Every drug approved in 2022: A year of fewer new products, but gene-therapy firsts and new blockbusters

The year’s crop of US drug approvals won’t rival past vintages for quantity. But of the 45 drugs cleared for use by the FDA in 2022, a third are shaping up to be blockbusters, with high expectations for Lilly’s new diabetes drug, Bristol Myers Squibb’s TYK2 inhibitor and Gilead’s new HIV treatment.

The promise of gene therapy became a reality in 2022, with new funding for the FDA to work through its backlog of applications, and three new approvals for bluebird bio (for beta thalassemia and early cerebral adrenoleukodystrophy) and CSL (for hemophilia B). With those approvals came new breakthroughs in price: The $2 million-plus treatment costs are likely to further test the bounds of the commercial market.

One trend stayed constant: Oncology dominated the pack in 2022, with nine new treatments hitting US doctors’ armamentaria for everything from uveal melanoma to multiple myeloma to acute myeloid leukemia to relapsed or refractory follicular lymphoma.

Critics will point to this year’s shortened list as a sign that the FDA’s approval process is becoming more difficult or rigorous. But the potential behind at least a dozen of the new drugs shows how the biopharma industry may be intensifying its focus on only the cream of the crop as pricing pressures hit a fever pitch with new Medicare negotiations coming soon.

Looking ahead, an Alzheimer’s approval for Eisai and Biogen should be in the books come early January, testing CMS’ payment denial decision for all amyloid-targeted drugs, and expectations are high for new cancer drugs and indications as well. — Zachary Brennan

1. QUVIVIQ

Active ingredient: daridorexant

Indication: Insomnia

Designations: N/A

Snapshot: Quviviq is Idorsia’s first FDA-approved drug since the company was spun out of Actelion in 2017. Its label marks what some analysts considered a best-case scenario, with no black box or addiction safety warning, and no notable risks for the higher of two approved doses. As it joins two other dual orexin receptor antagonists, or DORAs, from Eisai and Merck on the market to treat insomnia, the lack of side effects was a key factor that execs hoped would help Quviviq stand out. Late-stage trials were mostly positive, with both doses showing improvements in brain wave tests as well as self-reported sleep. While the company works toward the drug’s blockbuster aspirations, it has sold regional rights to China’s Simcere for $30 million upfront. — Amber Tong

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2. CIBINQO

Active ingredient: abrocitinib

Indication: Refractory, moderate-to-severe atopic dermatitis

Designations: N/A

Snapshot: Early in his tenure as Pfizer CEO, Albert Bourla had confidently put the JAK1 inhibitor abrocitinib at the top of a list of blockbuster drugs in the late-stage pipeline, predicting peak sales of $3 billion. Despite serious concerns about safety — which have plagued the whole JAK class — and a delay in the FDA’s review, the pharma giant still steered the once-a-day pill toward an approval. Pfizer, cognizant of the competitors in the atopic dermatitis space, released multiple rounds of data, including head-to-head comparisons with Sanofi and Regeneron’s Dupixent. But with AbbVie’s Rinvoq, Eli Lilly’s Olumiant and others also in the mix, it remains to be seen how well doctors and patients will receive Cibinqo. — Amber Tong

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3. KIMMTRAK

Active ingredient: tebentafusp-tebn

Indication: Unresectable or metastatic uveal melanoma

Designations: Breakthrough Therapy, Priority Review

Snapshot: Immunocore can lay claim to a series of firsts with the approval of Kimmtrak. Not only is it the first T cell receptor bispecific engager to be endorsed by the FDA, but it’s also the first approved therapy for unresectable or metastatic uveal melanoma, a rare cancer of the eye — which makes it a first for bispecifics in solid tumors, too. When compared to checkpoint inhibitors and chemotherapy, Kimmtrak significantly extended overall survival. It took 14 years and a long roller coaster ride, complete with a major shakeup, to get here, but Immunocore is hoping this will only be the first of many new T cell engagers to come. — Amber Tong

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4. VABYSMO

Active ingredient: faricimab-svoa

Indication: Neovascular (wet) age-related macular degeneration and diabetic macular edema

Designations: N/A

Snapshot: Genentech’s rival to Regeneron’s Eylea was approved early in 2022, with many analysts at first not entirely certain how it would fare. But in September, Roche revealed that it expected the drug’s sales to reach $2.3 billion by 2025. While the battle between the two drugs is ongoing, Vabysmo works slightly differently. In addition to blocking the receptor VEGF as Eylea does, Vabysmo also inhibits another blood-vessel growth regulator dubbed angiopoietin-2, or Ang-2. It holds a clear advantage over Eylea in dosing, as well, and can be given every 16 weeks, compared to every 8 or 12 weeks for Eylea. — Tyler Patchen

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5. SPIKEVAX

Active ingredient: Covid-19 vaccine, mRNA

Indication: Active immunization to prevent coronavirus disease 2019 (Covid-19) caused by severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) in individuals 18 years of age and older

