Rare Daily Staff

The Committee for Medicinal Products for Human Use of the European Medicines Agency has granted accelerated assessment for Orchard Therapeutics’ OTL-200, a gene therapy in development for the treatment of metachromatic leukodystrophy.

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, the kidneys, and the spleen. Over time, the nervous system is damaged and patients with MLD will experience neurological problems such as motor, behavioral and cognitive regression and 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 five years from onset is estimated at 50 percent and 44 percent at 10 years for juvenile patients.

OTL-200 is an ex vivo, autologous, hematopoietic stem cell-based gene therapy being studied for the treatment of MLD. OTL-200 is one of four cell-based gene therapy programs acquired from GlaxoSmithKline in April 2018 that 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, initiated in 2010 in Milan.

“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, chief regulatory officer of Orchard Therapeutics.

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 an application requesting marketing authorization 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 whether a marketing authorization should be granted.

Photo: Anne Dupraz-Poiseau, chief regulatory officer of Orchard Therapeutics

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