Pharmac seeking feedback on funding a second spinal muscular atrophy treatment

Media release Medicines

Te Pātaka Whaioranga - Pharmac is initiating a consultation today, seeking feedback on a proposal to fund of risdiplam (branded as Evrysdi) for people with the rare genetic disorder spinal muscular atrophy, who meet the eligibility criteria.

Pharmac’s director of operations Lisa Williams says Pharmac is thrilled to be taking the next step towards progressing a second treatment for those with spinal muscular atrophy for funding, with nusinersen (branded as Spinraza) funded from 1 January 2023.

“Risdiplam has the potential to make a substantial difference to the lives of young people in New Zealand living with spinal muscular atrophy and for their whānau and support networks too,” says Ms Williams. “Having a treatment available that can be taken orally, from home and once a day has major benefits to improve access for those who need it.”

“We have received clinical advice from our Rare Disorders and Neurological Advisory Committees along with our Pharmacology and Therapeutics Advisory Committee (PTAC) recommending that we fund risdiplam. The eligibility criteria we are proposing are based on the expert advice we have received and clinical data available, similar to the criteria for nusinersen,” says Ms Williams.

The consultation is available on the Pharmac website for anyone wanting to have their say. “Consultation is a very important step in our process,” says Ms Williams. “It’s how we check that the people who will get the most benefit from the medicines will be able to access them.

“As well as seeking feedback on the funding of risdiplam, we are also interested in feedback on how community distribution of risdiplam could be managed. If the decision is approved, we will be working closely with the supplier, Roche and our colleagues across the health sector to plan for the implementation of risdiplam

If approved, funding would start from 1 May 2023.

Learn more

SMA is a disorder that spans a wide spectrum of severity. There are different types of SMA, such as type I, II or IIIa. These types of SMA are generally diagnosed when symptoms of SMA occur before three years of age. SMA affects the control of muscle movement and people with different types of SMA generally experience different levels of symptom severity.

In severe cases spinal muscular atrophy can impact a baby’s ability to swallow, hold their head up, sit and roll over. Sadly, infants with the most severe types of the condition often pass away in their first few months or years of life. In later onset spinal muscular atrophy, such as type IIIa, people can experience significant muscle weakness and disability, with some people losing the ability to walk over time. 

Future eligibility criteria considerations

Pharmac has recently received an application to fund risdiplam for the treatment of pre-symptomatic SMA which will be considered by the Rare Disorders Advisory Committee in March 2023. We have agreed with Roche that we would widen access to risdiplam to include pre-symptomatic use if risdiplam is granted Medsafe approval for this use, and we receive a positive funding recommendation from our clinical advisors.

Pharmac also intends to seek further advice and assess the use of both risdiplam and nusinersen in wider populations (adults and additional subtypes) in 2023.