Designations: Fast Track

Snapshot: Spikevax’s approval was simultaneously historic and unremarkable. While Moderna’s Covid-19 mRNA vaccine got its emergency use authorization in late 2020, the BLA rolled in at the end of January 2022, long after the shot’s distribution efforts were in full swing. The approval came through five months after Moderna filed its application. That was quick, but slower than Pfizer’s full approval for its Covid vaccine in August 2021, about three months after the Big Pharma filed, as the government tried to quell vaccine hesitancy. The vaccine isn’t commercially available yet and is still distributed by the government, but Moderna and Pfizer have suggested significant price hikes to $100 per shot once they do begin to sell on the market. — Lei Lei Wu

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6. ENJAYMO

Active ingredient: sutimlimab-jome

Indication: To decrease the need for red blood cell transfusion due to hemolysis in cold agglutinin disease

Designations: Breakthrough Therapy, Orphan Drug

Snapshot: Enjaymo had quite the journey in getting the thumbs-up from the FDA in February. The drug was the lead asset in Sanofi’s 2018 acquisition of Bioverativ for $11.6 billion, but the agency rejected the compound two years later, citing manufacturing issues. After a positive Phase III readout showed that 54% of participants responded to the drug, Enjaymo was cleared, giving Sanofi the only approved treatment for people with cold agglutinin disease, a rare autoimmune disorder. — Tyler Patchen

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7. PYRUKYND

Active ingredient: mitapivat

Indication: Hemolytic anemia in pyruvate kinase deficiency

Designations: Orphan Drug, Fast Track, Priority Review

Snapshot: In 2021, Agios took a gamble and sold off its entire oncology portfolio, including the drug Tibsovo, to Servier for $1.8 billion in cash. At the time of the sale, the company said it was focused on what would eventually become Pyrukynd. The FDA cleared the drug in the first quarter of 2022 for use in treating the rare blood disorder pyruvate kinase deficiency, in which red blood cells are destroyed faster than they can be made. It’s the first disease-modifying therapy approved for the condition. — Tyler Patchen

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8. VONJO

Active ingredient: pacritinib

Indication: Intermediate or high-risk primary or secondary myelofibrosis in adults with low platelets

Designations: Accelerated Approval, Priority Review, Fast Track, Orphan Drug

Snapshot: CTI got its first drug approval in Vonjo, a JAK inhibitor for a rare blood cancer. But it doesn’t come with a black box warning that others in the same class have been issued, thanks in part to how it works. The drug targets two points in an important immune system pathway, JAK2 and IRAK1, without hitting a third, JAK1. Vonjo’s accelerated approval came from data from a Phase III study in which around one-third of myelofibrosis patients saw their spleen volume reduced by at least 35% compared with 3% on best available therapy. However, Vonjo enters a field in which two other JAK inhibitors from more established players have already been approved — Incyte’s Jakafi and Bristol Myers Squibb’s Inrebic. According to CTI’s Q3 report, Vonjo has made $32.9 million since its launch. But still coming up for CTI is Vonjo’s full approval, which hinges on data expected in mid-2025. — Lei Lei Wu

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9. CARVYKTI

Active ingredient: ciltacabtagene autoleucel

Indication: Adult patients with relapsed or refractory multiple myeloma after four or more prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody

Designations: Breakthrough Therapy, Orphan Drug

Snapshot: Carvykti was the first biologic and CAR-T cell therapy to be approved by the FDA in 2022, and will take on Bristol Myers Squibb’s Abecma. The therapy first jumped on the scene in 2017 as an ASCO showstopper after Legend showed a 100% ORR and 94% clinical remission in 35 relapsed, drug-resistant multiple myeloma patients. Those results were enough for Janssen to step in and offer $350 million in cash to partner up. More data at ASCO in 2022 showed that the therapy stayed effective near the 28-month mark, this time touting a 98% ORR in 97 patients. Approved as a fifth-line treatment, it only netted about $55 million in its first quarter on the market. However, Legend CEO Ying Huang said just a couple of weeks ago that with a planned manufacturing revamp over the next three or four years, the companies will be able to secure more than $5 billion in sales. — Paul Schloesser

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10. ZTALMY

Active ingredient: ganaxolone

Indication: Seizures in cyclin-dependent kinase-like 5 deficiency disorder

Designations: Orphan Drug, Priority Review

Snapshot: Marinus enters its 20th year in 2023 with a single drug approval in hand, which it won for the seizure drug Ztalmy. The biotech labored for years (and years) to build positive data around its candidate. In 2019, another Phase II study failed. Given one more year, a Phase III study in CDKL5 deficiency disorder, or CDD, injected new life into the drug candidate. In that study, patients who got Ztalmy had a median 30.7% reduction in the frequency of their major motor seizures over 28 days compared to a 6.9% decrease for those on placebo, setting the stage for its singular approval. — Lei Lei Wu

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11. OPDUALAG

Active ingredients: nivolumab and relatlimab-rmbw

Indication: Unresectable or metastatic melanoma

Designations: Priority Review, Fast Track, Orphan Drug

Snapshot: In clearing Opdualag, the FDA approved a new class of checkpoint inhibitors for the first time in eight years. Opdualag is an infusion of two antibodies — Bristol Myers Squibb’s blockbuster PD-1 inhibitor Opdivo and a new antibody known as relatlimab. Relatlimab is a checkpoint inhibitor but targets a different T cell protein in LAG-3. In doing so, it blocks the immune system’s “off” signal from being broadcasted, freeing T cells to attack tumor cells. The combo antibody infusion was approved based on data from 714 melanoma patients, showing those on the combo drug had a median of 10.1 months of PFS, compared to 4.6 months on Opdivo alone. Two oncologists wrote in the New England Journal of Medicine that if the future survival data for the LAG-3 antibody in combination with Opdivo looked similar to the CTLA-4 combo, the former could become “the new standard of care for previously untreated patients with advanced melanoma.” — Lei Lei Wu

