Latest FDA Decisions Expected in September 2025: Five Innovative Therapies Under Review

In 2024, the U.S. FDA approved more than 60 innovative therapies and vaccines, highlighting a record year for medical progress.

Looking ahead, based on the Prescription Drug User Fee Act (PDUFA) timelines, the FDA is expected to issue regulatory decisions on five groundbreaking therapies this September.

Here is an overview of the pipeline at Dengyue:

🔹 Apitegromab – Scholar Rock

• Indication: Spinal Muscular Atrophy (SMA)
• Mechanism: A first-in-class monoclonal antibody that selectively binds the precursor and latent forms of myostatin in skeletal muscle, inhibiting its activation.
• Highlights:
  • First muscle-targeted therapy for SMA to show positive results in a Phase 3 pivotal trial (SAPPHIRE).
  • FDA granted Fast Track, Orphan Drug, and Rare Pediatric Disease designations; EMA awarded PRIME and Orphan Drug status.
  • If approved, apitegromab would become the first muscle-directed therapy for SMA.

🔹 Paltusotine – Crinetics Pharmaceuticals

• Indication: Acromegaly
• Mechanism: An oral, once-daily, non-peptide SST2 receptor agonist.
• Highlights:
  • NDA accepted in Dec 2024; designed for treatment and maintenance of acromegaly.
  • Phase 3 PATHFNDR-2 results: 56% of patients achieved normalized IGF-1 levels at week 24 vs. 5% on placebo (p<0.0001).
  • Well tolerated with no serious adverse events reported.

🔹 Tolebrutinib – Sanofi

• Indication: Non-relapsing Secondary Progressive Multiple Sclerosis (nrSPMS)
• Mechanism: A brain-penetrant oral BTK inhibitor targeting chronic neuroinflammation, a key driver of MS progression.
• Highlights:
  • NDA accepted with Priority Review; decision expected by September 28, 2025.
  • Phase 3 HERCULES study: delayed 6-month confirmed disability progression by 31% vs. placebo.
  • Also supported by GEMINI 1 & 2 studies in relapsing MS.

🔹 Narsoplimab – Omeros

• Indication: Transplant-Associated Thrombotic Microangiopathy (TA-TMA)
• Mechanism: A fully human monoclonal antibody targeting MASP-2 in the lectin pathway of complement activation.
• Highlights:
  • Granted Breakthrough Therapy and Orphan Drug designations.
  • Resubmitted BLA under FDA review with decision expected by late September 2025.
  • Clinical data showed significantly improved overall survival in TA-TMA patients compared to external controls.

🔹 Ataluren – PTC Therapeutics

• Indication: Nonsense mutation Duchenne Muscular Dystrophy (nmDMD)
• Mechanism: A readthrough therapy enabling ribosomes to bypass premature stop codons and produce functional full-length proteins.
• Highlights:
  • NDA resubmission accepted in October 2024.
  • Phase 3 Study 041: significant improvements in 6MWD, NSAA, and time-to-climb tests.
  • Long-term STRIDE study: delayed loss of ambulation by 3.5 years and preserved lung function by nearly 2 years.

📌 Outlook:
If approved, these therapies would mark major breakthroughs in neuromuscular disorders, endocrine diseases, multiple sclerosis, rare transplant complications, and genetic muscular dystrophies—expanding treatment options and reshaping standards of care.

More FDA news at HK Dengyue.