grabbed full FDA approval for adults at the beginning of 2022. Spikevax was the second COVID-19 shot to get full FDA approval, behind Pfizer-BioNTech’s Comirnaty. A recent study flagged higher risks of heart inflammation tied to the Moderna jab versus the rival from Pfizer and BioNTech. In June, Spikevax got the green light for Americans as young as 6 months, and Moderna is currently building the case for its bivalent booster against the BA.4 and BA.5 omicron subvariants. For the third quarter of 2022, Spikevax garnered $3.4 billion for Moderna. The company lowered its full-year sales estimate to between $18 billion and $19 billion from its previous forecast of $21 billion for 2022, which would still be more than last year, when the vaccine raked in $17.7 billion for the biotech.



Active ingredient: Sutimlimab Disease: Cold agglutinin disease Peak sales estimate: $500 million Approved: Feb. 4 Company: Sanofi

Summary: Over the last five years, Sanofi has pivoted away from the cardio and diabetes fields and doubled down in rare diseases and specialty care. Part of that effort has been through M&A, and in 2018 the company inked an $11.6 billion deal to buy Bioverativ, a hemophilia-focused biotech. Fast-forward to 2022, and the top pipeline asset in that deal finally won its FDA nod. In February, Enjaymo snagged an FDA approval to treat the hemolysis—the destruction of red blood cells—that accompanies the rare blood disorder cold agglutinin disease (CAD). Enjaymo’s path to the market was not without hitches; in 2020, the FDA hit Sanofi with a complete response letter citing flaws at a contract manufacturer’s site. CAD affects roughly 5,000 people in the U.S., and Enjaymo is the first approved treatment for the disease. Evaluate Pharma has estimated the drug could generate $500 million by 2026.



Active ingredient: Mitapivat Disease: Hemolytic anemia associated with pyruvate kinase deficiency Peak sales estimate: $1 billion Approved: Feb. 17 Company: Agios

Summary: After pivoting away from cancer in 2020, Agios is starting to see its vision for its genetically defined diseases portfolio play out. In February, the company secured FDA approval for its first-of-its-kind pyruvate kinase activator for adults suffering from hemolytic anemia with PK deficiency. The approval came after Agios in 2020 sold off its cancer unit to Servier for $1.8 billion. With the FDA approval earlier this year, Agios once again entered the commercial arena. Around the time of approval, Cantor Fitzgerald analyst Alethia Young wrote that her team expected 2022 sales of $21 million and 2023 sales of $73 million. Aside from PK deficiency, Agios is also testing Pyrukynd in thalassemia and sickle cell disease. If the drug succeeds in those conditions, Pyrukynd could become a blockbuster, now-former Agios CEO Jackie Fouse, Ph.D., told The Boston Globe earlier this year.



Active ingredient: Ciltacabtagene autoleucel Disease: Multiple myeloma Peak sales estimate: $5 billion Approved: Feb. 28 Companies: Johnson & Johnson, Legend Biotech

Summary: While Johnson & Johnson and Legend Biotech’s CAR-T therapy Carvykti was a tad late to the regulatory finish line, the drug is quickly gaining market traction. In the third quarter, the cell therapy generated $55 million in sales, up from $24 million in the three months ending in June, which was also the first full quarter the multiple myeloma med was on the market. Still, that’s a far cry from the $5 billion in global sales J&J has prophesied. As for the med’s prospect over the next few years, Evaluate Vantage has pinned potential 2026 sales for Carvykti at around $1.69 billion. Unlike its chief rival from Bristol Myers Squibb—Abecma—Carvykti is undergoing a phased launch, with a gradual scale-up in availability and the number of treatment centers based on constrained manufacturing capacity. The partners also recently boosted their investment in a Raritan, New Jersey, cell therapy manufacturing facility to $500 million to get ahead of a potential demand spike should Carvykti move into earlier treatment.



Active ingredient: Pacritinib Disease: Myelofibrosis Peak sales estimate: $1 billion Approved: Feb. 28 Company: CTI BioPharma

Summary: Seattle-based drugmaker CTI BioPharma traveled a winding path to approval, but in late February finally scored an FDA nod for JAK2 and IRAK1 inhibitor Vonjo. Approved to treat the rare blood marrow cancer myelofibrosis, the drug could become a blockbuster over time. Specifically, the drug is the first to treat patients with cytopenic myelofibrosis, or those with a blood platelet count below a certain level. Vonjo costs $19,500 per month, or around $240,000 per year, before any rebates or discounts. CTI had to persist with the development of Vonjo, formerly known as pacritinib, for several years. In 2016, FDA slapped a clinical hold on Vonjo’s trials, asking for modifications. CTI BioPharma is also testing the drug in graft-versus-host disease. Citing Vonjo's price, analysts at BTIG wrote to clients that the drug could generate $1 billion in peak sales. For their part, Evaluate Pharma analysts project the drug will generate $368 million by 2026.



Active ingredient: Ganaxolone Disease: Cyclin-dependent kinase-like 5 deficiency disorder Peak sales estimate: $100 million Approved: March 18 Company: Marinus Pharmaceuticals

Summary: Ztalmy is the first drug approved to treat seizures associated with the rare genetic disease called cyclin-dependent kinase-like 5 deficiency disorder, which can impair brain development. The disorder usually becomes apparent in babies in their first few months of life. The thrice-a-day neuroactive steroid is approved for those age 2 and older and was launched in late July. It is the first approval for Marinus, which hopes to show that the therapy can treat other types of epileptic seizures. The company also has a government contract supporting its development of an intravenous formulation to tame refractory status epilepticus, the emergency convulsions that strike 150,000 people per year in the U.S. and require immediate treatment. Ztalmy is priced at $133,000 annually for the average-sized patient. The average age of recipients is 4.5 years, but the company expects that to escalate to 11 years as the treatment demonstrates success. In August 2021, Orion picked up European rights to Ztalmy for $30 million. It is on track for a decision in Europe in late 2023. SVB Leerink forecasts Ztalmy's peak sales will be $100 million.



