Orchard Therapeutics, a UCLB spinout and global gene therapy leader, has announced that it has reached an agreement with the National Health Service (NHS) that enables access to Libmeldy® (atidarsagene autotemcel) for all children with metachromatic leukodystrophy (MLD) in England and Wales who fall within the scope of the European marketing authorisation. The agreement coincides with publication of the positive National Institute for Health and Care Excellence’s (NICE) final evaluation determination, which recognised the clinical impact and economic benefit of Libmeldy according to the institute’s Highly Specialised Technologies (HST) process. In addition, the company announced the first two commercially treated patients in Germany and France under reimbursement mechanisms for early access. A third patient from the Middle East was referred for international reimbursed treatment abroad in Italy.
“Today’s landmark agreement with NHS England follows a thoughtful and comprehensive value assessment by NICE and represents a major milestone for the MLD community, Orchard, and the entire field of HSC gene therapy,” said Professor Bobby Gaspar, M.D., Ph.D., chief executive officer of Orchard Therapeutics. “We are delighted that NICE and NHS England have recognised the transformational clinical impact and significant economic value of Libmeldy for eligible MLD patients, and thank the leading clinicians and advocates involved for their tireless engagement throughout the process. A deep body of evidence now points to the potential for durable effects in HSC gene therapy for certain severe genetic diseases including MLD. I am grateful for the opportunity we have at Orchard to commercially scale the reach of our therapeutic approach for patients in need—starting with MLD.”