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12. PLUVICTO

Active ingredient: lutetium (177Lu) vipivotide tetraxetan

Indication: Prostate-specific membrane antigen-positive metastatic castration-resistant prostate cancer following other therapies

Designations: Breakthrough Therapy

Snapshot: After showing that its drug could extend patients’ lives four months longer than the current treatment and reduce the risk of death by 38% when combined with the best standard-of-care, Novartis won approval for Pluvicto. The initial nod allows patients with the difficult-to-thwart cancer to receive the treatment after they have received other therapies. By December, Novartis had data showing the radioligand treatment worked in earlier lines as well, setting it up for another regulatory filing in 2023, five years after acquiring the asset via its $2.1 billion Endocyte buyout. The green light adds to Novartis’ experience in the field with Lutathera, as the radioligands aim to strike cancer directly at the source through a maximum of six infusions. — Kyle LaHucik

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13. VIVJOA

Active ingredient: oteseconazole

Indication: To reduce the incidence of recurrent vulvovaginal candidiasis (RVVC) in females with a history of RVVC who are not of reproductive potential

Designations: Fast Track, Qualified Infectious Disease Product

Snapshot: For a while, there was no treatment for recurrent vulvovaginal candidiasis, better known as chronic yeast infection. The acute episodes can occur at least three times in the span of 12 months, including vaginal itching, burning and other irritative symptoms, and lead to abnormal vaginal discharge. After conducting two global Phase III trials and a US-focused late-stage study, Mycovia got the all-clear to market the first tablets for the disease, but only to people who are “NOT of reproductive potential.” The antifungal became available in July, and Mycovia’s partner Jiangsu Hengrui Pharmaceuticals is testing the capsules in China. In the waning months of 2022, Mycovia’s competitor Scynexis received an expanded label for its own antifungal, known as Brexafemme, for the reduction in the incidence of recurrent vaginal yeast infection. Mycovia might have a leg up, though, as its New Jersey competitor laid off employees earlier in the fall to focus on antifungals for the hospital setting. — Kyle LaHucik

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14. CAMZYOS

Active ingredient: mavacamten

Indication: Certain classes of obstructive hypertrophic cardiomyopathy

Designations: Breakthrough Therapy, Orphan Drug

Snapshot: At the heart of Bristol Myers’ $13 billion MyoKardia buyout was Camzyos, known as mavacamten at the time of the 2020 deal. The Big Pharma went through hiccups getting the drug across the finish line after bringing the asset into the fold. The FDA extended its decision by a quarter to assess the proposed Risk Evaluation and Mitigation Strategy after nine patients had paused the treatment in a Phase III trial due to pumping out too little blood from their left heart ventricles. Eight of those patients returned to treatment. The agency cleared it in April, and by October the drug giant was already on the docket at FDA for an expanded label decision for a subset of patients with the condition in which blood flow is hampered by thickened heart muscle. — Kyle LaHucik

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15. VOQUEZNA

Active ingredients: vonoprazan, amoxicillin, and clarithromycin

Indication: Helicobacter pylori infection

Designations: Qualified Infectious Disease Product, Priority Review

Snapshot: Voquezna is the newest brainchild of Phathom Pharmaceuticals, a spinout build-out of an alliance between Takeda — right after it acquired Shire — and the healthcare VC Frazier. The heart of the approved drug is vonoprazan, a member of a class of drugs that blocks ATPase (the enzyme responsible for acidification in the stomach) and is developed by Takeda. Vonoprozan was approved in Japan back in 2015 for a variety of GI conditions, including ulcers and infections due to H. pylori, a bacteria that spreads through direct contact with saliva, vomit or stool — or through contaminated food or water. The bacteria has been shown to cause gastritis and/or peptic ulcers in certain people. With Voquezna, the Takeda drug was combined with either previously-approved antibiotics amoxicillin in a “Dual Pak” or as a “Triple Pak” with the addition of clarithromycin. — Paul Schloesser

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16. MOUNJARO

Active ingredient: tirzepatide

Indication: To improve blood sugar control in diabetes, in addition to diet and exercise

Designations: Priority Review

Snapshot:  Eli Lilly’s Mounjaro secured the OK from the FDA in a bid to treat adults with Type II diabetes. But the GIP/GLP-1 receptor agonist had another powerful impact as a weight-loss drug. That’s likely to set it up as a blockbuster competitor to Novo Nordisk’s Wegovy. Mounjaro has already been introduced as a crucial part of Lilly’s expansion plans, with a chronic weight management indication that has been given fast track designation. CEO David Ricks said on an earnings call that a rolling submission will be completed by mid-2023 as plans for increasing manufacturing capacity are underway. — Paul Schloesser