Active ingredients: Relatlimab and nivolumab Disease: Melanoma Peak sales estimate: $4 billion Approved: March 18 Company: Bristol Myers Squibb

Summary: Approved last spring, Opdualag is a fixed-dose combination of Bristol Myers Squibb’s PD-1 inhibitor Opdivo and first-in-class LAG-3 antibody relatlimab. As BMS has done with its other checkpoint inhibitors, the company scored Opdualag's approval in melanoma. The drug is a key component in BMS’ plan to overcome the patent cliff for the blockbuster trio of Revlimid, Eliquis and Opdivo—three of the company’s top-selling drugs—this decade. BMS figures Opdualag could reach over $4 billion sales in 2029, but some of that will likely come from Opdivo and CTLA-4 agent Yervoy. Because relatlimab boasts a safety edge over Yervoy, BMS hopes to convince doctors who have balked at Yervoy’s toxicity, and have therefore stuck to single-agent Opdivoy, to start using the new therapy. BMS is also testing the new combo in various other cancer types.



Active ingredient: Lutetium lu 177 vipivotide tetraxetan Disease: Prostate cancer Peak sales estimate: $2 billion Approved: March 23 Company: Novartis

Summary: Just weeks after approval, manufacturing concerns blunted Novartis’ blockbuster ambitions for its radiotherapy arriviste Pluvicto. Approved in March for certain patients with metastatic castration-resistant prostate cancer, the med could eventually snare more than $2 billion in peak sales, its Swiss developer has predicted. But shortly after Novartis touted the med’s strong start in April, the company revealed it was pausing production on both Pluvicto and its neuroendocrine tumor therapy Lutathera in Italy and New Jersey. Novartis said it executed the move “out of an abundance of caution as a result of potential quality issues identified in its manufacturing processes.” As of late June, Novartis said it had resolved its radioligand production problems. With Pluvicto presumably back on course to blockbuster land, Novartis this summer said it would expand production capabilities at the same sites where it paused work in May. Pluvicto is one of 20 potential billion-dollar launches Novartis has planned by 2026 to drive midterm growth.



Active ingredient: Oteseconazole Disease: Recurrent vulvovaginal candidiasis Peak sales estimate: N/A Approved: April 26 Company: Mycovia Pharmaceuticals

Summary: When Mycovia Pharmaceuticals’ Vivjoa scored an FDA approval this spring, the drug became the first and only therapy approved for recurrent vulvovaginal candidiasis (RVVC), otherwise known as yeast infections. The agency endorsed the drug for females who have a history of RVVC and who are not able to become pregnant, including post-menopausal and permanently infertile women. The drug, an azole antifungal, is Mycovia’s only approved product and became commercially available in the U.S. in July. Vivjoa scored big in trials, reducing RVVC recurrence for nearly a year (50 weeks) versus comparator treatments after a 12-week course. As for the company's other prospects, Mycovia’s pipeline targets fungal infections both in women and in the general population, including onychomycosis, emerging drug-resistant fungi, cryptococcal meningitis and valley fever. The company has several ongoing phase 1 and phase 2 trials.



Active ingredient: Mavacamten Disease: Hypertrophic cardiomyopathy Peak sales estimate: $4 billion-plus (2029) Approved: April 28 Companies: Bristol Myers Squibb, MyoKardia

Summary: Camzyos, née mavacamten, formed the heart of Bristol Myers Squibb’s $13.1 billion buyout of MyoKardia two years ago—and the drugmaker hopes it will grow into its next cardiovascular blockbuster. The first-in-class therapy targets one of the most common genetic heart diseases: obstructive hypertrophic cardiomyopathy, or HCM, where a thickening of the heart muscle can block the flow of blood. As a myosin inhibitor, Camzyos disengages some of the cardiac tissue’s cellular motors that drive contractions, and puts a brake on the strength of each heartbeat. One of the most anticipated launches of the year, the drug is expected to pick up some of the slack from Eliquis and Revlimid, BMS’ current top sellers, after they go over the patent cliff this decade. BMS also plans to pursue indications for Camzyos in heart failure and nonobstructive HCM.



Active ingredient: Vonoprazan Disease: Helicobacter pylori infection Peak sales estimate: $200 million Approved: May 3 Company: Phathom Pharmaceuticals

Summary: Several years after its spinoff from Takeda, Phathom Pharmaceuticals entered the commercial arena in 2022 with an approval for anti-infective therapy Voquezna in two combinations. In the first, dubbed Voquezna Triple Pak, the drug is approved with amoxicillin and clarithromycin to treat Helicobacter pylori infection, which typically affects a person’s stomach or small intestine. The other combo, a dual combo with amoxicillin, also treats H. pylori infection. Ahead of the approvals, Phathom inked a financing deal worth up to $260 million to support the commercial launch and continued R&D. H. pylori is a common infection for which most people do not need to seek treatment. The infection may be present in half of adults worldwide, according to the Mayo Clinic, but most people don’t develop serious symptoms. In H. pylori, the drug carries a $200 million peak sales estimate, Jefferies analyst Caleb Ezell wrote at the time of approval. The company is also testing the drug in non-erosive reflux disease, which represents a larger potential market.



