PTC Therapeutics Wins First FDA Approval for a Gene Therapy Dosed Directly Into the Brain

MedCity News November 14, 2024
Frank Vinluan

The PTC Therapeutics gene therapy, Kebilidi, treats an enzyme deficiency that affects the body’s ability to produce dopamine, a neurotransmitter important for motor control. FDA approval for the product comes two years after it won its first regulatory approvals in Europe.

An ultra-rare enzyme deficiency that keeps infants from achieving proper muscle function among other developmental delays now has its first FDA-approved treatment. The new product, a gene therapy developed by PTC Therapeutics, is also the first gene therapy approved in the U.S. that’s administered directly into the brain.

The PTC therapy was tested only in pediatric patients, but the FDA approval covers the treatment of both children and adults who have the inherited disease, called aromatic L-amino acid decarboxylase...

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