SMA drug Zolgensma finally makes Alberta's Rare Diseases Drug Coverage Program

Coverage for Zolgensma, a gene therapy used to treat spinal muscular atrophy, is now permanently available in Alberta.

The therapy was added to the province's Rare Diseases Drug Coverage Program after the pan-Canadian Pharmaceutical Alliance (pCPA) reached a long-term pricing agreement with manufacturer Novartis Pharmaceuticals Canada Inc. in October.

Alberta Health Minister Jason Copping said in a statement the government was "extremely pleased" at the development and Alberta's new ability to offer the treatment on a more secure basis.

The province had been providing access to the drug on its own pending a national drug review, approval and the price negotiations with Novartis.

Zolgensma, a one-time treatment with a price tag of $2.91 million, is administered by intravenous infusion to replace a missing or faulty gene that results in spinal muscular atrophy (SMA). The rare disorder can cause a loss of muscle function and is seen in about one in 10,000 Canadian babies each year, according to the National Organization for Rare Disorders.

Zolgensma is supposed to stop the disease's progression and restore protein production of the missing or defective SMN1 gene. It must be administered when a patient is a toddler.

Several Alberta families led the push to have it approved and covered in Alberta and Canada.

Spinraza, a spinal injection given three times a year that can be used as a treatment for SMA, is also covered under the Alberta Drug Benefit List.