Active ingredient: Tirzepatide Disease: Type 2 diabetes Peak sales estimate: $25 billion Approved: May 13 Company: Eli Lilly

Summary: Eli Lilly’s Mounjaro was one of the most eagerly anticipated approvals of 2022, and it dutifully nabbed an FDA approval for Type 2 diabetes in May. But while new to the market, and a crowded one at that, Lilly isn’t resting on its laurels. Despite only just nabbing that diabetes label, Lilly is already busy planning for a second label in obesity, getting a rolling submission with the FDA in the fall. Should it gain an approval here—and max out sales in both disease states—analysts at UBS predict a major $25 billion in peak annual sales for the drug, eclipsing the record $20 billion seen for AbbVie’s Humira. It has major competition on both fronts from longtime rival Novo Nordisk, which has a suite of GLP-1 drugs that treat diabetes in Rybelsus and Ozempic, and, more recently, in Wegovy in obesity. Lilly still believes it can not only match but beat these drugs, which it will need to do if it wants to reach that eye-watering $25 billion figure.



Active ingredient: Tapinarof Disease: Psoriasis Peak sales estimate: $2 billion* Approved: May 24 Company: Dermavant

Summary: Opzelura, Zoryve, Otezla and Duobrii. These are all the meds that Dermavant launched Vtama against after being approved May 24 for psoriasis. The topical treatment has faced down the pressure, managing to outpace the launches of its competitors to find space in the crowded market, which also includes highly effective biologics like AbbVie’s Humira. The company’s executives heralded the launch, reporting “good progress on market access,” according to SVB Securities.

Dermavant, part of the Roivant cluster of companies, is still waiting to see how payers will view the therapy, but SVB predicts that Vtama will fall into place as a “step therapy” option prior to more expensive systemic medications in psoriasis. The company is seeing about 3,000 scripts per week so far. Jefferies previously estimated sales of $50 million in 2023. Vtama is also being tested in a phase 3 clinical trial for atopic dermatitis, which would be a much more lucrative market at three to four times the size, according to SVB.

* This estimate is combined with the potential atopic dermatitis indication.



Active ingredient: Live attenuated measles, mumps and rubella viruses Disease: Measles, mumps, rubella Peak sales estimate: $364 million Approved: June 3 Company: GSK

Summary: Priorix is likely the oldest product in this report. It was approved in Germany in 1997 and now is in use in more than 100 countries. Why is it just now reaching the U.S. as another option to Merck’s M-M-R II vaccine? With the pandemic disrupting doctor’s visits and medical care through schooling, fewer children received these standard-issue shots. As a result, measles has made a comeback. In April, the World Health Organization said that cases in January and February were up 79% compared to the same period in 2021 and called it a “worrying sign.” Priorix is taken in two doses, first between ages 12 and 15 months and next between ages 4 and 6. The approval was based on Priorix showing comparable efficacy and side effects to M-M-R II. Children’s vaccines aren’t big sellers. In 2021, GSK reported (PDF) sales of 260 million euros ($279 million) for its trio of Priorix, Varilrix (chickenpox) and Priorix Tetra (measles and chickenpox). Priorix's peak sales are expected to be $364 million, according to Evaluate Pharma.



Active ingredient: Vutrisiran Disease: Transthyretin amyloidosis Sales estimate: $1.8 billion in 2026 Approved: June 13 Company: Alnylam

Summary: Amvuttra is Alnylam’s next-generation RNA interference therapy that targets transthyretin mRNA. After a summer 2022 FDA nod, the drug is approved to treat hereditary transthyretin amyloidosis (hATTR) polyneuropathy. Compared with its predecessor Onpattro, which is given via intravenous infusion once every three weeks, Amvuttra can be administered via under-the-skin injections once every three months. Evaluate Vantage has previously put Amvuttra’s estimated 2026 sales at $1.8 billion, but that was before Onpattro succeeded in its own ATTR cardiomyopathy study. Compared with the Onpattro trial, Amvuttra’s HELIOS-B uses a hard endpoint measuring patients’ cardiovascular outcomes, is twice as big and follows patients nearly three times longer. Looking forward, the phase 3 HELIOS-B trial is expected to report data for Amvuttra in the all-important ATTR cardiomyopathy indication in early 2024. In the ATTR space, pharma giant Pfizer has also been working to build its presence with Vyndaqel and Vyndamax.



Active ingredient: Betibeglogene autotemcel Disease: Beta thalassemia Peak sales estimate: $206 million in 2027 (Raymond James) Approved: Aug. 17 Company: Bluebird bio

Summary: The gene therapy sector is off and running, and near the front of the pack is bluebird bio’s beta thalassemia treatment Zynteglo. The company nabbed FDA approval in August after a rough and tumble-process with European counterparts stalled out over the company’s $2.8 million price tag. A report from the Institute for Clinical and Economic Review estimates that there are as many as 1,500 people in the U.S. with transfusion-dependent thalassemia, but 1.25 million people with the genetic mutation that can cause the disease. Prior to bluebird’s approval, the only curative treatment was a blood stem cell transplant with a matched donor. With few options, the median life expectancy among patients with the disease is 37 years. Given that the company just hopped onto the U.S. market in the third quarter of 2022, there aren’t sales figures available yet. But bluebird says some nine patients have progressed to prior authorization of the treatment and estimates it will have its first blood removal in the fourth quarter.



