PTC Therapeutics, Inc., announced that the European Commission has granted a 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 non-profit, FDA-licensed manufacturer of vaccines, biologics and viral vector gene therapies in the United States. The European Commission’s approval paves the way for making this first-of-its-kind treatment available to patients. PTC’s announcement can be read here.
In partnership with PTC Therapeutics, MassBiologics provided contract manufacturing, process development and registration support.
“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 the few gene therapy treatments approved worldwide to improve the lives of patients with a range of diseases. Our partnership with PTC Therapeutics builds on our long history and mission to bring ‘Medicines for Better Lives’.”
AADC deficiency is a fatal genetic disorder that typically causes severe disability and suffering from the first few months of life and affects every aspect of life – physical, mental and behavioral. AADC is exceptionally rare and the lives of affected children are severely affected and shortened.
Stuart W. Peltz, PhD, chief executive officer, PTC Therapeutics, called the approval “important for patients, for PTC, as well as for the greater gene therapy community. We are proud to bring this innovative therapy to market so patients can benefit from it.” Upstaza is the first and only approved disease-modifying treatment for patients with AADC deficiency. We are poised to deliver this highly anticipated treatment to patients as soon as possible.”