The European Medicines Agency has granted Orphan Drug Designation (ODD) to Amsterdam Molecular Therapeutics’ gene therapy product AMT-080 for the treatment of Duchenne muscular dystrophy.
ODD is awarded to pharmaceutical companies to encourage research into rare diseases and for products which have a small market.
ODD for Duchenne muscular dystrophy (DMD) entitles AMT to ten-year market exclusivity in Europe following marketing approval for AMT-080.
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.
AMT chief executive officer Jorn Aldag said that this designation is an important next step in the development of a treatment for this progressive and devastating disease.
DMD is a severe disease characterised by progressive muscle degeneration in children (almost exclusively boys) leading to paralysis and death in young adulthood.
AMT’s gene therapy is based on technology that results in skipping the defective portion of the dystrophin gene, which causes the disease, resulting in the formation of functional protein. Positive long-term health effects of this approach have been demonstrated in animals.