Active ingredient: Olipudase alfa Disease: Niemann-Pick Peak sales estimate: $500 million Approved: Aug. 31 Company: Sanofi

Summary: Further cementing its rare disease credentials, the approval of Xenpozyme saw Sanofi bag the approval for a drug to treat the non-central nervous system manifestations of acid sphingomyelinase deficiency. The genetic disease is certainly rare, with fewer than 120 diagnoses in the U.S. each year. The drug proved its value in a randomized, placebo-controlled study of 31 patients, which showed it reduced the size of the spleen and liver and improved lung function. Sanofi pitched the drug, which is taken every two weeks, at a U.S. list price of $7,142 per vial. Jefferies analysts in 2021 assigned a $500 million peak sales target to the drug, with the caveat that there is a 50% chance the drug could reach that mark. The drug has already got a head start in Japan, where it launched in June, contributing to the 2 million euros ($2 million) Xenpozyme has already brought in through third-quarter sales (PDF).



Active ingredient: Spesolimab Disease: Generalized pustular psoriasis flares Peak sales estimate: $364 million Approved: Sept. 1 Company: Boehringer Ingelheim

Summary: Generalized pustular psoriasis (GPP) flares is a rare and life-threatening skin condition that can cause painful blisters. The flares can be so disruptive that they can trigger heart failure, renal failure or sepsis. Because it is so rare, GPP is under-diagnosed and is often mistaken for other skin disorders. Spevigo is an IL-36 receptor inhibitor and is the first drug approved to treat the disease. It also is Boehringer’s first nod for a dermatology treatment. Spevigo was sanctioned in Europe in October. Competition is likely on the way from another IL-36 receptor, AnaptysBio’s imsidolimab, which is scheduled to read out results from a phase 3 trial by the end of 2023. Spevigo got its thumbs-up thanks to a phase 2 trial that showed it cleared the skin of 54% recipients compared to 6% on placebo. Boehringer Ingelheim also is investigating Spevigo as a maintenance treatment and against other skin disorders such as palmoplantar pustulosis and hidradenitis suppurativa. The drug is forecast to reach peak sales of $364 million, according to Evaluate Pharma.



Active ingredient: DaxibotulinumtoxinA Disease: Glabellar lines Peak sales estimate: $1 billion Approved: Sept. 7 Company: Revance

Summary: The long-awaited competitor to AbbVie’s Botox finally got its green light. The regulatory nod came nearly two years after its original PDUFA date. Pandemic-related inspection delays, followed by manufacturing problems derailed the approval. Like Botox, Daxxify is an injected neuromodulator but it is the first product based on peptide exchange technology. The key differentiator is staying power. An injection lasts six to nine months versus three months for Botox. The market is huge. Botox, which was approved in 2002, generated revenue of $4.7 billion in 2021 between its cosmetic and therapeutic applications. Revance has yet to launch or designate a price for Daxxify. The company is using a pre-launch preview strategy to get it “into the hands of a small group of physicians that can use this product to understand how to get the best results,” CEO Mark Foley told Fierce Pharma. Daxxify is approved for frown lines, also known as glabellar lines, but Revance also is investigating the drug for its therapeutic benefits. An FDA submission is planned in cervical dystonia. The drug is forecast to reach peak sales of $1 billion by 2030, according to Cowen.



Active ingredient: Deucravacitinib Disease: Moderate to severe plaque psoriasis Peak sales estimate: $4 billion Approved: Sept. 9 Company: Bristol Myers Squibb

Summary: In another 2022 drug approval for Bristol Myers Squibb, Sotyktu scored a green light in September thanks to its impressive trial data. Pitted against placebo and Amgen's Otezla in plaque psoriasis studies, Sotytku came out on top. The results affirmed BMS' decision to keep the drug when the company bought Celgene and subsequently sold Otezla to Amgen. BMS reported (PDF) $1 million in revenues from Sotyktu during the third quarter, but the company thinks it has the potential to rake in $4 billion, or more, by the end of the decade, Chief Medical Officer Samit Hirawat, M.D, told Fierce Pharma. Sotyktu is also under review at the European Medicines Agency for moderate to severe plaque psoriasis and is approved in Japan for the same indication, plus pustular psoriasis and erythrodermic psoriasis. Midstage readouts in Crohn’s disease and ulcerative colitis are expected for next year. The approval followed the FDA's endorsement of BMS' Camzyos earlier in the year.



Active ingredient: Eflapegrastim-xnst Disease: Infections/neutropenia Peak sales estimate: N/A Approved: Sept. 9 Company: Spectrum Pharmaceuticals

Summary: Nothing has been easy for Spectrum Pharmaceuticals, but the approval of Rolvedon sealed its transformation into a commercial-stage company when the FDA nod arrived Sept. 9. Previously known as Rolontis, the therapy was approved to reduce infections that occur with neutropenia in cancer patients receiving chemotherapy. It was not an easy road to FDA approval. In 2021, the injection that would become Rolvedon, eflapegrastim-xnst, was kicked back by the agency because of manufacturing concerns, and layoffs at the company followed. But Spectrum put those woes aside and in October, Rolvedon became commercially available, launching into a market worth a potential $2 billion.

Let’s hope the same perseverance can pay off for Spectrum’s follow-up, lung cancer medication Pozenveo, aka poziotinib, which just received a bad omen from the FDA’s Oncologic Drugs Advisory Committee.



