Peptide News Digest

#Crl-Resolved

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Regulatory · View digest

FDA Grants Standard Full Approval of Ultragenyx FAYUVI (Rebisufligene Etisparvovec-Hopf) as First-Ever Treatment for Pediatric Sanfilippo Syndrome Type A / MPS IIIA; $3.95M WAC Price and Priority Review Voucher Awarded

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced Thursday September 17, 2026 that the FDA granted standard full approval to FAYUVI (rebisufligene etisparvovec-hopf, previously UX111) — a one-time intravenous AAV9-delivered gene therapy carrying a functional copy of the SGSH (N-sulfoglucosamine sulfohydrolase) gene — for the treatment of pediatric patients with mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome Type A). MPS IIIA is a progressive, ultimately fatal autosomal-recessive lysosomal storage disorder that causes rapid neurodegeneration in early childhood; before Thursday, no disease-modifying therapy existed. The approval landed two days ahead of the September 19 PDUFA action date, followed the July 2025 complete response letter that had cited chemistry-manufacturing-controls issues plus manufacturing-site observations. The clinical package supporting standard full approval (not accelerated): up to 8 years of follow-up in treated children, sustained cerebrospinal fluid heparan sulfate reduction (the accumulating substrate), and a 23.5-point cognitive-score advantage over natural-history controls. Ultragenyx received a Rare Pediatric Disease Priority Review Voucher — historically valued $150-350 million on the secondary market — alongside the approval, plus U.S. per-patient wholesale acquisition cost set at $3.95 million. RARE shares closed the day up 13% and extended gains after hours. FAYUVI is manufactured domestically at Andelyn Biosciences in Columbus, Ohio and Ultragenyx's own Bedford, Massachusetts facility. Second gene therapy approval for the company.

Regulatory · 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.