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17. VTAMA

Active ingredient: tapinarof

Indication: Plaque psoriasis

Designations: N/A

Snapshot: Tapinarof, branded as Vtama when the FDA approved it in May, was nabbed from GSK by Vivek Ramaswamy’s team back in 2018 for $200 million upfront and $330 million total. Clinical data over the following years from Phase III trials showed a minimum 2-grade improvement compared to placebo, and data in plaque psoriasis patients showed that 20% of those with moderate to severe disease achieved 90% disease clearance by 12 weeks. After FDA approval for Vtama as the first steroid-free topical medication in its class, Dermavant chief Todd Zavodnick laid out the marketing plan for Vtama, while noting the highly competitive space filled with blockbusters like Stelara, Skyrizi and Cosentyx. Zavodnick said that Vtama’s market share could be as a replacement for steroids in the topical space and leave the most severe cases to the biologics. The cream, in those cases, could also serve as an add-on therapy. — Paul Schloesser

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18. PRIORIX

Active ingredient: Measles, mumps and rubella vaccine live

Indication: For active immunization for the prevention of measles, mumps, and rubella in individuals 12 months of age and older

Designations: N/A

Snapshot: With the FDA approval of Priorix, GSK brought one of its widely-licensed vaccines to the US, giving pediatricians a new option for a common childhood shot for the first time in more than 50 years. GSK tested the measles, mumps and rubella vaccine across six clinical studies, with more than half of the 12,000-plus trial participants in the US, to demonstrate safety and tease out the adverse reactions — while efficacy was determined based on immunogenicity data versus a comparator vaccine. It can be used as either a two-shot series or as a second dose if children had already received another MMR vaccine, likely Merck’s M-M-R II. The CDC followed up with a recommendation for use of the vaccine. — Amber Tong 

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19. AMVUTTRA

Active ingredient: vutrisiran

Indication: Hereditary transthyretin-mediated amyloidosis (hATTR)

Designations: Orphan Drug

Snapshot: Alnylam notched a win for its second-generation hereditary transthyretin-mediated (hATTR) amyloidosis drug Amvuttra just four years after the approval of its first, Onpattro. Both are RNA interference (RNAi) meds that shut down the production of TTR proteins from the liver. While Amvuttra‘s approval is the same as Onpattro to treat polyneuropathy, or peripheral nerve disease, in adult patients, it takes a different route, via subcutaneous delivery once every three months, while Onpattro is an IV infusion every three weeks. The newer Amvuttra’s open-label Phase III study beat historical placebo controls — used for ethical reasons, Alnylam said — in significantly reducing polyneuropathy, and improving quality of life and gait speed after nine months of the trial. More than 50% of patients experienced halted or reversed symptoms. Alnylam has not yet turned a profit as a company, but Amvuttra’s success — especially if it gets a second indication approval for people with cardiac ATTR amyloidosis subtype currently in the HELIOS-B study — could change that. — Beth Snyder Bulik

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20. ZYNTEGLO

Active ingredient: betibeglogene autotemcel

Indication: Adult and pediatric patients with beta thalassemia who require regular red blood cell transfusions

Designations: Orphan Drug, Breakthrough Therapy

Snapshot: The thumbs-up from the FDA in August for Zynteglo followed a unanimous advisory committee vote in June, with outside experts pointing to extraordinary efficacy as 89% of subjects with TDT who received beti-cel achieved transfusion independence. Bluebird says there are about 1,500 patients with TDT in the US, and between 800 and 850 of them will be eligible to receive the gene therapy. — Zachary Brennan

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21. XENPOZYME

Active ingredient: olipudase alfa

Indication: Non-CNS manifestations of acid sphingomyelinase deficiency (ASMD)

Designations: Fast Track, Breakthrough Therapy, Orphan Drug, Priority Review

Snapshot: Sanofi’s infused Xenpozyme is the first drug approved to treat the rare genetic disease ASMD, which was previously known as Niemann-Pick disease types A, A/B and B. The disease affects the liver, lungs, spleen, blood and digestive system, and over time can cause organ damage and premature death. In the US, fewer than 120 people have been diagnosed with ASMD and two-thirds are children, according to Sanofi. The enzyme replacement therapy, administered every two weeks by IV, replaces patients’ deficient acid sphingomyelinase enzyme and had already been approved in Europe and Japan. The FDA approved Xenpozyme after the FDA delayed its application by three months, capping decades of research by Sanofi’s Genzyme unit, which first won orphan indication for the disease in 2001. — Beth Snyder Bulik

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22. SPEVIGO

Active ingredient: spesolimab-sbzo

Indication: Generalized pustular psoriasis (GPP) flares

Designations: Breakthrough Therapy, Orphan Drug

Snapshot: The approval of IL-36R inhibitor Spegivo marks a first in dermatology conditions for Boehringer Ingelheim. Spevigo treats generalized pustular psoriasis (GPP), a rare autoimmune condition that not only causes painful skin flares, but also can lead to serious complications, including heart failure, renal failure and sepsis. BI’s pivotal Phase II study showed that more than half (54%) of the patients on treatment who began with a high density of pustules on their bodies, no longer had any visible pustules after 12 weeks compared to only 6% of patients on placebo. The win for BI gives it the lead over competitor AnaptysBio, which is developing a similar IL-36R antibody (imsidolimab) for the same indication and is looking to out-license it. Meanwhile, BI’s overall program for spesolimab is facing downgraded expectations after an August shutdown of its Phase II study for Crohn’s disease patients who suffered from bowel obstructions. — Beth Snyder Bulik

