Orchard Therapeutics announced the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has granted an accelerated assessment for OTL-200, a gene therapy in development for the treatment of metachromatic leukodystrophy (MLD) in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy.
“We are pleased that OTL-200 has been granted accelerated assessment and 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,” said Anne Dupraz-Poiseau, Ph.D., chief regulatory officer of Orchard Therapeutics. “We look forward to working with the EMA to ensure this potentially transformative new treatment, if approved, reaches patients in the EU as quickly as possible, and continuing our efforts to expand patient access outside the EU.”
The EMA awards an accelerated assessment to medicines that are expected to be of major public health interest, particularly in the area of therapeutic innovation. Accelerated assessment potentially provides a reduced review timeline from 210 to 150 days once the Marketing Authorization Application (MAA) is filed and validated, not counting clock stops when applicants are requested to provide additional information. The decision to grant accelerated assessment has no impact on the eventual CHMP and Committee for Advanced Therapies (CAT) opinion on whether a marketing authorization should be granted.
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Metachromatic leukodystrophy (MLD) is a rare and life-threatening inherited disease of the body’s metabolic system occurring in approximately one in every 100,000 live births. MLD is caused by a mutation in the arylsulfatase-A (ARSA) gene that results in the accumulation of sulfatides in the brain and other areas of the body, including the liver, the gallbladder, kidneys, and/or spleen. Over time, the nervous system is damaged and patients with MLD will experience neurological problems such as motor, behavioral and cognitive regression, severe spasticity and seizures, finding it more and more difficult to move, talk, swallow, eat and see. Currently, there are no effective treatments for MLD. In its late infantile form, mortality at 5 years from onset is estimated at 50% and 44% at 10 years for juvenile patients.1 OTL-200 is an ex vivo, autologous, hematopoietic stem cell-based gene therapy being studied for the treatment of MLD. OTL-200 was acquired from GSK in April 2018 and originated from a pioneering collaboration between GSK and the Hospital San Raffaele and Fondazione Telethon, acting through their joint San Raffaele-Telethon Institute for Gene Therapy in Milan, initiated in 2010.