PTC Therapeutics

FDA approves PTC Therapeutics' Kebillidi for AADC deficiency, marking the first gene therapy in the US. Based on a study showing motor milestone gains in most patients, Kebillidi improves gross motor function in severe cases. Common adverse events include dyskinesia and procedural complications. Kebillidi is contraindicated in immature skulls and approved under Accelerated Approval.

PTC Therapeutics announces FDA accelerated approval of Kebilidi, the first gene therapy directly administered to the brain for AADC deficiency, with launch preparations underway.

The FDA approved PTC Therapeutics' Kebilidi gene therapy for AADC deficiency, marking the first FDA-approved treatment for the rare genetic disorder. Kebilidi, administered via four infusions into the brain, aims to increase dopamine production. The approval was based on a study showing its safety and effectiveness in 13 pediatric patients. Adverse reactions include dyskinesia and fever. The FDA used the Accelerated Approval pathway, with a confirmatory trial ongoing.

The FDA approved eladocagene exuparvovec (Kebilidi) for AADC deficiency, marking the first gene therapy for this disorder. The treatment, delivered via a recombinant adeno-associated virus serotype 2, increases AADC enzyme and dopamine production in the brain. It showed improvement in gross motor function in a trial of 13 pediatric patients with severe AADC deficiency. Common adverse reactions include dyskinesia and fever, with procedural complications reported. The therapy is contraindicated in patients with immature skulls and not studied in those under 16 months or over 65. A confirmatory trial is ongoing.

Terumo BCT launches Global Therapy Innovations unit to enhance patient access to advanced therapies. FDA approves PTC Therapeutics' gene therapy KEBILIDI for AADC deficiency, the first brain-administered gene therapy in the US. Autolus Therapeutics' CAR-T cell therapy AUCATZYL receives FDA approval for relapsed/refractory B-cell acute lymphoblastic leukemia in adults.

PTC Therapeutics wins FDA approval for Kebilidi, the first U.S. gene therapy for direct brain administration, treating AADC deficiency. The approval, via the FDA’s accelerated pathway, is based on early motor function improvements in study patients. Despite recent setbacks, including a failed Phase 3 study and European regulatory rejections, the approval offers PTC a rare pediatric disease priority review voucher, which can be sold.

FDA approves first gene therapy, Kebilidi, for aromatic L-amino acid decarboxylase (AADC) deficiency, administered directly into the brain. Tested in 13 pediatric patients, Kebilidi showed gross motor function improvement in 8 of 12 patients at 48 weeks, with better outcomes for younger patients. The therapy, already approved in Europe, aims to replace the mutated DDC gene with a functional one, increasing dopamine production. The FDA's accelerated approval requires additional data, with long-term follow-up planned. PTC projects peak revenue of $266.3 million in 2026, and the approval includes a priority review voucher.

FDA grants accelerated approval for PTC Therapeutics’ KEBILIDI, the first US gene therapy directly delivered to the brain for AADC deficiency, based on clinical trial efficacy. KEBILIDI replaces the defective gene via a neurosurgical procedure, restoring dopamine production and motor function.

PTC Therapeutics receives FDA approval for Kebilidi, a gene therapy for AADC deficiency, marking the first US-approved brain-administered gene therapy.

FDA grants conditional approval to PTC Therapeutics' gene therapy for AADC deficiency, the first to be directly administered to the brain. Marketed as Kebilidi, it treats children and adults with AADC deficiency, improving motor function by synthesizing dopamine. Administered via four infusions in one surgical session, it demonstrated efficacy in 8 of 12 treated patients.