Objective:

To outline upcoming FDA regulatory decisions for various therapies and products in September 2026.

Approach:

• Oncology: TLX101-Px for glioma and zidesamtinib for ROS1-positive non-small cell lung cancer.
• Neurology: UX111 for Sanfilippo syndrome type A, zilganersen for Alexander disease, and apitegromab for spinal muscular atrophy.
• Rheumatology: Zilurgisertib for fibrodysplasia ossificans progressiva.
• Dermatology: Aminolevulinic acid for superficial basal cell carcinoma.
• Endocrinology: Tiratricol for monocarboxylate transporter 8 deficiency.
• Cardiology: Mavacamten for symptomatic obstructive hypertrophic cardiomyopathy.

Key Findings:

• TLX101-Px received Orphan Drug and Fast Track designations.
• Zidesamtinib has Breakthrough Therapy designation.
• UX111's resubmission includes updated long-term clinical data.
• Zilganersen demonstrated stabilization in gait speed in clinical trials.
• Zilurgisertib reduced new heterotopic ossification lesions in patients.
• Aminolevulinic acid's indication may expand to superficial basal cell carcinoma.
• Tiratricol's application is based on multiple clinical trials.
• Mavacamten met its primary endpoint in the SCOUT-HCM trial.

Interpretation:

The FDA is reviewing several significant applications across various therapeutic areas, with many products receiving designations that may expedite their approval process.

Limitations:

• The outcomes of the FDA reviews are uncertain until official decisions are made.
• The article does not provide detailed clinical trial results for all therapies.

Conclusion:

The upcoming PDUFA dates highlight a range of innovative therapies under review by the FDA.

Sources:

• Telix Pharmaceuticals
• Nuvalent, Inc.
• Ultragenyx Pharmaceutical Inc.
• Ionis Pharmaceuticals
• Incyte
• Biofrontera
• Egetis Therapeutics
• Bristol Myers Squibb
• Scholar Rock