The Japan-based pharma is acquiring Orchard Therapeutics for approximately $477.6 million, if all conditions are met. Orchard’s pediatric gene therapy has a PDUFA date set for March 2024.
Pictured: DNA particle complex/iStock, peterschreiber.media
On the potential cusp of FDA approval for its rare disease gene therapy, Orchard Therapeutics is being purchased by Japanese pharma Kyowa Kirin for close to $478 million if all conditions are met, the companies announced on Thursday.
Orchard’s pediatric gene therapy Libmeldy (atidarsagene autotemcel) is approved in the European Union and U.K., and is awaiting the FDA’s decision on approval with a PDUFA date of March 18, 2024. The Biologics License Application was accepted in September 2023.
Libmeldy is an ex vivo gene therapy in which the patient’s stem cells are taken, the faulty gene is replaced and reinjected back into the patient. It’s the first therapy approved for eligible patients with early-onset metachromatic leukodystrophy (MLD), a rare metabolic disease that causes severe damage to the nervous system and organs.
In its severest form, babies with MLD begin life developing normally but late in infancy rapidly lose the ability to walk, talk and interact with the world around them. The majority pass away within five years. In clinical studies, Orchard’s gene therapy preserved motor function and cognitive development in the treated children.
Despite great potential, gene therapies have been slow to enter the market. Due to the complexity of manufacturing, pricing is typically set at seven figures.
Libmeldy is currently considered the world’s most expensive drug at $3.4 million for the one-time treatment, though NHS England is said to have negotiated a “significant confidential discount” last year. Germany was able to negotiate a price of $2.6 million, while a consortium from Belgium, Ireland and the Netherlands cut off reimbursement negotiations with Orchard, calling the pricing for Libmeldy unjustified.
Orchard has faced other gene therapy challenges. In March 2022, the company axed Strimvelis, a gene therapy program picked up from GSK for a rare immune condition. Since its approval, only 16 patients had received the therapy.
Now Kyowa Kirin will take the reins on marketing Libmeldy. In addition to the MLD treatment, the biopharma also gets two clinical-stage programs for a group of severe pediatric neurometabolic disorders, mucopolysaccharidosis type I Hurler’s syndrome (MPS-IH) and Sanfilippo syndrome. Additional early research programs include targets like a genetic form of Crohn’s disease and frontotemporal dementia.
Kyowa Kirin hopes to leverage Orchard’s gene therapy platform for areas it has targeted before such as oncology and autoimmune disease.
Kate Goodwin is a freelance life science writer based in Des Moines, Iowa. She can be reached at kate.goodwin@biospace.com and on LinkedIn.