Active ingredient: Terlipressin Disease: Hepatorenal syndrome Peak sales estimate: $300 million Approved: Sept. 14 Companies: Mallinckrodt

Summary: First approved in Europe in 2010, Terlivaz finally got its U.S. nod just this year. Hepatorenal syndrome, which, when accompanied by a rapid reduction in kidney function and untreated, has a median survival of two weeks. It strikes between 30,000 and 40,000 people in the U.S. annually. HRS affects those with liver cirrhosis, constricting blood vessels in the kidneys and leading to a buildup of toxins. Terlivaz can lessen the need for dialysis. Mallinckrodt got the drug across the FDA finish line after two other companies failed. Under Orphan Therapeutics, Terlivaz flunked a phase 3 trial. Then Ikaria did the same after purchasing the drug’s rights for North America. When Mallinckrodt bought out Ikaria in 2016, the FDA encouraged the Ireland-based company to give it another shot. The drug was first due for a decision in 2020. While the FDA issued two complete response letters, the regulator has now finally blessed the treatment. The drug is forecast to reach peak sales of $300 million, according to Cantor Fitzgerald.



Active ingredient: Elivaldogene autotemcel Disease: Cerebral adrenoleukodystrophy Peak sales estimate: N/A Approved: Sept. 16 Company: Bluebird bio

Summary: About a year after folding its European commercial operations following reimbursement setbacks, bluebird bird won an FDA approval for gene therapy Skysona in the ultrarare neurological disorder of cerebral adrenoleukodystrophy (CALD). The one-time treatment’s $3 million price tag broke the record for the world’s most expensive therapy merely weeks after sister gene therapy Zynteglo’s $2.8 million revelation. As reflected in an FDA black box warning, Skysona is linked to a risk of blood cancers, which are suspected to be caused by the lentiviral vector that’s used to deliver the gene therapy. But the lack of treatment options tilted the benefit-risk profile in the bluebird drug’s favor. Altogether about 40 patients with CALD in the U.S. could be eligible for Skysona each year, according to bluebird’s estimate. Unlike with Zynteglo, bluebird isn't offering an outcomes-based payment program for Skysona because the rarity and complexity of CALD makes such an arrangement “extremely challenging to implement for both bluebird and payers,” CEO Andrew Obenshain told investors in September.



Active ingredient: Gadopiclenol Disease: Contrast-enhanced MRI Peak sales estimate: N/A Approved: Sept. 21 Company: Guerbet

Summary: Elucirem aims to usher in a new generation of MRI imaging agents by relying on half the dose of the rare earth metal gadolinium. The FDA approved Guerbet’s injection, which makes it easier to see abnormally vascular lesions on a scan, after granting it priority review. The agency had previously issued warnings for the entire class of gadolinium-based contrast agents—saying they can be retained by the body and the brain for years—though they have not been linked directly to adverse effects. Still, Elucirem is designed to address clinicians’ concerns about gadolinium exposure. France-based Guerbet has a long history of GBCAs, with its Dotarem first launched in 1989 and Artirem in 2002. The company said it plans to produce Elucirem in North Carolina through its Liebel-Flarsheim subsidiary and is awaiting approval for the contrast agent in Europe.



Active ingredient: Omidenepag isopropyl Disease: Primary open-angle glaucoma and ocular hypertension Peak sales estimate: $145 million Approved: Sept. 22 Companies: Santen and UBE

Summary: At the end of September, for the second year in a row, Santen picked up an FDA approval for a new eye drop formulation. Evaluate estimated that Omlonti could reach $145 million in sales by 2028, as it caters to the nearly 80 million people around the world with either primary open-angle glaucoma or ocular hypertension, which can lead to elevated intraocular pressure—the specific condition Omlonti aims to treat. However, Santen has yet to announce pricing or a U.S. launch date for the ophthalmic solution, with CEO Takeshi Ito noting in a November earnings call that it’s planning to refocus its hemorrhaging business before moving forward with any expansion efforts in the region: “Basically, I believe that in order to break into the U.S. market, we must enter with products that are highly differentiated and have decisive product appeal,” he said (PDF) on the call, adding, “Preparations are now underway in the U.S. for the launch of this product. On the other hand, I believe that the resulting label makes it difficult to differentiate between this and conventional prostaglandins.”



Active ingredient: Sodium phenylbutyrate, taurursodiol Disease: Amyotrophic lateral sclerosis Peak sales estimate: $1 billion-plus Approved: Sept. 29 Companies: Amylyx

Summary: Amylyx’s amyotrophic lateral sclerosis (ALS) drug finally secured an FDA nod in 2022, though it wasn’t without significant challenges. In March, an initial FDA advisory committee meeting ended with a negative 6-4 vote for the drug based off phase 2 trial data. However, the committee reconvened in September to discuss a new analysis Amylyx had submitted. At the meeting, some panelists voiced a desire to wait for the results of an ongoing phase 3 trial assessing Relyvrio, though the committee ultimately ended with a positive 7-2 vote.The oral drug is the first ALS treatment for adults to significantly slow both disease progression and functional decline in a randomized clinical trial. The drug will carry a list price of about $158,000 per year in the U.S. SVB Securities analyst Marc Goodman projected that the drug will bring in more than $1 billion in U.S. sales in 2026.



