Health
Kyowa Kirin and Orchard Therapeutics Announce OTL-200 Granted Orphan Regenerative Medicine Product Designation for Early-onset MLD in Japan
– First and only disease-modifying therapy for the treatment of eligible children with MLD also receives Orphan Drug Designation and Priority Review in the Kingdom of Saudi Arabia –TOKYO, LONDON and BOSTON, Oct. 28, 2025 (GLOBE NEWSWIRE) -- Kyowa Kirin Co., Ltd., (TSE: 4151, President and COO: Abdul Mullick, "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, also know
About this update from Kyowa Kirin Co., Ltd.
[{"type":"image","alt":"Orchard Therapeutics (Europe) Limited","displaySize":"","headline":null,"caption":"Orchard Therapeutics (Europe) Limited","className":"","disableSlideshowImg":false,"size":{"original":{"width":300,"height":149,"url":"https://media.zenfs.com/en/globenewswire.com/f7c5ab86662cc16a57f6f696e0294e9f"},"resized":{"url":"https://s.yimg.com/ny/api/res/1.2/835KIgttjwaVXyE.TnVDAg--/YXBwaWQ9aGlnaGxhbmRlcjt3PTQyMDtoPTIwOTtjZj13ZWJw/https://media.zenfs.com/en/globenewswire.com/f7c5ab86662cc16a57f6f696e0294e9f","width":300,"height":149}},"lazy":false},{"type":"text","content":"– First and only disease-modifying therapy for the treatment of eligible children with MLD also receives Orphan Drug Designation and Priority Review in the Kingdom of Saudi Arabia –","length":181,"tagName":"p"},{"type":"text","content":"TOKYO, LONDON and BOSTON, Oct. 28, 2025 (GLOBE NEWSWIRE) -- Kyowa Kirin Co., Ltd., (TSE: 4151, President and COO: Abdul Mullick, "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, also known as atidarsagene autotemcel, an investigational ex vivo autologous hematopoietic stem cell (HSC) gene therapy for the treatment of pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ), and early-symptomatic early juvenile (ESEJ)—collectively referred to as early-onset—metachromatic leukodystrophy (MLD).","length":662,"tagName":"p"},{"type":"text","content":"MLD is an ultra-rare, rapidly progressive, irreversible and ultimately fatal neurometabolic disease that affects approximately one in 100,000 live births based on existing literature. It is caused by an error in the gene responsible for encoding the enzyme arylsulfatase A (ARSA) leading to neurological damage and developmental regression. In the most severe form of MLD, babies develop normally but in late infancy start to rapidly lose the ability to walk, talk and interact with the world around them. These children eventually deteriorate into a vegetative state, which may require 24-hour intensive care, and the majority pass away within five years of symptom onset, creating an enormous emotional and financial burden on the family. There are currently no approved therapies for MLD in Japan beyond supportive and end-of-life care.","length"...