FDA Approves PTC Therapeutics’ Kebilidi

FDA has approved PTC Therapeutics’ Kebilidi (eladocagene exuparvovec-tneq), an adeno-associated virus vector-based gene therapy indicated to treat adult and pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency. An agency statement says Kebilidi is the first FDA-approved gene therapy to treat AADC deficiency.

The therapy’s safety and effectiveness were demonstrated in a clinical study involving 13 pediatric patients with a confirmed diagnosis of AADC deficiency. The most common adverse reactions are dyskinesia; fever; low blood pressure; anemia; increased saliva production; insomnia; low levels of potassium, phosphate, and/or magnesium; and procedural complications such as respiratory and cardiac arrest, FDA says.

Kebilidi was approved via the accelerated approval pathway and was granted priority review, orphan drug designation, and a rare pediatric disease priority review voucher by FDA.