The agency is set to decide by September on new therapies for Duchenne cardiomyopathy, multiple myeloma and spinal muscular atrophy.
Amid changing leadership and priorities at the Food and Drug Administration, drug company executives and investors have stayed focused on one important metric: whether the agency reviews and approves new medicines on time.
So far, despite widespread layoffs and disruptions in resources, the FDA has mostly delivered. Through mid-June, a few decisions had run over time, but it wasn’t clear whether the cuts were directly to blame, especially as those particular reviews already had question marks.
On June 13, however, Kalvista Pharmaceuticals said that, because of a “heavy workload and limited resources” the FDA wasn’t able to complete its review of the company’s rare disease drug on schedule. Analysts had expected an easy approval, and were surprised by the missed deadline. According to a later report by Endpoints News, agency head Martin Makary had intervened and sought to issue a rejection, but was convinced to hold back due to legal concerns.
Moving forward, investors and analysts will be paying even closer attention to approval deadlines and the FDA’s review process. The next three months will bring several important decisions, as well as at least one controversial case. BioPharma Dive has added five new drugs to watch to the running list below, which we update at the start of each quarter.
Capricor’s dermiocel
For Duchenne cardiomyopathy
Cardiomyopathy, a type of progressive weakening of the heart muscle, is the leading cause of death in people with Duchenne muscular dystrophy. The symptom isn’t specifically addressed by available therapies, either.
Capricor Therapeutics aims to change that with a cell therapy the FDA could approve by Aug. 31. Yet leadership turmoil at the regulator appears to hang over what already wasn’t a clear-cut case.
Capricor’s therapy, deramiocel, is derived from a special type of heart cell known to help preserve function. The company evaluated it in a Phase 2 trial, which showed treatment improved measures of upper limb function and the heart’s ability to pump blood. However, the results came from only 20 participants – eight of whom received therapy.
To add further support to its case, Capricor has also marshalled data from a historical study of heart function in Duchenne patients.
The FDA accepted Capricor’s approval application in March. Since then, though, multiple top officials have been forced out of the FDA office that regulates gene therapies. Vinay Prasad – who has long been critical of Sarepta Therapeutics’ Elevidys, another Duchenne medicine with imperfect data – is now that office’s leader.
Initially, the agency scheduled an advisory committee meeting to help it review Capricor’s application. But it later canceled its request — a step Stat News reported last month was taken because Prasad was “skeptical of the treatment.” In a June note to clients, Leerink Partners’ Mani Foroohar added how there’s been talk that Prasad and Nicole Verdun, a high-ranking gene therapy regulator placed on leave by the FDA, “disagreed over the regulation” of deramiocel.
In a June 24 statement, Capricor CEO Linda Marban said “all regulatory milestones have proceeded as expected” without major issues. An in-person meeting with FDA staff is scheduled for late July, roughly a month before a final verdict is expected. – Ben Fidler
Scholar Rock’s apitegromab
For spinal muscular atrophy
The treatment landscape for spinal muscular atrophy, a rare disease causing progressive muscular weakness, has shifted dramatically over the last decade. A drug from Scholar Rock called apitegromab could add to that progress if the FDA approves it by a Sept. 22 deadline.
Since 2016, U.S. regulators have approved a gene-targeting medicine from Biogen, a gene therapy from Novartis and an oral treatment from Roche. All three can change SMA’s course and help babies survive and hit growth milestones they otherwise wouldn’t. All three therapies have “mixed” effects on motor function, however, resulting in eventual decline that a muscle-preserving therapy might help to address, Piper Sandler analysts wrote in a report last year.
Scholar Rock believes it has that therapy in apitegromab, which blocks a protein called myostatin that can limit muscle growth. Phase 3 testing showed apitegromab together with standard therapy significantly improved motor function after one year versus typical care and a placebo. The result vaulted Scholar Rock’s market value into the billions of dollars and made it an acquisition target.
Since then, Scholar Rock has been gearing up to launch apitegromab. The company switched up its management team, naming industry veteran David Hallal its CEO and ex-Alnylam Pharmaceuticals president Akshay Vaishnaw its R&D chief. It’s built up a commercial team. On a conference call in June, Hallal noted how its leaders want to “build and scale” the company “into the next great global biotech powerhouse.”
