BMS, Novartis, Akebia and more face FDA approval decisions soon, with big sales on the line

An interesting and varied group of drugs—ranging from two first-in-class cancer therapies to a treatment that removes frown lines—face target dates for potential FDA approvals this month. Many of the decisions bear watching as their status could have far-reaching implications for the industry and for patients.

Take for example Akebia’s vadadustat, which could be the first in its class of HIF-PH oral inhibitors to reach the market in the United States. The drug, which treats the anemia that comes with chronic kidney disease, is due for a March 29 decision and is pegged by analysts with Evaluate to generate sales of $532 million in 2026.  

Vadadustat’s day of reckoning comes on the heels of a high-profile FDA rejection of AstraZeneca and FibroGen’s HIF-PH roxadustat for safety concerns. While trials of vadadustat have not shown the same safety issues for kidney patients on dialysis, they have cropped up for non-dialysis patients.

Anxiously awaiting the decision is GlaxoSmithKline, which is getting ready to file applications for approval in the U.S. and Europe for its HIF-PH drug daprodustat, which has succeeded in both dialysis and non-dialysis patients. Meanwhile, AZ and FibroGen are considering another clinical trial for roxa, which is already on the market in China and Europe.

Another company hoping to push through a first-in-class nod is Bristol Myers Squibb with relatlimab, an anti-LAG-3 drug that the company hopes to apply to multiple immune-oncology indications. This effort is in first-line metastatic melanoma, in combination with Opdivo.

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The combo staved off cancer progression by a median of 10.1 months, as opposed to Opdivo alone, which worked for a median of 4.6 months. Evaluate projects relatlimab’s 2026 sales to reach $437 million.

Interested parties in the decision, which is due on March 19, include Roche, which gained breakthrough designation for its anti-LAG-3 med tiragolumab last year, and Merck which is investigating its anti-LAG-3 drug favezelimab in combination with Keytruda against colorectal cancer.

Two cancer candidates from China

To foster more competition, with the hopes of bring down drug prices, the FDA has encouraged Chinese biopharmaceutical companies to seek approval for their treatments in the U.S. Two oncology drugs from China are scheduled for decisions soon, one this month and the other sometime in the first half of this year.

The chances are slim for Innovent’s Tyvyt to gain an FDA nod. Last month an advisory committee voted 14-1 to recommend rejection of the non-small cell lung cancer therapy, largely because its phase 3 trial took place exclusively in China. In the past, the U.S. regulator has signed off on drugs that were tested in non-diverse populations but in those cases, there was often an unmet need that the drug could address. With NSCLC however, there are a variety of treatments on the market.

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There were other problems with the filing as well, one being that the drug's trial didn’t use overall survival as its primary endpoint. Additionally, Innovent had virtually no communication with the FDA during the trial, the regulator pointed out. The company’s marketing partner Eli Lilly made a last-ditch effort to win favor by offering a steep discount compared with existing meds, but that did little to sway the panel.

A range of drug candidates from pharma companies large and small face March FDA decisions. (Getty Images)


After that sound drubbing, it will be interesting to see how the FDA approaches its decision on Akeso and Sino Biopharmaceutical’s penpulimab. The third-line candidate for metastatic nasopharyngeal carcinoma was the first Chinese drug to be reviewed under the FDA’s Real-Time Oncology Review (RTOR) procedure.

Most of the more than 20 drugs that have gone through this program have been approved in less than six months. But it has taken longer for penpulimab, which received the designation last May.

Two prospects with major sales potential

In 2018, Novartis forked over $2.1 billion to acquire Endocyte and its prostate cancer therapy 177Lu-PSMA-617. The price tag was seen by many analysts as exorbitant but recent clinical results suggest the drug could eventually pay off.

Evaluate projects the drug's sales could reach $851 million by 2026, but that’s just for the late-stage application under review and due for decision sometime in the first half of this year. The radioligand therapy targets the particularly stubborn metastatic castration-resistant prostate cancer. Novartis also is investigating 177Lu-PSMA-617 as an early-stage treatment.

A Cardinal Health survey of providers showed that the drug is likely to be in demand upon approval but there are logistical challenges as screening for the treatment requires a rare scanning method. In addition, the drug must be administered in a nuclear-licensed facility, requiring the patient to travel off-site.

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Meanwhile, due for a March 25 decision but unlikely to gain approval is TG Therapeutics’ combination of Ukoniq and ublituximab, dubbed U2. The treatment, for chronic lymphocytic leukemia, was put on clinical hold in January for concerns that it increased the risk of death.

Ukoniq was previously approved for relapsed or refractory marginal zone lymphoma a year ago. But a CLL nod would catapult it into blockbuster territory, according to Cantor Fitzgerald analyst Alethia Young who projects its peak sales will reach $1.6 billion.

Other drugs due for a March decision

While a phase 3 study of an intravenous version of Marinus’ ganaxolone was recently halted because of a supply disruption in clinical trial material caused by the pandemic, the oral version of the drug still has an FDA target date of March 20. There are better drugs on the market for the epilepsy condition it treats, but some patients who don’t respond to GW’s Epidolex or Novartis’ Afinitor have had success with ganaxolone. The med is expected to generate sales of $384 million in 2026.

Evive Biotech’s Ryzneuta, which treats a common side effect of chemotherapy, neutropenia, is up for a potential approval on March 30. The drug, which ensures infection fighting white blood cell counts remain high, allows patients to continue chemo treatments without interruption.

Hugel America and Croma Pharma GmbH’s Botulax for the treatment of frown lines is up for a decision on March 31. Already approved in 28 countries, including South Korea where it is the market leader, Botulax’s sales are pegged to reach $249 million in 2026.

One more drug has a pending decision date. Sol-Gel Technologies and Galderma’s Epsolay for the skin condition rosacea was due for a decision last year but inspection delays forced a postponement. The inspection was set to be conducted last month, so a decision could happen quickly.