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PTC Therapeutics announces European Commission marketing authorization for Upstaza

First approved gene therapy treatment for AADC deficiency

PTC Therapeutics, Inc., announced that the European Commission has granted marketing authorization for Upstaza (eladocagene exuparvovec), the first approved gene therapy treatment developed for aromatic L-amino acid decarboxylase (AADC) deficiency. Upstaza  is manufactured in partnership with MassBiologics of UMass Chan Medical School, the only nonprofit, FDA-licensed manufacturer of vaccines, biologics and viral vector gene therapies in the United States. The European Commission’s approval clears the regulatory pathway for this first-of-its-kind treatment to be available to patients. PTC’s announcement can be read here.

In partnership with PTC Therapeutics, MassBiologics provided contract manufacturing, process development and registration support.

Mireli Fino, MBA

“We are proud to contribute to PTC Therapeutic’s drive to bring this much needed treatment to market,” said Mireli Fino, MBA, executive vice chancellor of MassBiologics. “Upstaza is one of only a handful of gene therapy treatments to be approved, worldwide, to improve the lives of patients diagnosed with a range of diseases. Our partnership with PTC Therapeutics builds upon our long history and mission of bringing ‘Medicines for Better Lives.’”

AADC deficiency is a fatal genetic disorder that typically causes severe disability and suffering from the first months of life, affecting every aspect of life—physical, mental and behavioral. AADC is exceptionally rare and the lives of affected children are severely impacted and shortened.

Stuart W. Peltz, PhD, chief executive officer, PTC Therapeutics, called the approval “momentous for patients, for PTC, as well as for the larger gene therapy community. We are proud to bring this innovative therapy to the marketplace so that patients may benefit. Upstaza is the first and only approved disease-modifying treatment for patients living with AADC deficiency. We are ready to deliver this long-awaited treatment to patients as soon as possible.”