Ultragenyx Pharmaceutical, Inc. RARE

Andelyn Biosciences ("Andelyn"), a leading, patient-focused cell and gene therapy Contract Development and Manufacturing Organization ("CDMO"), today announced it is now manufacturing FAYUVI™ (rebisufligene etisparvovec-hopf), an FDA-approved gene therapy, for commercial supply at its Columbus, Ohio facility. The announcement follows the U.S. Food and Drug Administration's approval of Ultragenyx Pharmaceutical Inc.'s FAYUVI™ for treating Sanfilippo syndrome type A, also known as mucopolysacchari

Wall Street raised Ultragenyx’s price target after the U.S. Food and Drug Administration approved Fayuvi to treat children with Sanfilippo syndrome Type A.

- FDA approval represents a monumental milestone for patients with MPS IIIA and validates Abeona’s foundational R&D and clinical development efforts - - Ultragenyx successfully brought a life-changing therapy across the finish line for MPS IIIA patients and families - CLEVELAND, Sept. 18, 2026 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO), a commercial-stage biopharmaceutical company developing genetic medicines for serious diseases with high unmet need, today congratulated Ultrage

Ultragenyx’s second gene therapy approval since August gives it another sellable voucher. Elsewhere, Roche declared success in a blood cancer trial and a cell therapy maker is cutting costs.

Abeona stands to collect mid-single-digit royalties on sales of Ultragenyx’s Fayuvi plus up to $30 million in commercial milestone payments as part of a licensing deal.

The Food and Drug Administration (FDA) granted full approval of Fayuvi, also known as UX111, for pediatric patients.

The biopharmaceutical company received FDA approval for a first-of-its-kind treatment for a rare neurodevelopmental disease.

Ultragenyx Pharmaceuticals won approval for its newest gene therapy on Thursday, helping shares partially reverse a catastrophic dive.

FAYUVI is a highly anticipated, first-ever treatment option with the potential to stop or slow the devastating, irreversible neurologic progression and loss of function associated with Sanfilippo syndrome Type A Ultragenyx’s UltraCare® program will support access, and commercial product is expected to be available to ship to Qualified Treatment Centers within 30-60 days FAYUVI marks the second gene therapy approval, and sixth FDA approval overall, for Ultragenyx The Company received a Priority R

RARE investors are now awaiting an FDA decision on UX111, the company’s one-time gene therapy for Sanfilippo syndrome type A.

Large-cap healthcare outperformed this week: XLV gained 1.4% through Tuesday, while XBI fell 1.4% and IBB declined 0.5%.