British Columbia to fund life-changing spinal muscular atrophy treatment

Victoria Tuesday, October 2, 2018 11:30 AM

Babies living with spinal muscular atrophy (SMA), a rare, severe and degenerative neuromuscular disease, soon will have access to Spinraza, a life-changing treatment.

In line with the recommendation made by the independent Common Drug Review (CDR), starting in November, British Columbia will provide coverage for Spinraza for infants with an onset of SMA symptoms at or before seven months of age. In addition, through the efforts of the Province as part of the pan-Canadian Pharmaceutical Alliance, other SMA patients can receive this drug from the manufacturer while the CDR examines additional clinical evidence that may support expanded coverage.

Spinraza is the first and only available treatment that is shown to slow or even reverse the effects of SMA. SMA destroys motor neurons in the spinal cord, leading to paralysis and death in the most severely affected patients. Worldwide, it is the leading genetic cause of infant death.

Spinraza received a positive CDR recommendation based on the submitted clinical evidence in November 2017. The recommendation was conditional upon a substantial price reduction and limited to the type 1 SMA patient group (babies with an onset of signs and symptoms at or before seven months of age). British Columbia, as part of the pan-Canadian Pharmaceutical Alliance (pCPA), subsequently entered into pricing negotiations with Spinraza’s manufacturer, Biogen.

The CDR is reviewing data resubmitted by Biogen for Spinraza coverage for other SMA patient groups, including types 2 and 3. B.C. supports this resubmission. Biogen will provide Spinraza to eligible SMA type 2 and 3 patients through its compassionate access program, called SMA360, during this review. It is anticipated that the CDR will complete its evaluation in January 2019.

When released in 2019, the final CDR recommendation on the resubmission will inform whether B.C. and other participating pCPA jurisdictions will fund Spinraza for patients with other types of SMA.

Quick Facts:

  • Individuals born with SMA do not make enough of the survival motor neuron protein, which is key to motor development. Spinraza is injected into the spinal fluid and boosts production of the gene responsible for protein development.
  • SMA patients require Spinraza (nusinersen) treatment for the duration of their lives. The dosing schedule is six doses in the first year and three doses in subsequent years. Each drug injection is usually given in a hospital setting.
  • There are three main types of SMA:
    • Type 1: patients show signs and symptoms at or before seven months of age and cannot sit unsupported. These patients typically have a life expectancy of two years of age. Type 1 patients represent approximately 60% of diagnosed cases.
    • Type 2: patients show signs and symptoms between seven months and 18 months of age. Although most can sit unsupported, they usually cannot walk independently. Life expectancy is over 25 years of age and is greatly improved by aggressive supportive care.
    • Type 3: patients may show signs and symptoms between 18 months and 18 years. Patients can walk independently, however, continued declining mobility may result in the use of a wheelchair. Type 3 patients typically have a normal life expectancy and represent roughly 10% to 20% of cases.
  • Approximately one in 40 people carry the SMA gene. If both parents are carriers, there is a one-in-four probability that the SMA gene will be passed to children.
  • While SMA primarily affects infants, it can also affect older children and adults.
  • The list cost of Spinraza, as set by Biogen, is substantial at approximately $708,000 per patient for the first year and roughly $354,000 every year following. The pCPA and Biogen recently completed pricing negotiations for Spinraza after opening them in February 2018 for type 1 patients.

Learn More:

For more information about B.C.’s PharmaCare program:

To learn more about SMA360, Biogen’s SMA support service, visit:

This is on BC Govt website go to this link click here


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