The FDA has approved the first gene therapy for aromatic L-amino acid decarboxylase deficiency, a rare enzyme deficiency that leads to muscle and developmental issues. Developed by PTC Therapeutics, the gene therapy, known as eladocagene exuparvovec, is administered directly into the brain to replace the mutated DDC gene with a functioning version. The therapy has shown efficacy in pediatric patients, improving motor function and milestones in many patients. The approval of this gene therapy is seen as a positive development in the gene therapy field and could bring significant revenue to PTC Therapeutics.
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PTC Therapeutics Wins First FDA Approval for a Gene Therapy Dosed Directly Into the Brain
