Federal Health Minister Greg Hunt

Nusinersen (Spinraza) will become available on the PBS from 1 June for the treatment of infantile and childhood-onset spinal muscular atrophy (SMA) in patients 18 years or under at initiation of treatment.

However relief for affected families kicks in before then, according to Federal Health Minister Greg Hunt.

“I want to thank Biogen for immediately opening up compassionate access to Spinraza from 1 May for Type 1, Type 2 and Type 3a SMA, ahead of the 1 June listing on the PBS,” he said in a statement.

Families would have to pay more than $367,850 a year for the medicine without PBS listing.

“This listing will now mean they only pay a maximum of $39.50 per script, with concessional patients paying just $6.40.”

An initial application to list nusinersen on the PBS was rejected in November 2017 ‘…because of uncertainty about its clinical effectiveness in terms of the extent and durability of response across the spectrum of SMA for which the submission has sought subsidy’.

However the Committee acknowledged there was a high and urgent clinical need for treatments for SMA and especially for the most severe forms of the condition.

“The PBAC considered that the available evidence suggests all patients may receive some benefit from nusinersen, but that this benefit needs to be better quantified,” the PBAC Meeting Summary said.

The March 2018 PBAC meeting found new information on the cost-effectiveness of the treatment was more compelling.

The cost to the government is estimated at $241 million.