Active Ingredient: Futibatinib Disease: Cholangiocarcinoma Peak sales estimate: $80 million Approved: Sept. 30 Company: Taiho Oncology

Summary: Now approved and sporting the flashy title Lytgobi, FGFR2 inhibitor futibatinib has a tough life ahead in cholangiocarcinoma. That’s because the drug, developed by Otsuka subsidiaries Taiho Oncology and Taiho Pharmaceutical, is wading into an increasingly crowded bile cancer crucible where it will have to contend with Incyte’s Pemazyre (pemigatinib) and BridgeBio and QED Therapeutics’ Truseltiq (infigratinib). Given that competition, analysts don’t expect Lytgobi to lead the market by sales in cholangiocarcinoma. GlobalData has suggested the drug could reach $50 million in 2028 revenue, according to reports, while Evaluate Vantage has prophesized a 2028 sales high of $80 million. Lytgobi bagged an accelerated approval in late September courtesy of the FDA’s Real-Time Oncology Review pilot. To maintain the med’s green light, Taiho has said it may have to validate Lytgobi’s benefits in confirmatory trials. The approval leveraged data from the midphase study FOENIX-CCA2, where Taiho’s med yielded a 42% objective response rate, meeting the trial’s primary endpoint. Lytgobi also charted a 9.7-month median duration of response.



Active ingredient: Tremelimumab Disease: Liver cancer Peak sales estimate: N/A Approved: Oct. 21 Company: AstraZeneca

Summary: The history of failure for AstraZeneca’s CTLA-4 inhibitor Imjudo runs long, encompassing lung cancer, head and neck cancer and bladder cancer. But after years of trying, AstraZeneca finally won an October FDA approval for the checkpoint inhibitor in a novel regimen alongside PD-L1 agent Imfinzi in liver cancer. The go-ahead came in unresectable hepatocellular carcinoma, and the nod features a priming dose of Imjudo added to Imfinzi, followed by Imfinzi monotherapy. The AZ combo represents the first pure immunotherapy in liver cancer, but it still needs to compete with Roche’s Tecentriq and Avastin in the field. The good news is that AZ doesn’t have to worry about Merck’s Keytruda or Bristol Myers Squibb’s Opdivo as both have failed in liver cancer. Other competitors such as the partnerships between Novartis and BeiGene, and between Jiangsu Hengrui Pharma and Elevar Therapeutics, are emerging.



Active ingredient: Teclistamab Disease: Multiple myeloma Peak sales estimate: More than $2 billion Approved: Oct. 25 Company: Johnson & Johnson

Summary: Tecvayli is the first BCMAxCD3 bispecific antibody approved by the FDA, but, for Johnson & Johnson, it’s yet another weapon in the company's multiple myeloma arsenal, following CD38 antibody Darzalex and Legend Biotech-partnered CAR-T therapy Carvykti. Tecvayli comes with an FDA-mandated safety monitoring program, which SVB Securities analysts argue might give some community physicians pause. Still, the drug’s “compelling efficacy, likely unconstrained supply, and ease of adoption relative to CAR-T therapies” could drive high demand, the SVB team said. Tecvayli is currently only approved as a fifth-line therapy, but J&J is combining it with Darzalex in earlier treatment, and that potential has SVB pegging over $2 billion in peak sales for Tecvayli. In Europe, Tecvayli is approved after three prior lines of treatment. J&J priced its new multiple myeloma drug at $39,500 per month and expects the therapy to cost between $355,500 and $395,000 on average per year before any rebates or discounts.



Active ingredient: Mirvetuximab soravtansine Disease: Advanced ovarian cancer Peak sales estimate: $300 million Approved: Nov. 14 Company: ImmunoGen

Summary: It took 41 years for ImmunoGen to score its first FDA approval for a wholly owned product. Its Elahere approval also marked the first antibody-drug conjugate for platinum-resistant disease. It also is the first FDA nod in eight years for advanced ovarian cancer. Importantly, Elahere can be used regardless of prior treatment with Roche’s Avastin. The broad label opens Elahere to approximately 5,200 patients in the U.S. ImmunoGen also is working toward an approval of Elahere plus Avastin, which would add another 1,800 eligible patients. Accompanying the endorsement is another from the FDA for a diagnostic to identify patients eligible for Elahere: Roche’s Ventana FOLR1 RxDx Assay. ImmunoGen priced Elahere at $6,220 for a 100-mg dose, with patients typically receiving three or four vials in a cycle of treatment. A trial of 106 patients showed that Elahere shrunk tumors in 32% versus a 12% figure seen in an earlier trial of single-agent chemotherapy. BMO Capital Markets forecasts the drug to have peak sales of $300 million.



Active ingredient: Teplizumab Disease: Type 1 diabetes Peak sales estimate: $2 billion Approved: Nov. 18 Company: Provention Bio

Summary: Thirty-six years after the creation of teplizumab, Provention Bio was able to get the drug across the FDA finish line. Tzield, as it's branded, became the first treatment to delay the progression of Type 1 diabetes. The green light is for those ages 8 and older who are in stage 2 of the disorder. Testing showed that patients on the infused treatment moved from stage 2 to stage 3 diabetes 25 months later than those on placebo. Stage 3 can present significant health risks including potentially fatal ketoacidosis. There are roughly 30,000 patients in the U.S. who will become immediately eligible for Tzield, but there are an estimated 1.4 million with the condition. Tzield will be listed at $13,850 per vial. Sanofi will market the drug in the U.S. after ponying up $35 million for licensing rights in October. Tzield was conceived by Jeff Bluestone, Ph.D., at the University of California, San Francisco and has made the rounds from MacroGenics to Eli Lilly in 2007 to Provention in 2011. Bluestone joined Provention’s board in 2019. The drug is expected to make $2 billion in peak sales, according to Illumination Capital.



