The European Medicines Agency (EMA )has granted Orphan Drug Designation to Amsterdam Molecular Therapeutics’ (AMT) gene therapy product AMT-080 for the treatment of Duchenne muscular dystrophy.
Reportedly, Orphan Drug Designation for Duchenne muscular dystrophy (DMD) entitles AMT to 10 year market exclusivity in Europe following marketing approval for AMT-080 if this product candidate is the new drug with a major medical benefit receiving marketing approval for the European Union.
Moreover, the designation 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.
Jorn Aldag, CEO of AMT, said: “We are proud to have received this Orphan Drug Designation for the treatment of Duchenne muscular dystrophy. This designation is an important next step in the development of a treatment for this progressive and devastating disease.”