World's most expensive drug given the green light in 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 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 muscles and 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 life-changing for patients and their families.

“However, it is extremely expensive for the single-dose required, even with the Patient Access Scheme discount offered by the company.

“These are incredibly difficult decisions. Allocating resource to new innovations diverts that resource from other important needs.

“We expect the pharmaceutical industry will work on improving its efficiency to deliver such high-cost innovations at lower cost, which is essential for the sustainability of healthcare systems around the world.”

Babies born with severe type 1 SMA, the most common form of the condition, have a life expectancy of just two years.

But studies show a one-time intravenous infusion of Zolgensma can help infants breathe without ventilators, sit up without help, and crawl and walk.

Meanwhile, entrecitinib, brand name Rozlytrek, was accepted for the treatment of cancer growths that have a rare genetic abnormality called NTRK gene fusion.

It is a new type of medicine, sometimes called a tumour agnostic, which can be effective in growths with specific genetic features, regardless of where the tumour is situated.

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