Pharmac funds second treatment for spinal muscular atrophy

Media release Medicines

Te Pātaka Whaioranga - Pharmac has announced the funding of risdiplam (branded as Evrysdi) for people with the rare genetic disorder spinal muscular atrophy, who meet eligibility criteria from 1 May 2023.

“We’re pleased to announce that we now have another treatment for people with spinal muscular atrophy,” says Pharmac’s director of operations Lisa Williams. “This means that there will be two funded options with the same access criteria for symptomatic and pre-symptomatic conditions, providing a choice for the person and their whānau to determine the treatment best suited to them.

“Making sure that there are easy ways to access treatment is incredibly important. Those using risdiplam will be able to pick it from an agreed collection point near them, like a community pharmacy or at a Te Whatu Ora hospital. The supplier, Roche, has supported the direct delivery of the treatment, which aims to improve the equity of access for those who are unable to travel to a specified location, usually in the cities.”

Alex Muelhaupt, General Manager of Roche Products (New Zealand), says the company is pleased to be helping improve access to medicines for people with spinal muscular atrophy. “It’s great that young people with spinal muscular atrophy, their families and healthcare practioners will now have another choice for treatment, especially for those people who find it hard to travel to hospitals or prefer to have an oral, at-home solution.”

“I want to acknowledge the work done by community advocates to help generate awareness around this condition and the need for the funding of innovative medicines like risdiplam. Roche looks forward to ongoing collaboration with everyone who helped us arrive at this decision, and to continue to improve the lives of people living with the condition.

Pharmac understands that there are still people who would like to see wider criteria for funded access to spinal muscular atrophy treatments, including for those who are aged over 18 years at the time they start treatment. Pharmac is actively considering this, having taken advice from its expert advisors on the Rare Disorders Advisory Committee in March 2023.

Risdiplam will be listed on the Pharmaceutical Schedule from 1 May 2023. The first treatment funded for spinal muscular atrophy, nusinersen (branded as Spinraza), was funded from 1 January 2023.