FDA Action Alert: Key Decisions Expected in March for GSK, Alnylam, Sanofi, and Others

The U.S. Food and Drug Administration (FDA) is gearing up for a busy March, with several important regulatory decisions expected across a wide range of therapeutic areas. From novel antibiotics to RNA-based treatments for rare diseases, the coming weeks could see significant advancements in the pharmaceutical landscape.

RNA Therapies for Rare Diseases Take Center Stage

Two RNA interference (RNAi) therapies are among the most anticipated FDA decisions this month. Alnylam Pharmaceuticals is seeking to expand the label of its RNAi therapy vutrisiran (Amvuttra) to include transthyretin amyloidosis (ATTR) with cardiomyopathy, with a decision due on March 23. Vutrisiran works by targeting and knocking down both wildtype and mutant forms of transthyretin RNA, potentially addressing the underlying pathology of ATTR and its cardiomyopathy manifestations.

In a separate application, Sanofi and Alnylam are awaiting the FDA's verdict on fitusiran, an siRNA therapy proposed for the treatment of hemophilia A and B. The decision is expected by March 28. Fitusiran, administered via subcutaneous injection, aims to lower the expression of antithrombin, a protein that inhibits clotting.

Novel Antibiotic and Cardiovascular Treatments Under Review

GSK's gepotidacin, a first-in-class triazaacenaphthylene antibiotic, is under FDA review for the treatment of uncomplicated urinary tract infections in adult women and adolescents. The agency's decision is due by March 26. Gepotidacin's novel mechanism of action has shown promise against uropathogens, including strains resistant to current treatments.

In the cardiovascular space, Milestone Pharmaceuticals' Cardamyst, an etripamil nasal spray for paroxysmal supraventricular tachycardia (PSVT), is expecting an FDA decision by March 27. The company claims to have submitted "the largest data package ever studied in PSVT," demonstrating that Cardamyst was "twice as effective and three times as fast as placebo" in normalizing heart rhythm.

Additional Notable Decisions on the Horizon

Other significant FDA decisions expected this month include:

• Scienture's liquid formulation of losartan (SCN-102) for hypertension, with a target action date of March 17.
• Elevar Therapeutics' rivoceranib in combination with camrelizumab for first-line treatment of unresectable or metastatic hepatocellular carcinoma, due by March 20.
• Theratechnologies' F8 formulation of tesamorelin for reducing stomach fat in people with HIV and lipodystrophy, with a decision expected on March 25.
• Soleno Therapeutics' diazoxide choline for Prader-Willi syndrome, scheduled for March 27.

As the pharmaceutical industry eagerly awaits these decisions, the outcomes could significantly impact treatment options for patients across multiple disease areas and potentially reshape competitive landscapes within the sector.