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23. DAXXIFY

Active ingredient: daxibotulinumtoixnA-lanm

Indication: Moderate-to-severe glabellar lines associated with corrugator and/or procerus muscle activity

Designations: N/A

Snapshot: The Botox competitor Daxxify won approval in September after a Phase III clinical trial that included more than 2,700 participants and followed about 20 years of market dominance for Botox. Revance is looking to take a share of the $2.2 billion Botox market with its longer-lasting, potentially twice-yearly injection, whereas those using Botox may have to return for more injections every three to four months. The company Evolus also scored an FDA approval for a Botox rival in 2019, known as Jeuveau, but only mustered 2021 sales of almost $100 million, and another nearly $150 million in 2022. — Zachary Brennan

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24. SOTYKTU

Active ingredient: deucravacitinib

Indication: Moderate-to-severe plaque psoriasis

Designations: N/A

Snapshot: The new once-daily psoriasis treatment, a TYK2 inhibitor, outperformed Amgen’s twice-daily blockbuster Otezla in a head-to-head study. “With a clean label, deucravacitinib’s ability to hit out-year consensus forecasts ($3B+; our estimate is $3.4B in 2032) seems very achievable, if not too low,” said Wolfe’s Tim Anderson.“Sotyktu has the potential to become the new standard of care oral treatment for people with moderate-to-severe plaque psoriasis, given its profile in helping patients achieve clearer skin as demonstrated in the POETYK PSO clinical program,” said USC’s April Armstrong, clinical investigator in the trial. – Zachary Brennan

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25. ROLVEDON

Active ingredient: eflapegrastim-xnst

Indication: To decrease the incidence of infection in patients with non-myeloid malignancies receiving myelosuppressive anti-cancer drugs associated with clinically significant incidence of febrile neutropenia

Designations: N/A

Snapshot: After a CRL due to manufacturing question marks in 2021, the September approval for Rolvedon, which is in the class of drugs known as colony stimulating factors, was supported by data from two identically designed Phase III, randomized, open-label, noninferiority clinical trials, which evaluated the drug in 643 early-stage breast cancer patients for the management of neutropenia due to myelosuppressive chemotherapy. In both studies, the company said its treatment demonstrated non-inferiority in mean duration of severe neutropenia and a similar safety profile to Amgen’s Neulasta (pegfilgrastim). — Zachary Brennan

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26. SKYSONA

Active ingredient: elivaldogene autotemcel

Indication: To slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy (CALD)

Designations: Orphan Drug, Rare Pediatric Disease, Breakthrough Therapy

Snapshot: Skysona was one of two bluebird bio gene therapies to win approval within the span of a month this summer, with the other being Zynteglo. Both were priced higher than Novartis’ Zolgensma, which previously held the title of most expensive drug in the world. The disease Skysona is intended to treat — early, active cerebral adrenoleukodystrophy — is so rare that bluebird isn’t expected to surpass more than $100 million in peak sales, however. Still, the drug’s price likely marks the start of a new normal for rare disease gene therapies, some experts said. The high prices come with risk-sharing agreements and refunds if the products don’t work. Skysona also comes with a black box warning for hematologic malignancy, but an advisory committee in June resulted in a unanimous vote in favor of approval. — Max Gelman

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27. TERLIVAZ

Active ingredient: terlipressin

Indication: To improve kidney function in adults with hepatorenal syndrome with rapid reduction in kidney function

Designations: Priority Review, Fast Track, Orphan Drug

Snapshot: Despite being approved in countries outside the US for more than three decades, Terlivaz snagged its first FDA approval on Sept. 14. The injection, also known as terlipressin, is cleared to treat patients with hepatorenal syndrome, or HRS, with rapid reduction in liver function. The disease occurs in patients with advanced liver disease — most commonly those with advanced cirrhosis and ascites. If left untreated, HRS with rapid reduction in kidney function has a median survival of about two weeks. However, Mallinckrodt saw a bumpy road to approval. Terlipressin was first rejected in 2009, and US regulators rejected it a second time in 2020 despite an advisory committee narrowly voting in favor of approval. That time, the FDA asked for “more information to support a positive risk-benefit profile for terlipressin” as a treatment for HRS type 1. The company received another rejection back in February, but chalked it up to packaging and labeling facility struggles. The approved label warns of a risk of serious or fatal respiratory failure, and cautions patients with low oxygen in their blood against starting the medication. — Nicole DeFeudis

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28. ELUCIREM

Active ingredient: gadopiclenol

Indication: To detect and visualize lesions, together with MRI, with abnormal vascularity in the central nervous system and the body

Designations: Priority Review

Snapshot: Back in 2017, the FDA required a new warning for gadolinium-based contrast agents (GBCAs), which are used to help doctors see abnormalities in MRIs, after it was discovered that gadolinium was staying in patients’ bodies for months to years after receiving them. French pharma Guerbet won approval for Elucirem back in September, its macrocyclic gadolinium-based contrast agent that can be administered at half the conventional dose of other GBCAs. The approval was based on data from two Phase III studies that showed Elucirem was non-inferior to gadobutrol in MRI tests of the brain and body at half the dose. Despite adding a warning to GBCAs back in 2017, the FDA concluded that the benefit still outweighed “any potential risks.” — Nicole DeFeudis

