Upcoming September 2026 Prescription Drug User Fee Act dates include expected regulatory decisions for therapies and products targeting glioma, ROS1-positive non–small cell lung cancer, Sanfilippo syndrome type A, Alexander disease, fibrodysplasia ossificans progressiva, superficial basal cell carcinoma, monocarboxylate transporter 8 deficiency, symptomatic obstructive hypertrophic cardiomyopathy, and spinal muscular atrophy.
Oncology
TLX101-Px
Telix announced a September 11, 2026, Prescription Drug User Fee Act (PDUFA) goal date for its resubmitted new drug application for TLX101-Px (floretyrosine F 18), an investigational positron emission tomography imaging agent for glioma. The US Food and Drug Administration (FDA) accepted the application, which seeks approval of TLX101-Px for the characterization of recurrent or progressive glioma from treatment-related changes in adult and pediatric patients. The FDA previously granted TLX101-Px Orphan Drug and Fast Track designations.
Source: Telix Pharmaceuticals
Oncology
Zidesamtinib
Nuvalent announced a September 18, 2026, PDUFA target action date for its new drug application for zidesamtinib, an investigational ROS1-selective inhibitor. The FDA accepted the application, which seeks approval of zidesamtinib for adult patients with locally advanced or metastatic ROS1-positive non–small cell lung cancer who received at least 1 prior ROS1 tyrosine kinase inhibitor. The application is supported by results from the phase 1/2 ARROS-1 trial. Zidesamtinib has received Breakthrough Therapy designation for patients with ROS1-positive metastatic non–small cell lung cancer previously treated with 2 or more ROS1 tyrosine kinase inhibitors and Orphan Drug designation for ROS1-positive non–small cell lung cancer.
Source: Nuvalent, Inc.
Neurology
UX111
Ultragenyx announced a September 19, 2026, PDUFA action date for its resubmitted biologics license application seeking accelerated approval of UX111 (rebisufligene etisparvovec), an investigational adeno-associated virus 9 gene therapy, to treat patients with Sanfilippo syndrome type A. The FDA accepted the application for review. The resubmission includes updated long-term clinical data with up to 8 years of follow-up. The FDA previously granted the application Priority Review and the UX111 program Regenerative Medicine Advanced Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug designations.
Source: Ultragenyx Pharmaceutical Inc.
Neurology
Zilganersen
Ionis Pharmaceuticals announced a September 22, 2026, PDUFA target action date for its new drug application for zilganersen, an investigational RNA-targeted medicine for Alexander disease. The FDA accepted the application and granted Priority Review. The application is supported by results from a pivotal phase 1 to 3 study in pediatric and adult patients with Alexander disease. In the study, zilganersen 50 mg demonstrated stabilization in gait speed compared with control at 61 weeks. Breakthrough Therapy, Orphan Drug, and Rare Pediatric Disease designations were previously granted by the FDA to zilganersen.
Source: Ionis Pharmaceuticals
Rheumatology
Zilurgisertib
Incyte and Mirum Pharmaceuticals announced a September 26, 2026, PDUFA target action date for the new drug application for zilurgisertib, an investigational oral activin receptor-like kinase 2 inhibitor, to treat fibrodysplasia ossificans progressiva in patients aged 12 years or older. The FDA accepted the application and granted Priority Review. The application is supported by results from the pivotal phase 2 PROGRESS study. At 24 weeks, fewer patients receiving zilurgisertib developed new heterotopic ossification lesions, and the total volume of new lesions was lower with zilurgisertib vs placebo.
Source: Incyte
Dermatology
Aminolevulinic Acid
Biofrontera announced a September 28, 2026, PDUFA target action date for its supplemental New Drug Application for aminolevulinic acid hydrochloride (Ameluz) topical gel used with the RhodoLED red-light lamp series to treat superficial basal cell carcinoma. The FDA accepted the application for filing and identified no filing deficiencies. If approved, the indication would expand the current FDA approval of aminolevulinic acid hydrochloride photodynamic therapy beyond actinic keratosis to include superficial basal cell carcinoma.
Source: Biofrontera
Endocrinology
Tiratricol
Egetis Therapeutics announced a September 28, 2026, PDUFA target action date for its new drug application for tiratricol (Emcitate) to treat monocarboxylate transporter 8 deficiency. The FDA accepted the application and granted Priority Review. According to Egetis Therapeutics, the application is based on clinical data from Triac Trial I, Triac Trial II, ReTRIACt, the EMC Cohort Study, the EMC Survival Study, and the US Expanded Access Program. Tiratricol previously received Breakthrough Therapy, Rare Pediatric Disease, and Orphan Drug designations.
Source: Egetis Therapeutics
Cardiology
Mavacamten
Bristol Myers Squibb announced a September 30, 2026, PDUFA date for its supplemental new drug application for mavacamten (Camzyos) to treat adolescent patients aged 12 to younger than 18 years with symptomatic obstructive hypertrophic cardiomyopathy. The FDA accepted the application and granted Priority Review. The application is supported by results from the phase 3 SCOUT-HCM trial, which met its primary endpoint. At 28 weeks, mavacamten reduced Valsalva left ventricular outflow tract gradient from baseline compared with placebo. Mavacamten is currently approved in the US for adult patients with symptomatic New York Heart Association class II to III obstructive hypertrophic cardiomyopathy.
Source: Bristol Myers Squibb
Neurology
Apitegromab
Scholar Rock announced a September 30, 2026, PDUFA action date for its biologics license application for apitegromab to treat spinal muscular atrophy. The FDA review is ongoing, and Scholar Rock continues to expect a decision by the September 30 PDUFA date.
Source: Scholar Rock
