
Dive Brief:
- PTC Therapeutics on Wednesday won Food and Drug Adminitration approval for Kebilidi, the first gene therapy cleared in the U.S. for direct administration to the brain.
- The treatment is designed for patients with aromatic L-amino acid decarboxylase, or AADC, deficiency, a condition that affects the way neurons transmit information to other cells. The potentially fatal disorder typically manifests in the first six months after babies are born and affects all aspects of their lives, both physical and mental.
- Kebilidi is designed to deliver a functioning DDC gene into the body, correcting the genetic defect that causes the disorder. It’s administered by a neurosurgeon in four infusions in one session.
Dive Insight:
The approval is welcome news for PTC, which has struggled with a number of setbacks that dented its share price. PTC stock climbed above $58 in May 2023, just before the company announced a Phase 3 study failure for a Friedreich ataxia drug. By Nov. 2023, after a negative regulatory decision for a Duchenne muscular dystrophy medicine in September, the shares were trading below $19.
PTC tried to overcome the setbacks by restructuring and slashing costs. Last May, the company fired its CFO, ended early research programs and announced plans to cut about 8% of its workforce. The decision by European regulators four months later to deny PTC’s bid to convert a conditional clearance for its Duchenne treatment Translarna to full approval triggered another round of layoffs.
Shares have climbed higher this year, even as the company tried and failed to overcome European regulators objections to Translarna.
The FDA clearance for Kebilidi is also conditional, using the FDA’s accelerated approval pathway. Regulators are allowing the therapy on the market based on a study that showed early improvements in motor function in eight of the 12 patients evaluated. The agency could revoke approval if additional data doesn’t show further clinical benefits.
Still, FDA officials touted Kebilidi’s promise in a press release, saying it represented “important progress” in treating debilitating genetic disorders. The agency also awarded PTC a rare pediatric disease priority review voucher, which the company plans to sell. The “fast passes” are valuable to drugmakers; just last week, Acadia Pharmaceuticals agreed to sell one for $150 million.
Kebilidi is already approved in Europe, where it’s sold as Upstaza. PTC shares fell about 3% to trade under $43 apiece in early trading Thursday.
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- Source: https://www.biopharmadive.com/news/ptc-fda-approval-aadc-deficiency-gene-therapy-kebilidi/732916/