Active ingredient: Etranacogene dezaparvovec-drlb Disease: Hemophilia B Peak sales estimate: $1.2 billion Approved: Nov. 22 Companies: CSL and uniQure

Summary: UniQure made history in 2012, securing the world’s first approval of a gene therapy, which is called Glybera. Ten years later, the company’s CSL Behring-partnered Hemgenix made a name for itself as the most expensive drug in the world at $3.5 million. The one-time infusion also became the first gene therapy approved to treat hemophilia B. CSL Behring maintains that despite the high price, the drug will generate “significant cost savings for the overall healthcare system” as well as lower the “economic burden of hemophilia B,” the company said in an email to Fierce Pharma shortly after approval. Patients with the genetic bleeding disorder would typically undergo regular prophylaxis to maintain enough factor IX, a protein needed to create blood clots. Results of the HOPE-B trial, the largest ever conducted in the patient population, showed 94% of the 54 participants stopped using prophylaxis after treatment with Hemgenix. The drug is expected to hit peaks sales of $2.4 billion in 2030, according to SVB Securities.



Active ingredient: Fecal microbiota, live-jslm Disease: Clostridium difficile Peak sales estimate: Unknown Approved: Nov. 30 Company: Ferring Pharmaceuticals

Summary: As the first FDA-approved fecal microbiota product, Rebyota represents a significant milestone, potentially clearing the way for other such treatments. The single-dose, enema-delivered therapy is designed to restore proper bacterial balance in the digestive system for those with Clostridium difficile. The antibiotic-resistant disorder affects roughly 500,000 people annually, causing between 15,000 and 30,000 deaths in the U.S. To address the condition, patients have turned to loosely regulated investigational fecal microbiota transplants, performed through colonoscopy or upper endoscopy. In September, an FDA advisory committee voted 13-4 to recommend Rebyota for approval, which would help standardize treatment. The subsequent FDA nod was based on six studies that included more than 1,000 participants and showed 70% were free of infection within eight weeks compared to 58% in the placebo group. Switzerland-based Ferring beat out Seres Therapeutics, which is targeting an April 2023 decision for its microbiome therapy in C. diff. Ferring picked up Rebyota in a 2018 acquisition of Minnesota-based Rebiotix.



Active ingredient: Olutasidenib Disease: Acute myeloid leukemia Peak sales estimate: Unknown Approved: Dec. 1 Company: Rigel Pharmaceuticals

Summary: Coming in more than two months before its PDUFA date, Rezlidhia is an unexpected addition to this year’s list. The approval also arrived just four months after Rigel gained rights to the drug in a deal that will pay Forma Therapeutics $235 million if commercial milestones are met. The nod is for adults with relapsed or refractory acute myeloid leukemia with isocitrate dehydrogenase-1 (IDH1) as detected by Abbott’s RealTime IDH1 diagnostic tool. The oral treatment was approved based on a study showing it provided a 35% complete remission rate and a median duration of 26 months. Rezlidhia will compete with the only other drug approved specifically for the condition: Servier’s Tibsovo, which was approved in 2018 and generated $120 million for Agios in 2020 before Servier purchased the company for $1.8 billion. Rezlidhia is Rigel’s second approved drug. In 2018, it received a green light for Tavalisse, which treats a rare bleeding condition.



Active ingredient: Adagrasib Disease: Non-small cell lung cancer Peak sales estimate: $1.3 billion Approved: Dec. 12 Company: Mirati Therapeutics

Summary: Mirati's Krazati became just the second cancer drug on the market that targets the KRAS mutation. With that December approval, Mirati is finally ready to take on Amgen’s Lumakras, which was blessed by the FDA in May 2021 for the same indication: previously treated KRAS G12C-mutated non-small cell lung cancer (NSCLC). Krazati also marks Mirati’s first commercial product and carries blockbuster potential, according to J.P. Morgan analyst Eric Joseph, Ph.D. Krazati goes for a list price of $19,750 for a 30-day supply, compared to $17,900 for Lumakras. Krazati was endorsed based on it shrinking tumors in 43% of patients in a phase 2 trial, compared to a 36% rate for Lumakras. Just like Amgen’s drug, Krazati is undergoing a phase 3 confirmatory trial—comparing the drug to the chemotherapy docetaxel—and using a smaller dose than that in the phase 2 study. Mirati also plans to launch two phase 3 trials of Krazati in combination with Keytruda in front-line NSCLC this year.



Active ingredient: Nadofaragene firadenovec Disease: Bladder cancer Peak sales estimate: N/A Approved: Dec. 16 Company: Ferring Pharmaceuticals

Summary: Even after an FDA rejection in May 2020, Ferring persisted in advancing its bladder cancer gene therapy. Its efforts paid off, and the drug finally made it past the agency’s gatekeepers in late 2022. Adstiladrin is cleared for high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer with carcinoma in situ with or without papillary tumors. In granting the approval, the FDA cited data showing more than half of patients who received the drug achieved a complete response. Adstiladrin is given every three months into the bladder via a urinary catheter. The gene therapy offers patients a new treatment in a cancer setting where few effective options exist. Historically, patients who develop BCG-unresponsive disease would undergo bladder removal surgery, Ferring Chief Science Officer Armin Metzger, Ph.D., said in an interview. On the market, Adstiladrin will go up against Merck & Co.’s Keytruda.



