UK NICE Expands Access to Spinraza for SMA Type 3

A decision from England’s National Institute for Health and Care Excellence (NICE) will make Spinraza (nusinersen) available to people with spinal muscular atrophy (SMA) type 3 who are unable to walk.

In England, Spinraza is available through a managed access agreement (MAA) between NICE, the National Health Service (NHS, England’s publicly funded healthcare system), and Biogen, Spinraza’s manufacturer. The MAA allows patients to be treated with Spinraza, while also continuing to collect data on the therapy’s safety, efficacy, and financial considerations.

Under the terms of the original MAA, Spinraza was available to nearly all SMA patients — except, notably, individuals with type 3 SMA who have lost their ability to walk.

“There are people with SMA who are not able to access treatment with nusinersen [Spinraza] under the terms of the MAA which began in July 2019. At the time we made a commitment that we would review new evidence on the potential benefits of nusinersen for type III SMA patients who are not currently receiving it,” Meindert Boysen, deputy chief executive and director of the Centre for Health Technology Assessment at NICE, said in a press release.

NICE began a review last fall to assess whether SMA type 3 patients who are unable to walk should be included in the MAA. The review is now complete and suggests that such inclusion is appropriate.

NICE is “pleased that the review has concluded that it is appropriate to extend the clinical eligibility criteria to allow access to nusinersen for type III SMA patients who aren’t able to walk,” Boysen said.

The new expansion will remove a rule that patients who could not regain walking function within 12 months of Spinraza treatment no longer would be eligible for the treatment.

“Biogen is delighted by this decision for non-ambulant type III SMA patients who previously had no therapeutic options available. Biogen remains fully committed to supporting NHS England to ensure this positive decision translates into services for all eligible patients, enabling access to nusinersen, without delay,” said Samuel Cooper, head of rare diseases business unit at ‎Biogen UK and Ireland.

NICE’s positive decision was pushed for by patient organizations, such as SMA UK.

“This is significant and much anticipated progress for people living with SMA Type 3,” Angela Smith-Morgan, CEO at SMA UK, said in a separate press release.

“I am incredibly proud of the role that SMA UK has been able to play in advocating for the need for the treatment to be accessible to all for whom it is clinically safe,” Smith-Morgan added. “I am delighted for our Community that their voices have been listened to.”

Efforts are underway to expand the infrastructure needed to deliver Spinraza to this wider group of patients, with plans for further expansion as restrictions made necessary by the COVID-19 pandemic are lifted. Individual care centers will be in touch with patients as the medication is rolled out.