Kyowa Kirin and Orchard Therapeutics Announce OTL-200 Granted Orphan Regenerative Medicine Product Designation for Early-onset MLD in Japan

Metachromatic Leukodystrophy (MLD)

https://ir.orchard-tx.com/news-releases/news-release-details/kyowa-kirin-and-orchard-therapeutics-announce-otl-200-granted

Kyowa Kirin and Orchard Therapeutics announced that Japan’s Ministry of Health, Labor and Welfare (MHLW) has granted Orphan Regenerative Medicine Product Designation to OTL-200, a gene therapy for the treatment of pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ), and early-symptomatic early juvenile (ESEJ) which are collectively referred to as early-onset metachromatic leukodystrophy (MLD). There are currently no approved therapies for MLD in Japan beyond supportive and end-of-life care. Kyowa Kirin is currently preparing to initiate a clinical trial for OTL-200 in Japan. OTL-200 has also been granted Orphan Drug Designation and Priority Review from the Saudi Food and Drug Authority (SFDA) for the treatment of early-onset MLD. These programs are intended to encourage development of new therapies for rare and ultra-rare diseases by streamlining and expediting the review process.