PTC Therapeutics

FDA approves Kebilidi, the first gene therapy for aromatic L-amino acid decarboxylase (AADC) deficiency, administered via 4 infusions into the brain's putamen to restore dopamine production. Based on a study of 13 pediatric patients, 8 of 12 showed improved gross motor function. Common adverse reactions include dyskinesia and fever. Approved via the Accelerated Approval pathway, continued approval may depend on ongoing confirmatory trials.

FDA approves PTC Therapeutics' Kebilidi, a one-time gene therapy for AADC deficiency, administered directly to the brain. This marks the first FDA-approved treatment for AADC deficiency and the first U.S. gene therapy directly administered to the brain. Kebilidi uses a modified virus to deliver a healthy DDC gene, easing symptoms and improving motor function. The therapy is administered via a one-time surgical procedure and is contraindicated in patients with immature skulls. PTC Therapeutics received a Rare Disease Priority Review Voucher.

PTC Therapeutics' gene therapy for AADC deficiency, KEBILIDI™, is the first FDA-approved treatment directly administered to the brain. Indicated for children and adults, it aims to restore dopamine production via a minimally invasive neurosurgical procedure. Expected to net over $250M by 2028, it received accelerated approval based on ongoing trial results.

The FDA granted accelerated approval for Kebilidi, a gene therapy for AADC deficiency, a rare genetic disorder causing severe disability. Developed by PTC Therapeutics, Kebilidi is the first U.S. gene therapy directly administered to the brain, delivered via a stereotactic surgical procedure. It showed efficacy in a trial with 13 pediatric patients, with 8 meeting new gross motor milestones, compared to none in an untreated cohort. Common adverse reactions include dyskinesia and procedural complications.

William Blair analyst Sami Corwin maintains a Buy rating on PTC Therapeutics (PTCT) stock due to the FDA approval of Kebilidi, a groundbreaking gene therapy for AADC deficiency, and the company's proactive commercial launch preparations. Corwin's rating is further supported by the priority review voucher and promising clinical trial results.

FDA grants accelerated approval for PTC Therapeutics' KEBILIDI, the first US gene therapy directly delivered to the brain for AADC deficiency, showing promising clinical results in restoring dopamine production.

PTC Therapeutics announces FDA approval of KEBILIDI™, the first gene therapy directly administered to the brain for AADC deficiency, covering children and adults. The therapy, delivered via stereotactic neurosurgery, restores dopamine synthesis, with a priority review voucher granted.

FDA approves PTC Therapeutics' gene therapy Kebilidi for AADC deficiency in the US, marking the first gene therapy administered directly into the brain. The therapy, already available in Europe and the UK, aims to replace the mutated DDC gene, enabling dopamine synthesis and improving motor milestones in patients.

The FDA approved PTC Therapeutics’ gene therapy Kebilidi for aromatic L-amino acid decarboxylase deficiency, marking the first brain-delivered gene therapy. Kebilidi, indicated for all ages and severities, showed 67% of treated patients achieving new gross motor milestones, contrasting with untreated patients.