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29. OMLONTI

Active ingredient: omidenepag isopropyl ophthalmic solution

Indication: To reduce elevated intraocular pressure in patients with open-angle glaucoma or ocular hypertension

Designations: N/A

Snapshot: After launching in a handful of countries, including Japan back in 2018, Omlonti snagged a US approval in September to help relieve fluid pressure in the eye in patients with a type of glaucoma or ocular hypertension, a condition when pressure in the eye is higher than normal. Glaucoma and ocular hypertension affect millions worldwide, and can lead to vision loss if untreated, according to Santen and UBE. Reducing intraocular pressure, also known as IOP, is crucial to avoiding damage to the optic nerve, the companies said in a news release. Omlonti’s approval was based on 12 clinical studies, including ones in which the drug proved non-inferior to timolol and latanoprost, two standards of care. While the approval came on the heels of Santen’s approval of its Verkazia eye drops to treat vernal keratoconjunctivitis (VKC), Omlonti marks the company’s first glaucoma nod in the US. — Nicole DeFeudis

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30. RELYVRIO

Active ingredient: sodium phenylbutyrate/taurursodiol

Indication: Amyotrophic lateral sclerosis (ALS)

Designations: Priority Review, Orphan Drug

Snapshot: Relyvrio is just the third drug approved to treat ALS, coming in after riluzole in 1995 and edaravone in 2017. After a modestly positive Phase II readout in 2020, ALS patients rallied around Relyvrio and lobbied loudly for its approval. The FDA originally asked Amylyx to run a Phase III study before initiating a formal review, but soon backtracked to accept just the one mid-stage trial — with the larger, pivotal study acting in an informal confirmatory capacity. Regulators also held two adcomms for the drug, an unusual move with the first resulting in a negative vote and the second in a positive vote. Amylyx, however, has no legal obligation to pull the drug should the Phase III fail when it reads out in 2024. The lack of a hard requirement, plus the drug’s lofty $158,000 per-year price tag, drew criticism from patients and experts when the approval finally came through in late September. — Max Gelman

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31. LYTGOBI

Active ingredient: futibatinib

Indication: Intrahepatic cholangiocarcinoma harboring fibroblast growth factor receptor 2 (FGFR2) gene fusions or other rearrangements

Designations: Breakthrough Therapy, Orphan Drug, Priority Review

Snapshot: Otsuka and Taiho are looking to differentiate this drug, a new kinase inhibitor to treat adults with a type of previously treated bile duct cancer, from other, previously approved FGFR inhibitors such as Janssen’s Balversa (erdafitinib) and Incyte’s Pemazyre (pemigatinib). Those compounds, Taiho claims, are reversible ATP-competitive inhibitors, whereas Lytgobi is an irreversible FGFR 1-4 inhibitor. The kind of cancer for which Lytgobi is approved to treat sees about 8,000 new cases diagnosed every year in the US. Lytgobi marked the third FDA OK for Taiho, after Lonsurf in 2015 for metastatic colorectal cancer and Inqovi in 2020 for intermediate and high-risk myelodysplastic syndromes. — Max Gelman

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32. IMJUDO

Active ingredient: tremelimumab

Indication: Unresectable hepatocellular carcinoma

Designations: Orphan Drug

Snapshot: It took AstraZeneca years of trying to find the right dose before it could get Imjudo approved. Then, it got two approvals in under a month. AstraZeneca plans to sell Imjudo largely in combination regimens with its anti-PD-1 drug Imfinzi, similar to Bristol Myers Squibb’s Opdivo/Yervoy. Combination therapies are a likely blockbuster path forward in cancer R&D, as the big PD-1 patents are well into their patent lifespans, particularly for Merck and Keytruda. Imjudo gives AstraZeneca some more flexibility with Imfinzi, and it won’t be the only drug used in combination with the PD-1. But given how it and anti-CTLA-4 therapies are already validated and market successes, expect the Big Pharma to continue pursuing a range of indications for this new combo. – Max Gelman

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33. TECVAYLI

Active ingredient: teclistamab

Indication: Relapsed or refractory multiple myeloma among adults who have received at least four specific lines of therapy

Designations: Breakthrough Therapy

Snapshot: J&J’s BCMA/CD3 bispecific teclistamab may not ever get into the megablockbuster category of multibillion-dollar drug franchises, but the top execs have routinely touted the drug as part of its long lineup of therapies that should have a notable future on the bottom line. The FDA played its part in handing off a breakthrough drug designation back in the summer of 2021, where 65% of a small group of patients saw a response for their hard-to-treat cases of multiple myeloma. And they followed up in October with an accelerated approval based on the score. Bispecifics have been coming on strong as the BCMA field quickly filled up with a variety of contenders, where J&J also has a leading player in the BCMA CAR-T cilta-cel, allied with Legend, coming in close behind Abecma from Bristol Myers. High levels of neurotoxicity, though, required a REMS to monitor for adverse events.  — John Carroll

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34. ELAHERE

Active ingredient: mirvetuximab soravtansine

Indication: For adult patients with folate receptor alpha (FRα)-positive, platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer

