Kyowa Kirin’s bet on Orchard Therapeutics has paid off, furnishing the Japanese drugmaker with a newly approved gene therapy in the U.S. that’s already started to prove itself in Europe over the past several years.

The FDA on Monday gave a thumbs-up to Orchard’s Lenmeldy, also known as atidarsagene autotemcel, as the first U.S.-approved gene therapy for kids with certain types of metachromatic leukodystrophy (MLD).

MLD, which affects around one in every 40,000 people in the U.S., is a rare genetic disease that affects the brain and nervous system. The condition arises from a deficiency of the enzyme known as arylsulfatase A (ARSA), which causes a buildup of fatty substances in a person’s cells, in turn dealing damage to the central and peripheral nervous systems.

As a gene therapy, Lenmeldy is given as a one-time, bespoke infusion made from the patient’s own hematopoietic stem cells that have been genetically modified to include functional copies of the ARSA gene.

Prior to treatment with Lenmeldy, patients must undergo high-dose chemotherapy to remove cells from the bone marrow so they can be replaced by modified cells in the gene therapy.

The FDA based its approval on data from 37 children who were given Lenmeldy in a pair of single-arm, open-label clinical trials as well as an expanded access program.

Orchard’s studies compared kids treated with Lenmeldy to untreated children, finding that patients treated with Lenmeldy saw significantly reduced motor impairment or death versus untreated children, according to an FDA press release.

Orchard snagged the first approval for its MLD gene therapy in December 2020 in Europe, where the drug is known as Libmeldy. A month after that nod in the EU, Libmeldy picked up a regenerative medicine advanced therapy designation from the FDA to hasten the med’s potential arrival stateside.

Orchard’s latest approval in the U.S. marks a win for Kyowa Kirin, too. Last October, the Japan-based company shelled out $387.4 million upfront to take over Orchard and get its hands on the biotech’s gene therapy, still coded at the time as OTL-200.

At the time of the transaction, Kyowa said it figured it could maximize the value of Lenmeldy in tandem with advancing the biotech’s pipeline of gene therapies targeting a range of conditions. Kyowa’s acquisition was designed, in part, to fill a gap left in the company’s pipeline after it stopped work on one of three major next-generation candidates—the Parkinson’s disease prospect KW-6356.

Kyowa Kirin and Orchard haven’t yet revealed a price for Lenmeldy in the U.S., but cost previously proved an issue for the medication in the U.K.

Back in July 2021, England’s drug cost watchdog the National Institute for Health and Care Excellence rejected the gene therapy for use in the U.K., noting the drug was too expensive and had yet to be proven long-term.

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