Pharmac is initiating a consultation today on a proposal to fund Evrysdi (risdiplam), a potential second treatment for young Kiwis living with the rare genetic disorder of spinal muscular atrophy (SMA).
SMA can vary widely in severity. It affects the control of muscle movement and, in severe cases, can be fatal to infants or have people experience muscle weakness and lose their ability to walk.
The drug Spinraza (nusinersen) was the first SMA treatment to be funded for under-18s who meet the eligibility criteria in New Zealand, beginning January 1 this year.
The major difference between the two drugs is that Spinraza is administered periodically by a spinal injection, while Evrysdi is taken daily as a more convenient oral liquid.
"Pharmac is thrilled to be taking the next step towards progressing a second treatment for those with spinal muscular atrophy for funding," director of operations Lisa Williams said.
"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.
"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," Williams said.
The consultation process comes as Pharmac reached a provisional agreement with Roche to supply Evrysdi.
"We're pleased patients, families and healthcare professionals may now have treatment choice for SMA, especially for those patients who find it hard to travel to hospitals or prefer to have an oral, at-home solution," Alex Muelhaupt, general manager of Roche New Zealand, said.
The consultation is now available on the Pharmac website, seeking feedback on the funding of Evrysdi and how the drug's community distribution could be managed.
"Consultation is a very important step in our process," Williams said. "It's how we check that the people who will get the most benefit from the medicines will be able to access them."
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