Beginning in April, all SMA patients using NHS Scotland may start to receive Spinraza injections. Clearing the path for routine availability, the Scottish Medicines Consortium (SMC) granted the treatment the Ultra Orphan Drug designation.
The designation permits the therapy to be reviewed in SMA types 2 and 3, and later onset disease. The medicine, known generically as nusinersen and marketed by Biogen, has been available to type 1 patients through NHS Scotland since May 2018.
The SMC’s new process, launched last fall, means that ultra-orphan treatments may be made available in Scotland for at least three years while further efficacy data are collected.
“Biogen welcomes the opportunity to work with the SMC to formalise the data collection and reimbursement arrangements. Subject to successful sign off, nusinersen is expected to be routinely available across types 1, 2, and 3 from April 2019,” the company said in a statement prepared on TreatSMA’s request.
Between now and April, “Biogen will continue to work with all stakeholders in Scotland to finalise the data collection required to aid the SMC’s future assessment of the value of nusinersen,” it added.
Nusinersen continues to be appraised elsewhere in the United Kingdom, with decisions expected this year.
“TreatSMA shares the excitement of all the Scottish families,” the group said. “We are continuing to fight for the Spinraza treatment to be available in England, Wales and Northern Ireland, the same way as it is available in Scotland and most of the developed world.”
Spinraza is the sole approved treatment for nearly all types of SMA in several countries, including the United States, across the European Union, Australia, Brazil, Japan, Switzerland, South Korea, Canada, and Chile. But regulatory approval for use does not necessarily mean coverage under a respective country’s public health system.
Affecting up to 1 in 10,000 people, SMA is caused by a mutation in the survival motor neuron gene 1 (SMN1). In a healthy person, this gene makes a protein called the survival motor neuron protein, which is critical to the function of nerves that control muscles. Without an adequate level of SMN protein, motor neurons in the spinal cord are lost, preventing muscles from receiving brain signals.
Survival motor neuron 2 (SMN2) is a closely related gene that’s not thought to be affected in SMA, but does not produce enough functional SMN protein on its own. By targeting the SMN2 gene, Spinraza has been shown to increase the production of full-length SMN protein.
Cost is a factor, however. The treatment carries a list price in the U.S. of $750,000 for the first year, when a higher dose is administered, and $350,000 yearly after that.
Britain’s independent, government-funded National Institute of Health and Care Excellence (NICE) recommended against Spinraza’s inclusion in its health system in August, arguing that its price in terms of Quality Adjusted Life Year (QALY) gained “is too high for it to be considered a cost-effective use of NHS [National Health Service] resources.”
Spinraza is administered through an intrathecal injection, or injection into the cerebrospinal fluid around the spinal cord, every four months throughout a patient’s life. This type of administration can present challenges to patients with spine deformities.
In related news, the U.S. Food and Drug Administration (FDA) has granted priority review to Novartis’ Biological License Application (BLA) asking that Zolgensma — previously called AVXS-101 — be approved as a gene therapy for infants with SMA type 1.