PTC Receives Marketing Authorization from the European Commission for the Treatment of AADC Deficiency

The European Commission has granted marketing authorization to Upstaza (eladocagene exuparvovec) for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency.

PTC Therapeutics, a global biopharmaceutical company, announced on July 20, 2022 that it has received marketing authorization for Upstaza (eladocagene exuparvovec) from the European Commission for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency. in patients aged 18 months and older. According to a press release from the company, Eladocagene exuparvovec is the first approved disease-modifying treatment for AADC deficiency as well as the first marketed gene therapy infused directly into the brain.

AADC is a rare and fatal genetic disease that usually results in severe disability within the first few months of life. Eladocagene exuparvovec is a unique gene replacement therapy based on recombinant adeno-associated virus serotype 2 which contains the human DDC gene. It is designed to correct the underlying genetic defect of AADC by delivering a functional DDC gene into the putamen, increasing the AADC enzyme and restoring dopamine production.

“Today’s European Commission approval of Upstaza for the treatment of AADC deficiency is momentous for patients, for PTC, and for the broader gene therapy community,” said Stuart W. Peltz, CEO of PTC Therapeutics, in the press release. “We are proud to bring this innovative therapy to market so that patients can benefit [and] are ready to deliver this long-awaited treatment to patients as soon as possible. »

Source: PTC Therapeutics

About Antoine L. Cassell

Check Also

Learning Disabilities Treatment Market Size, Scope, Revenue, Opportunities and Growth by 2028 – Shanghaiist

New Jersey, United States – Verified Market Research recently released a new report titled Learning …