#Crl-Resolved

2 stories

Regulatory September 17, 2026 · View digest

FDA Grants Standard Full Approval to Ultragenyx FAYUVI (Rebisufligene Etisparvovec-Hopf), the First Approved Treatment for Sanfilippo Syndrome Type A

Ultragenyx Pharmaceutical announced on Thursday, September 17, 2026 that the FDA granted standard full approval to FAYUVI (rebisufligene etisparvovec-hopf), a single-dose intravenous AAV9 gene therapy, for the neurologic manifestations of mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome type A) in pediatric patients with preserved neurodevelopmental function. The company calls it the first FDA-approved treatment for Sanfilippo syndrome type A and its second gene therapy approval. Treated children (n=17) had a 23.5-point higher cognitive score than natural-history controls (n=27), with follow-up of up to nearly 8 years and reduced cerebrospinal fluid heparan sulfate. Ultragenyx received a priority review voucher, expects to ship within 30 to 60 days, and makes FAYUVI in Bedford, Massachusetts and at Andelyn Biosciences in Columbus, Ohio. BioSpace reported a $3.95 million wholesale acquisition cost, an initial FDA rejection in July 2025 over manufacturing issues, and a 13% share gain on the day.

Regulatory September 12, 2026 · View digest

FDA Approves Pharming's Joenja (Leniolisib) as First Treatment for Children Aged 4-11 With Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS); U.S. Launch October 2026

Pharming Group N.V. (NASDAQ: PHAR) announced Friday September 11, 2026 that the FDA approved the company's supplemental new drug application (sNDA) for Joenja (leniolisib, an oral selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor) at 40 mg and 50 mg twice-daily dosing for children aged 4 to 11 years weighing at least 27 kg with activated phosphoinositide 3-kinase delta syndrome (APDS). Joenja becomes the first FDA-approved treatment for children aged 4-11 with APDS, a rare primary immunodeficiency driven by gain-of-function mutations in the PIK3CD or PIK3R1 genes producing constitutive PI3Kδ pathway activation and recurrent sinopulmonary infections. Approval follows the February 2026 complete response letter, which required additional supportive data. The newly approved doses will be available through Pharming's specialty distribution network and patient-support infrastructure starting October 2026. Joenja was originally approved in March 2023 for APDS patients aged 12 and older; the pediatric label expansion extends the treated population substantially.