Ontario SMA Patients Granted Wider Access to Spinraza

Ontario has expanded publicly funded access to Spinraza (nusinersen) to cover spinal muscular atrophy (SMA) types 2 and 3.

The decision specifically covers presymptomatic patients with two or three copies of the SMN2 gene; patients with SMA for less than six months, two SMN2 copies, and symptom onset between the first week and 7 months of age; and those younger than 18 years with symptom onset after 6 six months of age and who have never been able to walk independently.

Other type 2 and 3 patients who may or may not have achieved the ability to walk independently are also encouraged to apply through their clinicians and will be considered on a case-by-case basis. Adults may also be covered exceptionally on a case-by-case basis.

This announcement follows Ontario’s government decision in November 2018 to provide publicly funded access for patients living with SMA type 1, the most common and severe form of the disease.

Canadian provinces Saskatchewan and Quebec, Non-Insured Health Benefits — which provides medical coverage to First Nations and Inuit people in Canada — and more than 44 countries worldwide had already granted broad public funding to Spinraza.

“We welcome the decision by the Ontario government to grant broader access to Spinraza,” Marina Vasiliou, Biogen Canada’s vice president and managing director, said in a press release. “We believe that all SMA patients, including adults, should have broad access and we will continue to work with all jurisdictions until this is achieved in Canada.”

Health Canada originally approved treatment with Spinraza in June 2017 covering presymptomatic patients and those with SMA types 1–3 regardless of age. Even though the U.S. Food and Drug Administration recently approved the SMA gene therapy Zolgensma (onasemnogene abeparvovec-xioi), Spinraza remains the only available option for Canadians with this disease.

“This is very good news,” said Jiri Vajsar, a pediatric neurologist at The Hospital for Sick Children in Toronto. Broad access to Spinraza is “critical” and gives patients and families hope to slow the natural progression of SMA and possibly improve motor function, strength, and independence.

“This is ultimately what we aspire to achieve for all SMA patients across the spectrum of the disease,” he added.

Craig Campbell, MD, a pediatric neurologist at the Children’s Hospital – London Health Sciences Centre in London, Ontario, said the expanded access to Spinraza is “an important, thoughtful and positive decision for the SMA community. It will allow a wide range of people with SMA to realize the substantial benefits of this medication.”

“This allows us to make sure nusinersen (Spinraza) gets to those who need it now, and collectively document the long-term real world data to further demonstrate [its] positive impact,” he said. 

Aaron Izenberg, MD, a neurologist at the Sunnybrook Health Sciences Centre in Toronto said he is “pleased with the Ontario government’s decision to include access for adults on a case by case basis,” while Susi Vander Wyk, Cure SMA Canada’s executive director, added: “We have been hoping this day would come and are thrilled that the Ontario government has recognized the need for Spinraza to be accessible more broadly.”

Wyk also urged all other Canadian jurisdictions to follow suit to make sure Spinraza is accessible “to all patients across Canada who can benefit.”

Barbara Stead-Coyle, Muscular Dystrophy Canada’s CEO, applauded the decision and noted the continuing advocacy for expanded access, “as the evidence supporting Spinraza shows that treatment can have a meaningful impact across all forms of the disease.”