Orchard Therapeutics Receives Rare Pediatric Disease Designation for MLD Therapy


Orchard Therapeutics announced that its gene therapy candidate, OTL-200, has been granted Rare Pediatric Disease designation for the treatment of metachromatic leukodystrophy.

This morning, Orchard Therapeutics announced that its gene therapy candidate, OTL-200, has been granted Rare Pediatric Disease designation for the treatment of metachromatic leukodystrophy (MLD), a progressive neurometabolic condition.

Today’s news marks the fourth time that the company has received the designation from the U.S. Food and Drug Administration (FDA) for an autologous ex vivo gene therapy.

MLD is a rare, life-threatening, neurodegenerative, genetic disease resulting from mutations in the ARSA gene. In its late infantile and juvenile forms — which represents most MLD patients – mortality at 5 years is approximately 75% and 30%, respectively.

“In clinical trials, early treatment with OTL-200 has demonstrated preservation of cognitive and motor development to levels comparable with healthy individuals,” said Mark Rothera, president and CEO of Orchard in a press release. “We look forward to working with Telethon / Ospedale San Raffaele to bring this potentially life-changing treatment to patients as rapidly as possible.”

The company’s autologous ex vivo gene therapy technology makes use of the patient’s own stem cells. The stem cells are taken from the patient, for example, via leukapheresis of peripheral blood cells and/or bone marrow extract, and then corrected outside of the body using a lentiviral vector carrying a functioning copy of the faulty gene. The genetically corrected cells are then transplanted back into the patient’s body.

OTL-200 was acquired by Orchard from GSK in April 2018 and developed from an innovative collaboration between GlaxoSmithKline (GSK) and the Hospital San Raffaele and the Telethon Foundation. The companies partnered at their joint Telethon Institute for Gene Therapy in Milan, opened in 2010, and this collaboration led to the development of Strimvelis, the world’s first approved autologous ex vivo gene therapy product.

As a part of the agreement, GSK announced that it would be transferring its portfolio of approved and investigational gene therapies to Orchard Therapeutics in exchange for a 19.9% stake in the company and other additional assets.

"The addition of these programs is a really big step up in terms of activity, so we are going to be looking to raise further funds through an additional private round." Rothera said. As part of the deal, the companies agreed to exchange manufacturing, scientific and commercial knowledge. Rothera also noted that Orchard could also consider going public.

Orchard has stated its intentions of filing OTL-200 for market authorization with regulatory authorities from 2019.

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