FDA Action Alert: Key Decisions Loom for Merck, Sanofi, Biogen and More

The U.S. Food and Drug Administration (FDA) is set to make a series of critical decisions in the latter half of September, potentially shaping the future of several pharmaceutical companies and their innovative therapies. From subcutaneous formulations of blockbuster drugs to novel treatments for rare genetic disorders, the agency's rulings could significantly impact patient care across multiple therapeutic areas.

Merck's Subcutaneous Keytruda Formulation Awaits Verdict

On September 23, the FDA is expected to decide on Merck's application for a subcutaneous version of its flagship cancer immunotherapy, Keytruda (pembrolizumab). This new formulation, if approved, would be applicable across Keytruda's more than 40 existing indications. The subcutaneous version combines the PD-1 inhibitor with Alteogen's berahyaluronidase alfa to enable under-the-skin delivery.

Merck's proposal is supported by Phase III data from November 2024, demonstrating that the subcutaneous formulation matches the efficacy of the intravenous version as a first-line treatment for metastatic non-small cell lung cancer. Approval of this formulation could help Merck maintain Keytruda's market dominance beyond its expected loss of exclusivity in 2028.

Sanofi and Biogen Target Neurological Disorders

Sanofi is anticipating an FDA decision by September 28 for tolebrutinib, a brain-penetrant BTK inhibitor proposed for non-relapsing secondary progressive multiple sclerosis. The application is based on data from the Phase III HERCULES study, which showed a 31% delay in confirmed disability progression compared to placebo. If approved, tolebrutinib would be the first brain-penetrant BTK inhibitor for this form of multiple sclerosis.

Meanwhile, Biogen is seeking approval for a higher-dose formulation of its spinal muscular atrophy (SMA) therapy, Spinraza (nusinersen). The FDA's decision, expected by September 22, will be based on data from the Phase II/III DEVOTE study. The proposed regimen includes a 28-mg maintenance dose, with a loading schedule of two 50-mg intrathecal injections given 14 days apart.

Rare Disease Treatments Under Review

Crinetics Pharmaceuticals awaits an FDA decision by September 25 for paltusotine, an oral SST2 agonist for treating acromegaly in adults. The application is supported by positive results from the Phase III PATHFNDR-1 and PATHFNDR-2 studies, which demonstrated significant reductions in key disease markers.

Fortress Biotech and Sentynl Therapeutics are anticipating an FDA verdict by September 30 for CUTX-101, a subcutaneous formulation of copper histidinate for the treatment of Menkes disease. Phase III data showed that CUTX-101 decreased the risk of death by nearly 80% compared to historical controls, with treated patients surviving a median of 177.1 months versus 16.1 months in untreated comparators.

These upcoming FDA decisions represent potential breakthroughs in various therapeutic areas, from oncology to rare genetic disorders, and could significantly impact both patient care and the competitive landscape of the pharmaceutical industry.