Rare Daily Staff

The U.S. Food and Drug Administration granted Orchard Therapeutics a Rare Pediatric Disease designation to the company’s gene therapy candidate OTL-200 for the treatment of patients with metachromatic leukodystrophy, a rare and fatal inherited metabolic disease.

MLD is caused by mutations in the ARSA gene. In its late infantile form, children generally live five to ten years after diagnosis. In the juvenile forms, patients generally live 10 to 20 years after diagnosis.

“This is the fourth Rare Pediatric Disease designation awarded by the FDA to one of Orchard’s autologous ex vivo gene therapy candidates, providing significant incentives to continue expanding our pipeline” said Mark Rothera, president and CEO of Orchard “MLD is a devastating disease in which most patients do not survive the first decade of life.”

Orchard acquired OTL-200 from GSK in April 2018. The company said in clinical trials, early treatment with OTL-200 has shown preservation of cognitive and motor development to levels comparable with healthy individuals.

The gene therapy was developed through a collaboration between GSK and the Hospital San Raffaele and the Telethon Foundation, acting through their joint Telethon Institute for Gene Therapy, in Milan. The collaboration led to the development of Strimvelis, the world’s first approved autologous ex vivo gene therapy product.

Orchard said it expects to file OTL-200 for market authorization with regulatory authorities in 2019.

The FDA grants Rare Pediatric Disease designation for diseases that primarily affect children from birth to 18 years old, and affect fewer than 200,000 persons in the United States. This program is intended to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.

If OTL-200 is approved by the FDA for MLD, the rare pediatric disease designation may enable Orchard to receive a priority review voucher. Priority review vouchers can be used by the sponsor to receive Priority Review for a future NDA or BLA submission which would reduce the FDA review time from twelve months to six months.

The vouchers are potentially lucrative because they are transferable. Most recently, Spark Therapeutics sold a priority review voucher at the end of April for $110 million.

May 3, 2018
Photo: Mark Rothera, president and CEO of Orchard Therapeutics

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