Pharmac to fund medicine for a rare genetic disorder for the first time in New Zealand

Pharmac to fund medicine for a rare genetic disorder for the first time in New Zealand. Photo / File.

A new medicine for those with a rare genetic disorder, and a chemotherapy drug used to slow or stop the growth of cancer cells are set to be publicly funded for the first time in New Zealand - while wider access to a meningococcal vaccine will also be ensured.

Only days after reaching an agreement with medicine supplier Vertex to fund the “life-extending” Trikafta for those with cystic fibrosis, Te Pātaka Whaioranga Pharmac has today announced it has approved funding for nusinersen (Spinraza) - the first medicine for spinal muscular atrophy (SMA) to be funded in New Zealand.

SMA affects the control of muscle movement which spans a wide spectrum of severity. It impacts infants through to adults and can cause disability and early death. People with later-onset SMA, such as type IIIa, may endure severe muscle weakness and impairment, with some individuals eventually losing their ability to walk.

Those aged 18 years and under who have pre-symptomatic, or symptomatic type I, II or IIIa spinal muscular atrophy, and meet certain criteria will be eligible for the treatment. Funding will begin on January 1 next year.

Pharmac’s Director of Operations Lisa Williams estimates 30 to 50 young people will be eligible for funded treatment.

“We are really pleased to announce that this medicine will be funded for New Zealanders,” Williams said.

“It will make a substantial difference to people’s lives.”

Health Minister Andrew Little said this has the potential to make a huge difference in the lives of the young people who receive it.

“Coming on top of Pharmac’s announcement on Sunday that it proposes funding Trikafta for people with cystic fibrosis, this is real evidence that the extra money we’re putting into the health system and into medicines is paying real dividends for people with rare diseases,” Little said.

Health Minister Andrew Little said this has the potential to make a huge difference in the lives of the young people who receive it. Photo / File

An oral version of the cancer cell shrinking chemotherapy drug, Vinorelbine, will also now be funded by the drug-buying agency.

This will make life easier for hundreds of cancer patients, Little said, as it’s a capsule you can take at home that you can take at home instead of having to travel to a hospital for intravenous chemotherapy.

“Vinorelbine is used to treat lung and breast cancer, as well as some non-cancerous growths, so making it available as an oral treatment will be a real benefit to rural people especially, who often find it difficult to get to places where infusion services are delivered,” Little said.

Pharmac also confirmed it will extend free access to the meningococcal B vaccine to infants and young people residing in close quarters with other people, such as hostels.

Catch-up programmes for children under five years and rangatahi already living in close-living situations will also be funded.

“Meningococcal disease has an inequitable impact on Māori and Pacific children, so we will work with Te Whatu Ora to support their implementation of the programmes to benefit Māori and Pacific people,” Williams said.

“We’re grateful for all of the feedback we received, and we and our sector partners will use this information to support an effective rollout of the meningococcal B vaccine in early 2023.”

On Sunday, Pharmac confirmed plans to fund Trikafta for an estimated 540 persons in New Zealand who have cystic fibrosis, a disorder that is ultimately fatal. According to experts, a person with cystic fibrosis has a mid-to-late 30s life expectancy on average.

Trikafta treats its underlying cause and has previously been described as a “miracle drug” by cystic fibrosis sufferers and their families.