The patient advocacy group, Cure SMA, announced that Quebec’s Institut national d’excellence en santé et en services sociaux (INESSS) does not recommend Spinraza (nusinersen) for any patients with spinal muscular atrophy (SMA).

SMA is a neurodegenerative disorder and children with the most severe form of SMA rarely living past a few years. However, some children may not begin to show signs of the disease till they are older infants. Spinraza is an antisense oligonucleotide designed to treat SMA caused by mutations or deletions in the SMN1 gene located in chromosome 5q that leads to a deficiency in survival motor neuron (SMN) protein. The drug is approved by Health Canada for all SMA patients with a 5q deletion and that approval was largely based on a clinical trial involving infants with SMA, the most severe form of the condition.

On Friday, the makers of Spinraza, Biogen, also shared their disappointment in the news but their news release did imply that Spinraza is recommended for infants with SMA. In the news release, Marina Vasiliou, Managing Director of Biogen Canada said, “Biogen believes that the benefits of Spinraza are clear for patients across the spectrum of the disease and that this recommendation clearly limits the hope many of them have for coping with their SMA.”

Vasiliou added, “We want to have an important and substantive dialog with the Quebec provincial drug formulary officials and health care providers about the demonstrated value of Spinraza for patients with SMA and their families and the importance of early treatment.  We are hopeful that this can be done in a manner that does not further delay patients’ abilities to access the drug”.

Outside of Quebec, other Canadian SMA patients are continuing to wait for Canadian Agency for Drugs and Technologies in Health (CADTH) to publish their recommendations for the drug.

The drug was approved by the U.S. Food and Drug Administration (FDA) in 2016.

For more information about SMA, visit Cure SMA.