Active ingredient: Lenacapivir Disease: HIV-1 Peak sales estimate: $1.5 billion Approved: Dec. 22 Company: Gilead Sciences

Summary: Gilead Sciences is adding to the wide range of HIV treatments with Sunlenca, which sets itself apart thanks to its long-acting treatment method. Patients only require two treatments a year, once every sixth months, to achieve viral suppression. It’s cleared for patients whose virus has become multidrug-resistant. Viral suppression occurs when the virus is reduced to undetectable levels, which makes people unable to spread the virus, Jared Baeten, M.D., Ph.D., Gilead’s vice president of clinical development, said in an interview with Fierce Pharma. It’s been a long road for the drug. It first scored breakthrough therapy designation back in May 2019. Earlier this year, the drug and 10 studies of it escaped an FDA clinical hold placed after the agency found a risk that the borosilicate vials Sunlenca was being stored in could interact with the drug and create “sub-visible” glass particles. The company then switched to vials made from aluminosilicate glass. The approval is “just the beginning” as the company looks to work on longer-acting treatment and prevention options for wider groups, Baeten said. Peak sales are expected to be around $1.5 billion, Morningstar analyst Karen Andersen said in an October analyst note.



Active ingredient: Mosunetuzumab Disease: Follicular lymphoma Peak sales estimate: $2 billion Approved: Dec. 23 Company: Roche

Summary: The first-in-class bispecific antibody was endorsed as a third-line therapy for follicular lymphoma, joining an interesting competition with three gene therapies—Novartis’ Kymriah, Bristol Myers Squibb’s Breyanzi and Gilead’s Yescarta. While Lunsumio can’t quite match the efficacy of the CAR-T treatments, it has a considerable edge in convenience, accessibility and, presumably, price, though Roche has not divulged what it will charge for the infused drug. While Lunsumio shrank tumors in 80% of patients, with a complete response rate of 60%, Yescarta’s corresponding figures, for example, come in at 94% and 79%, respectively. Approved under the accelerated pathway, Lunsumio will be subject to a confirmatory phase 3 trial. Lunsumio was endorsed in Europe for the same indication in June of this year. Roche has another bispecific from the same class, glofitamab, which has shown promise in diffuse large B-cell lymphoma, the most common form of lymphoma. Both drugs could reach peak sales of $2 billion, according to analysts at Jefferies.



Active ingredient: Xenon Xe 129 hyperpolarized Disease: MRI lung ventilation Peak sales estimate: N/A Approved: Dec. 23 Companies: Polarean

Summary: Xenoview is a hyperpolarized contrast agent used in magnetic resonance imaging (MRI) for evaluation of lung function in those age 12 and older. The agent is inhaled and held for 10 to 15 seconds, allowing for the regional mapping of lung ventilation. It is the first imaging agent for lung analysis that does not expose patients to radiation and its associated risks. Chronic lung disease affects more than 30 million people in the U.S., and there is unmet need for noninvasive diagnostics. Xenoview has not been evaluated for lung perfusion imaging. The approval comes along with the FDA signing off on two companion devices: Xenoview VDP image processing software and Xenoview 3.0T chest coil. The endorsement was based on results from two trials that compared Zenoview MRI favorably to xenon Xe 133 scintigraphy in adults with pulmonary disorders. Medical imaging technology company Polarean said Xenoview will become available in the U.S. in January.



Active ingredient: Ublituximab Disease: Multiple sclerosis Peak sales estimate: N/A Approved: Dec. 28 Companies: TG Therapeutics

Summary: The CD20-targeting monoclonal antibody was approved for relapsing forms of multiple sclerosis including clinically isolated syndrome, relapsed-remitting disease and active secondary progressive disease. It is dosed just twice a year, providing a convenience edge over other anti-CD20 treatments such as Ocrevus (Roche) and Kesimpta (Novartis). New York-based TG expects to launch Briumvi in the first quarter of 2023. While the company has not disclosed a price, Jefferies analysts expect it to cost $30,000 annually, which is less than half the price of Ocrevus. Sales by 2024 could reach $1 billion, according to Seeking Alpha. The FDA endorsement came five months after the agency pulled back its approval for TG’s lymphoma drug Ukoniq because of an increased risk of death. The concerns were raised in a phase 3 trial, testing Ukoniq in combination with Briumvi as a treatment for both chronic lymphocytic leukemia and small lymphocytic leukemia.



Active ingredient: Anacaulase-bcdb Disease: Thermal burns Peak sales estimate: N/A Approved: Dec. 29 Companies: MediWound and Vericel

Summary: Israel-based MediWound earned the final FDA approval of 2022 with its topical treatment for second- and third-degree thermal burns. The biological product selectively removes eschar (dead tissue) without harming surrounding viable tissue. As a nonsurgical option, it has the potential to become the standard of care for those with deep partial and/or full-thickness burns, MediWound said. Thermal burns occur with contact with boiling water, fire, steam, hot cooking oil or heated metals that can raise the temperature of the skin, causing tissue cell death or charring. Its U.S. submission was supported by a $9 million grant from BARDA. Massachusetts-based Vericel will commercialize NexoBrid in North America. MediWound received a $7.5 million milestone payment from Vericel upon the approval. NexoBrid will become available in the U.S. by the second quarter of 2023, Vericel said. It has been in use in Europe for 10 years but adoption has been slow. Evaluate Pharma's sell-side consensus puts sales potential at $71 million by 2024.

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