RARE Daily

Orchard Therapeutics Reaches Agreement with NHS England for MLD Gene Therapy

February 4, 2022

Orchard Therapeutics said it has reached an agreement with the National Health Service (NHS) that enables access to Libmeldy, the company’s gene therapy for metachromatic leukodystrophy (MLD) in England and Wales for children who fall within the scope of the European marketing authorization.

The agreement coincides with publication of the positive National Institute for Health and Care Excellence’s final evaluation determination, which recognized the clinical impact and economic benefit of Libmeldy according to the institute’s Highly Specialized Technologies (HST) process.

“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 Bobby Gaspar, CEO of Orchard Therapeutics. “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.”

The announcement comes as the company also reported that the first two commercial patients had been treated 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.

MLD is a rare and life-threatening inherited disease of the body’s metabolic system occurring in approximately one in every 100,000 live births. MLD is caused by a mutation in the arylsulfatase-A (ARSA) gene that results in the accumulation of sulfatides in the brain and other areas of the body, including the liver, gallbladder, kidneys, and/or spleen. Over time, the nervous system is damaged, leading to neurological problems such as motor, behavioral, and cognitive regression, severe spasticity, and seizures. Patients with MLD gradually lose the ability to move, talk, swallow, eat, and see. In its late infantile form, mortality at five years from onset is estimated at 50 percent and 44 percent at 10 years for juvenile patients.

Libmeldy is the first therapy approved for eligible patients with early-onset MLD. It has been approved by the European Commission for the treatment of MLD in eligible early-onset patients characterized by biallelic mutations in the ARSA gene leading to a reduction of the ARSA enzymatic activity in children with late infantile or early juvenile forms without clinical manifestations of the disease, or the early juvenile form with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline.

The most common adverse reaction attributed to treatment with Libmeldy was the occurrence of anti-ARSA antibodies. In addition to the risks associated with the gene therapy, treatment with Libmeldy is preceded by other medical interventions, namely bone marrow harvest or peripheral blood mobilization and apheresis, followed by myeloablative conditioning, which carry their own risks. During the clinical studies, the safety profiles of these interventions were consistent with their known safety and tolerability.

Author: Rare Daily Staff

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