Designations: Orphan Drug, Fast Track, Priority Review

Snapshot: ImmunoGen’s antibody-drug conjugate mirvetuximab soravtansine — now marketed as Elahere — has experienced more melodrama than an afternoon soap opera. Its years-long travails included a setback on one failed attempt at an accelerated approval in 2019, followed by a savage restructuring and slow comeback that peaked with the accelerated OK for ovarian cancer on Nov. 15. It hit the market as the first new ovarian cancer drug in seven years, but a record of high toxicity and the use of single-arm data on the FDA approval leaves some analysts wondering if there may be more drama ahead for this franchise when the confirmatory study wraps. The therapy targets folate receptor alpha, which is expressed at high levels in three or four out of every 10 ovarian cancer patients. — John Carroll

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35. TZIELD

Active ingredient: teplizumab-mzwv

Indication: To delay the onset of stage 3 Type I diabetes

Designations: N/A

Snapshot: Provention Bio rounded out the year with the first FDA-approved drug to delay the onset of Type I diabetes, along with a partnership and $35 million investment from Sanofi to market the drug in the US. The approval of the drug means that children, teenagers and young adults have more time before needing to start insulin treatments. The company said that it already has screening programs up and running to catch potential patients and lessen the challenge of establishing a market where none used to exist. Provention’s first goal is to target around 30,000 people in the US who have direct relatives with Type I diabetes. Tzield had its share of setbacks getting to market, first because Provention couldn’t show its commercial product was comparable to what was previously tested in clinical trials. Then more than a year before its eventual approval, Provention’s first application got pushback from the FDA in the form of a complete response letter, citing pharmacokinetic issues.  — Katherine Lewin

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36. HEMGENIX

Active ingredient: etranacogene dezaparvovec-drlb

Indication: Adults with hemophilia B (congenital factor IX deficiency) who: currently use factor IX prophylaxis therapy, or have a current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes

Designations: Orphan Drug, Breakthrough Therapy

Snapshot: CSL got its gene therapy approved by the FDA for hemophilia B with a price tag of $3.5 million, the highest of any single-use gene therapy approved in the US. The Australian company noted that the price of the therapy outweighs the cost of prophylactic infusions of factor IX, which can total anywhere from $200,000 to $750,000 a year. One thing to continue watching for on Hemgenix, however, will be durability. The FDA’s approval was based on a trial that showed 51 out of 54 patients with hemophilia B were able to stop prophylactic treatment for at least 18 months. Data presented at the American Society of Hematology meeting showed that the same 51 patients stayed off prophylaxis for an additional six months. — Paul Schloesser

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37. REBYOTA

Active ingredient: fecal microbiota, live-jslm

Indication: Prevention of recurrence of Clostridioides difficile infection (CDI) in individuals 18 years of age and older, following antibiotic treatment for recurrent CDI

Designations: Fast Track, Orphan Drug, Breakthrough Therapy

Snapshot: The FDA approved a poop-based drug implant to prevent the recurrence of Clostridioides difficile infection. While fecal microbiota transplantation — essentially replenishing a patient’s gut with bacteria from healthy feces — has been taking place without something FDA-approved, Ferring’s Rebyota marks the first live biotherapeutic drug approved by the federal agency to fight the infection. Ferring first got its hands on the drug when it bought the startup Rebiotix in 2018. Clinical trials helped cement the approval after data showed the overall estimated rate of success in preventing recurrent C. difficile infections through eight weeks was significantly higher in the group on Rebyota (70.6%) than in the placebo group (57.5%). — Paul Schloesser

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38. REZLIDHIA

Active ingredient: olutasidenib

Indication: Adults with relapsed or refractory acute myeloid leukemia with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation

Designations: N/A

Snapshot: Rigel bought the drug from Forma for a mere $2 million for full rights with the promise of another $17.5 million once it met certain milestones. Forma had long regarded the IDH1 inhibitor as a non-core program, but it became a direct competitor of Servier, which bought the first FDA-approved drug in the IDH1 class. According to the label, relapsed or refractory acute myeloid leukemia patients are recommended to take 150 mg orally twice daily at about the same time each day until the progression of the disease stops. — Katherine Lewin

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39. KRAZATI

Active ingredient: adagrasib

Indication: KRAS G12C-mutated locally advanced or metastatic non-small cell lung cancer in adults who have received at least one prior systemic therapy

Designations: Breakthrough Therapy, Orphan Drug, Fast Track

Snapshot: Once considered a tight competitor in the initial race to come out with the first FDA-approved drug to target the once-thought undruggable biomarker KRAS in cancer patients, Mirati was beaten by Amgen last year when the Big Pharma moved Lumakras past the regulatory finish line. Now more than 18 months later, Mirati has its first approved drug after trial data showed a 43% ORR and a median response duration of 8.5 months in 112 patients. Now approved to treat patients with KRAS-mutated NSCLC — present in 1 out of every 8 NSCLC patients — Krazati is priced at a slight premium to Lumakras, which costs just under $215,000 a year. — Paul Schloesser

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40. ADSTILADRIN

Active ingredient: nadofaragene firadenovec-vncg

Indication: Adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle-invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors

