FDA monthly preview: Key September decisions to watch
Several FDA decisions across multiple specialties are expected this month.
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By
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Kathryn Wighton
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August 28, 2026
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Clinical Scorecard: FDA monthly preview: Key September decisions to watch
At a Glance
| Category | Detail |
|---|
| Condition | Various conditions including glioma, ROS1-positive non–small cell lung cancer, and others |
| Key Mechanisms | Investigational therapies targeting specific genetic and disease mechanisms |
| Target Population | Adult and pediatric patients with specified conditions |
| Care Setting | Regulatory review and approval process |
Key Highlights
- TLX101-Px for glioma aims for PDUFA date on September 11, 2026.
- Zidesamtinib for ROS1-positive non–small cell lung cancer targets PDUFA date on September 18, 2026.
- UX111 for Sanfilippo syndrome type A has a PDUFA date on September 19, 2026.
- Zilganersen for Alexander disease targets PDUFA date on September 22, 2026.
- Mavacamten for obstructive hypertrophic cardiomyopathy aims for PDUFA date on September 30, 2026.
Guideline-Based Recommendations
Diagnosis
- Applications accepted for review based on clinical trial data.
Management
- Investigational therapies are under review for specific conditions.
Monitoring & Follow-up
- Long-term clinical data included in resubmissions for certain therapies.
Risks
- Applications granted Breakthrough Therapy and Orphan Drug designations indicate potential unmet medical needs.
Patient & Prescribing Data
Patients with specific conditions as outlined in the applications.
Applications supported by clinical trial results demonstrating efficacy.
Clinical Best Practices
- Monitor FDA PDUFA dates for updates on investigational therapies.
- Consider the implications of Breakthrough Therapy and Orphan Drug designations.
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