New Sanfilippo Syndrome Treatment
FDA approves the first-ever gene therapy to treat Sanfilippo syndrome type A.
Anonymous
Key Points
1
FAYUVI’s generic name is rebisufligene etisparvovec-hopf, also known as UX111, and the FDA approval came with a Priority Review Voucher for Ultragenyx.
2
Ultragenyx CEO Emil D. Kakkis said the company was working with treatment centers and payers to support timely access, describing the approval as an important first step toward bringing the therapy to families.
3
Ultragenyx shares rose 12% to $14.44 after the approval, although the stock was still down 37% for the year, according to MarketWatch.
4
The FDA approval had previously been scheduled for a Sept. 19 Prescription Drug User Fee Act action date.
The FDA approved FAYUVI, a gene therapy made by Ultragenyx Pharmaceutical, for children with Sanfilippo syndrome type A — a rare and fatal brain disease. Fierce Pharma reports this is the first treatment designed to fix the underlying cause rather than just manage symptoms. The company set the price at $3.95 million and expects to ship the therapy to treatment centers within 30 to 60 days.
Ultragenyx CEO Emil D. Kakkis said the company is working with hospitals and insurers to get the therapy to families quickly. The stock jumped 12% to $14.44 after the announcement, though shares remain down 37% for the year, MarketWatch reported.
A Fatal Childhood Brain Disease Gets Its First Cure
Sanfilippo syndrome type A, also called MPS IIIA, slowly destroys the brain and nervous system in young children. Freedom 96.9 reports the disease is caused by a genetic deficiency that prevents the body from breaking down certain molecules. Kids with Sanfilippo typically lose thinking and movement skills, and the disease is fatal. Until now, doctors could only treat symptoms — not stop the disease itself.
How FAYUVI Targets the Root Problem
FAYUVI's scientific name is rebisufligene etisparvovec-hopf, also known as UX111. Quartr explains that this gene therapy is the first approved treatment designed to fix the genetic problem causing Sanfilippo type A. Instead of just managing symptoms, the therapy addresses what's broken inside patients' cells. The FDA granted a Priority Review Voucher as part of the approval, recognizing the treatment's importance.
Ultragenyx's Major Win for a Struggling Biotech
This approval marks Ultragenyx's second gene therapy approval and sixth FDA approval overall. Market Screener reports the company set the U.S. list price at $3.95 million per treatment. After the announcement, Ultragenyx shares rose 12% to $14.44, though the stock has struggled this year. CEO Kakkis emphasized the company's focus on helping families access the therapy despite its high cost.
Sentiment
Mostly PositiveNEGATIVENEUTRALPOSITIVE
Publishers
19
Articles
77
Reach
96
A
AnonymousAuthor
