The European Commission has granted marketing authorization to Upstaza (eladocagene exuparvovec) for treatment of aromatic L-amino acid decarboxylase (AADC) deficiency.
PTC Therapeutics, a global biopharmaceutical company, announced on July 20, 2022 that it had been granted a marketing authorization for Upstaza (eladocagene exuparvovec) by the European Commission for treatment of aromatic L-amino acid decarboxylase (AADC) deficiency in patients aged 18 months and older. According to a company press release, Eladocagene exuparvovec is the first approved disease-modifying treatment for AADC deficiency as well as the first marketed gene therapy that is directly infused into the brain.
AADC is a rare, fatal genetic disorder that typically results in severe disability from the first months of life. Eladocagene exuparvovec is a one-time, recombinant adeno-associated virus serotype 2-based gene replacement therapy that contains the human DDC gene. It is designed to correct the underlying genetic defect of AADC by delivering a functioning DDC gene into the putamen, increasing the AADC enzyme and restoring dopamine production.
"Today's approval from the European Commission for Upstaza for the treatment of AADC deficiency is momentous for patients, for PTC, as well as for the larger gene therapy community," said Stuart W. Peltz, CEO, PTC Therapeutics, in the press release. "We are proud to bring this innovative therapy to the marketplace so that patients may benefit [and] are ready to deliver this long-awaited treatment to patients as soon as possible."
Source: PTC Therapeutics
Trump Directs FDA, EPA to Reduce Regulatory Barriers to Domestic Pharma Manufacturing
May 7th 2025The White House is instructing FDA to increase fees for and inspections of foreign drug manufacturing plants and reduce the time required to approve such sites that will be newly constructed in the US.
New Cancer Research and Treatment Center in Development in London
May 1st 2025Aviva Capital Partners and developer Socius are investing £1 billion in a 12-acre site to be located at the London Cancer Hub next to The Institute of Cancer Research, London and The Royal Marsden NHS Foundation Trust’s Sutton site.
Trump Directs FDA, EPA to Reduce Regulatory Barriers to Domestic Pharma Manufacturing
May 7th 2025The White House is instructing FDA to increase fees for and inspections of foreign drug manufacturing plants and reduce the time required to approve such sites that will be newly constructed in the US.
New Cancer Research and Treatment Center in Development in London
May 1st 2025Aviva Capital Partners and developer Socius are investing £1 billion in a 12-acre site to be located at the London Cancer Hub next to The Institute of Cancer Research, London and The Royal Marsden NHS Foundation Trust’s Sutton site.
2 Commerce Drive
Cranbury, NJ 08512