Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, announced that the European Medicines Agency has granted Orphan Drug Designation to AMT's gene therapy product AMT-021 for the treatment of acute intermittent porphyria (AIP).
Orphan Drug Designation for AIP entitles AMT to ten year market exclusivity in Europe following marketing approval for AMT-021 if this product candidate is the first new drug with a major medical benefit receiving marketing approval for the European Union. The designation also provides for special benefits, including research support, eligibility for protocol assistance and possible exemptions or reductions in certain regulatory fees during development or at the time of application for marketing approval.
"We are proud to have received this Orphan Drug Designation for the treatment of acute intermittent porphyria," said Sander van Deventer, chief executive officer of AMT. "This designation is an important step in the development of a treatment for this seriously debilitating and potentially lethal disease."
AMT has a unique gene therapy platform that to date appears to circumvent many if not all of the obstacles that have prevented gene therapy from becoming a mainstay of clinical medicine.