Scholar Rock is betting so heavily on its path that it’s sticking to rare diseases despite encouraging study results in obesity, one of the most lucrative markets in drug research. – Ben Fidler
GSK’s Blenrep
For multiple myeloma
GSK made history when U.S. regulators approved Blenrep in 2020. The drug was the first marketed multiple myeloma medicine to target BCMA, a protein that’s universally found on malignant cells.
Several, more effective BCMA-targeting therapies quickly followed Blenrep to market, however, and GSK’s drug, which is associated with eye toxicity, struggled. The company withdrew it from market in 2022 after it failed a confirmatory trial.
GSK has since found a way to revive Blenrep. The company continued testing Blenrep in earlier lines of care and alongside other standard myeloma drugs, rather than as monotherapy. In a pair of studies, it outperformed two widely used medicines, Darzalex and Velcade, on measures of disease progression. The findings prompted GSK to begin seeking new approvals.
Blenrep has already been cleared for a relaunch in the U.K. A return to the U.S. markets could follow if the FDA approves it by a July 23 deadline. While Blenrep will face even stronger competition now than before, doctors are also better able to manage Blenrep’s side effects through dose reductions and eye drops, which could help expand use. – Jonathan Gardner
Sanofi’s tolebrutinib
For non-relapsing secondary progressive multiple sclerosis
Sanofi spent nearly $4 billion on Prinicipia Biopharma five years ago in large part because of a drug now known as tolebrutinib. That investment could pay dividends on Sept. 28, when the FDA is scheduled to decide whether to clear it for a form of multiple sclerosis.
Tolebrutinib is one of a newer class of oral medications called BTK inhibitors designed to treat autoimmune diseases rather than cancer. Study results to date haven’t met initial expectations, however, and development has been slowed by safety concerns. Testing was previously halted due to signs of liver toxicity and Sanofi abandoned testing in myasthenia gravis.
Tolebrutinib, as well as another, similar drug from Merck KGaA, also failed multiple trials in people with the common, “relapsing” form of multiple sclerosis.
However, Sanofi did report success in a less prevalent form of the disease known as non-relapsing secondary progressive MS, or nrSPMS. People with that form of the condition accumulate disabilities over time instead of experiencing periodic disease flares. Notably, they don’t have any approved treatments available to them.
Success in nrSPMS could allow Sanofi to make something of tolebrutinib, even if the drug’s sales potential is lower than the company once envisioned.
In a research report in May, Leerink Partners analyst Faisal Khurshid noted how the liver toxicity seen in testing of tolebrutinib is likely to yield a strict safety warning from the FDA. Despite that potential restriction, consensus estimates have tolebrutinib generating nearly $1.6 billion in sales at its peak, assuming approval. – Ben Fidler
Ionis’ donidalorsen
For hereditary angioedema
Ionis Therapeutics started 2025 on a new footing. The biotech, which for years has regularly out-licensed the drug candidates it developed, had set a course to become a commercial-stage company of its own. The FDA’s Dec. 19 approval of its rare disease drug Tryngolza kick-started the company’s plans.
Ionis could add a second, wholly-owned drug to its sales shelf if the FDA grants approval to donidalorsen by a decision deadline of Aug. 21.
Marketing a drug is a challenge in its own right. But should Ionis succeed with donidalorsen, the company will enter a competitive market that’s set for further disruption as new kinds of therapies advance.
The drug is designed to prevent the recurrent swelling attacks experienced by people with hereditary angioedema. Multiple preventive options are already available, as are on-demand treatments. In June, the FDA approved a new drug, CSL’s Andembry, and continues to review another from Kalvista Pharmaceuticals, although the agency notably missed its decision deadline.
Further out, Intellia Therapeutics is advancing a gene editing therapy it sees as a potential one-time option that could allow patients to do without regular preventive treatment.
Ionis will have to carve out a place in the market for donidalorsen if it secures an OK as expected. Polling the company commissioned suggests it could make a strong case to patients: Two-third of patients contacted by the Harris Poll said they felt they hadn’t yet found the best preventive option for them. – Ned Pagliarulo
Original news source: https://www.biopharmadive.com/news/fda-approval-decisions-watch-2025/736277/
July 2, 2025