The EMA granted Orchard Therapeutics’ (NASDAQ:ORTX) OTL-200 accelerated assessment for the treatment of metachromatic leukodystrophy (MLD).
MLD is caused by a mutation in the arylsulfatase-A gene, which results in the accumulation of sulfatides in the brain and other organs. The disease is characterized by severe neurological problems and currently has no effective treatments.
OTL-200 is a stem cell-based gene therapy Orchard is developing in partnership with Italy’s San Raffaele-Telethon Institute for Gene Therapy.
In a statement, Orchard’s chief regulatory officer Anne Dupraz-Poiseau, said, “we believe this underscores the quality of our clinical data and the urgent need to bring this novel, potentially curative treatment option to patients living with MLD.”
