FDA Approves PTC Therapeutics' Neuro Gene Therapy

PTC Therapeutics has gained FDA accelerated approval for Kebilidi (eladocagene exuparvovec-tneq), a pioneering gene therapy designed to treat aromatic L-amino acid decarboxylase (AADC) deficiency. This landmark approval is the first in the U.S. for a gene therapy that is directly administered to the brain.

Kebilidi is approved for use in both children and adults who suffer from this severe genetic disorder. AADC deficiency disrupts the production of dopamine, a critical neurotransmitter for motor function. The disorder severely affects patients' quality of life, leading to major motor impairments and life-threatening complications such as frequent infections and respiratory issues.

The approval of Kebilidi is founded on substantial evidence from ongoing clinical trials that have demonstrated the therapy's safety and efficacy. Patients receiving Kebilidi have shown the ability to synthesize dopamine, which is crucial for motor development. Notably, the treated patients have exhibited significant improvements in their motor milestones.

The administration of Kebilidi requires a minimally invasive neurosurgical procedure that targets the putamen, a specific region in the brain. This direct approach addresses the genetic cause of AADC deficiency, offering new hope for patients who have previously relied on extensive supportive care to manage their symptoms.

In addition to the approval, PTC Therapeutics has received a rare disease priority review voucher from the FDA, which the company plans to monetize. This voucher is an incentive provided by the FDA to encourage the development of treatments for rare diseases, further highlighting the significance of Kebilidi’s approval.