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PTC Therapeutics' Gene Therapy Kebilidi Receives FDA Approval for AADC Deficiency

• The FDA has granted accelerated approval to PTC Therapeutics' Kebilidi, a gene therapy for Aromatic L-amino acid decarboxylase (AADC) deficiency, marking the first such therapy approved in the U.S. • Kebilidi delivers a functioning DDC gene directly into the brain, addressing the genetic defect underlying AADC deficiency, a severe neurological disorder affecting infants. • The approval is based on early motor function improvements observed in clinical studies, with continued monitoring required to confirm sustained clinical benefits. • This approval provides a significant boost for PTC Therapeutics, offering a new treatment option for a rare and debilitating genetic disease and a valuable priority review voucher.

PTC Therapeutics has secured FDA approval for Kebilidi, a gene therapy designed to treat Aromatic L-amino acid decarboxylase (AADC) deficiency. This marks the first gene therapy approved in the U.S. for direct administration to the brain. The approval, granted on Wednesday, offers a new therapeutic avenue for patients with this rare and often fatal enzyme disorder.
AADC deficiency is a genetic condition that disrupts the way neurons transmit information, severely impacting both physical and mental development. Typically manifesting within the first six months of life, the disorder affects all aspects of a child's life. Kebilidi is designed to correct the underlying genetic defect by delivering a functional DDC gene into the patient's body via neurosurgical infusions.
The FDA's decision is based on an accelerated approval pathway, relying on data demonstrating early improvements in motor function in a study of 12 patients, eight of whom showed positive responses. Continued approval is contingent upon further data confirming sustained clinical benefits. In its press release, the FDA highlighted the approval as representing "important progress" in treating debilitating genetic disorders.

Clinical Efficacy and Administration

Kebilidi is administered through four infusions in a single session, performed by a neurosurgeon. The therapy aims to provide a functional copy of the DDC gene, which is deficient in patients with AADC deficiency. The clinical trial data supporting the approval indicated improvements in motor skills following administration.

Financial Implications for PTC Therapeutics

The approval of Kebilidi is a significant milestone for PTC Therapeutics, particularly after facing setbacks with other pipeline products. The company's stock experienced volatility following a Phase 3 study failure for a Friedreich ataxia drug and a negative regulatory decision in Europe for its Duchenne muscular dystrophy medicine, Translarna. The FDA also awarded PTC a rare pediatric disease priority review voucher, which the company plans to sell. These vouchers are valuable assets, with Acadia Pharmaceuticals recently selling one for $150 million.

Global Availability and Regulatory Status

Kebilidi is already approved in Europe, where it is marketed as Upstaza. The U.S. approval expands the availability of this gene therapy to a broader patient population, offering hope for improved outcomes in children with AADC deficiency.
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Reference News

[1]
PTC wins US approval of gene therapy for fatal enzyme disorder | BioPharma Dive
biopharmadive.com · Nov 14, 2024

PTC Therapeutics wins FDA approval for Kebilidi, the first U.S. gene therapy for direct brain administration, treating A...

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