Go-ahead for therapy to treat rare genetic disease

A MEDICINE known as the most expensive in the world has been approved to treat a rare genetic disease in children through NHS Scotland.

The Scottish Medicines Consortium (SMC) has given approval to onasemnogene abeparvovec, sold under the brand name Zolgensma, for the treatment of spinal muscular atrophy (SMA).

It is the first gene therapy the SMC has accepted for use by NHS Scotland.

SMA is a very rare muscle-wasting disease caused by a mutation in a gene that is critical to the function of the nerves that control. It is one of the main genetic causes of death in infants.

The drug, which replicates a missing gene and restores nerve and muscle function, has a reported list price of nearly £1.8 million per dose.

SMC chairman Mark Macgregor said: “The committee is pleased to be able to accept these medicines for use by NHS Scotland. Committee members heard about the devastating impact SMA has on the lives of children with this condition and their families through our PACE process.

“This is the first gene therapy the committee has accepted for use by NHS Scotland using our process for medicines for rare conditions, which allows us greater flexibility in relation to cost-effectiveness.

“This medicine has the potential to be lifechanging for patients and their families.”