Designations: Priority Review, Breakthrough Therapy, Fast Track

Snapshot: In the aftermath of a May 2021 rejection thanks to chemistry, manufacturing and control issues, the FDA greenlit Ferring Pharmaceuticals’ AAV-based gene therapy to treat a very niche type of bladder cancer. The regulatory agency gave the thumbs-up after a single-arm trial showed a 51% complete response rate and a median response duration of 9.7 months in 157 patients. It’s still early in Ferring’s commercial aspirations for the therapy, now known as Adstiladrin. The company plans to make the treatment available in the second half of the year, and final price numbers are still undisclosed. — Paul Schloesser

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41. XENOVIEW

Active ingredient: hyperpolarized Xe-129

Indication: To evaluate pulmonary function and imaging

Designations: N/A

Snapshot: US-based medtech company Polarean Imaging snagged its first approval for a drug/device combination in lung imaging — in the latest update in a 26-month saga. The company’s product, now branded as Xenoview, is a hyperpolarized form of xenon gas to be used in MRI scans as a contrast agent, approved alongside two pieces of hardware/software. The biotech originally submitted an NDA to the FDA, along with a request for priority review, in October 2020. A year later, the federal regulator returned with a CRL, which Polarean said at the time was mostly around technical and manufacturing issues. Fast forward six months to March 2022, when the biotech resubmitted an NDA that was accepted by the FDA, setting a Sept. 30 PDUFA date. Come Sept. 22, FDA told the biotech that it needed more information from Polarean’s drug manufacturing partner — which would constitute a major amendment — extending the PDUFA date another 90 days to Dec. 30. The agency handed down the approval eight days before the deadline. — Paul Schloesser

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42. SUNLENCA

Active ingredient: lenacapavir

Indication: Adult patients living with human immunodeficiency virus type 1 (HIV-1), whose HIV infections cannot be successfully treated with other available treatments due to resistance, intolerance, or safety considerations

Designations: Breakthrough Therapy

Snapshot: Gilead won an approval in the US for lenacapavir for HIV in December after facing delays caused by concerns over its glass vials. The FDA placed a hold on 10 of Gilead’s trials late last year after finding that vials containing the drug could cause an increased risk of contamination from glass particulates. When regulators handed the drug a rejection in March, Gilead cited issues related to its use of borosilicate glass. The company switched to aluminosilicate glass, leading regulators to lift their hold in May. The drug — marketed in the US as Sunlenca — has since been approved in Europe, the UK and Canada. The twice-yearly injection is approved for heavily treatment-experienced patients, offering an easier alternative to daily pill regimens that many patients are used to, Jared Baeten, VP for HIV clinical development, told Endpoints News. — Nicole DeFeudis

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43. LUNSUMIO

Active ingredient: mosunetuzumab

Indication: Third-line treatment for adults with relapsed or refractory follicular lymphoma

Designations: Priority Review, Breakthrough Therapy

Snapshot: Many a biotech has lined up in the bispecific race for cancer treatments, and Roche’s Genentech became the first to win the FDA’s blessing for an antibody that targets CD20 on B cells and CD3 on T cells. Right before the Christmas holiday, Genentech said the FDA greenlit Lunsumio, which won approval in the European Union earlier in 2022 under the same brand name, for patients with follicular lymphoma. That gives Genentech a head start on the CD20xCD3 space, which includes AbbVie-allied Genmab, Regeneron and Johnson & Johnson-partnered Xencor. The US regulator attached a so-called boxed warning onto Genentech’s accelerated nod to warn patients on potential cytokine release syndrome, a side effect with certain cancer meds that can lead to an overactive immune response. The Swiss Big Pharma is testing a subcutaneous version as well, which would shorten treatment delivery from hours to minutes and potentially reduce those CRS events. The drug developer also submitted glofitamab, its bispecific for more aggressive lymphomas, for FDA approval in recent weeks. — Kyle LaHucik

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44. BRIUMVI

Active ingredient: ublituximab-xiiy

Indication: Relapsing forms of multiple sclerosis

Designations: N/A

Snapshot: TG Therapeutics received its second-ever FDA approval — now opening up the company to go against Big Pharma giants such as Roche and Novartis in multiple sclerosis. The biotech’s monoclonal antibody to be marketed as Briumvi was once before the FDA as part of a proposed two-drug combination called U2 for blood cancer. However, a Phase III readout on the other part of U2, already approved Ukoniq and TG’s first approval, led the biotech to voluntarily pull the request for ublituximab and Ukoniq in H1 of 2022. While priced at $59,000 a year, the biotech announced that it is working on getting it approved outside the US, predicting six to nine months for an EU approval. — Paul Schloesser

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45. NEXOBRID

Active ingredient: anacaulase-bcdb

Indication: Removal of eschar in adults with deep partial-thickness and/or full-thickness thermal burns

Designations: N/A

Snapshot: The last approval of 2022 comes from a BLA resubmission for NexoBrid, a topical drug for severe burns that has been approved outside the US for close to a decade. The drug removes eschar, dead tissue that falls off healthy skin and is caused by burns or cauterization, without harming healthy tissues. It was developed by Israeli biopharma MediWound as its flagship product and was licensed to Vericel for commercialization in the US. A Phase III study met the primary endpoint of complete eschar removal along with all secondary endpoints, including shorter time to eschar removal and a lower incidence of surgical eschar removal compared to standard of care. The resubmission was assisted in part by BARDA, which paid MediWound $9 million earlier in 2022 to support the filing. The drug is currently anticipated to be available in the US in Q2. — Paul Schloesser

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