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Health Canada Approves Evrysdi for At-home Treatment

Health Canada Approves Evrysdi for At-home Treatment
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Evrysdi (risdiplam) has been approved by Health Canada for the at-home treatment of spinal muscular atrophy (SMA) in people ages 2 months and older.

“The Health Canada approval of Evrysdi is a welcome addition in our ability to treat SMA. It is an efficacious treatment that significantly increases survival motor neuron (SMN) protein in SMA patients,” Hugh McMillan, MD, a pediatric neurologist at the Children’s Hospital of Eastern Ontario, said in a press release.

“We now have an additional and convenient treatment option with Evrysdi, as it’s the first at-home and oral option for patients,” McMillan said.

Given as a flavored liquid, Evrysdi is administered daily at home by mouth or feeding tube. It is a small molecule that works by increasing the levels of SMN, a protein essential for motor neuron and muscle health. SMN production is impaired in people with SMA.

Data from two ongoing Phase 2/3 trials — FIREFISH (NCT02913482) and SUNFISH (NCT02908685) — led to the Health Canada decision. Together, these trials are evaluating Evrysdi’s safety and efficacy in nearly 300 individuals, ages 2 months to 25 years, with SMA types 1, 2, and 3. Among the 43 global SUNFISH study locations are sites in Quebec, Ontario, and Alberta.

Top-line results from SUNFISH were recently presented at the 2021 MDA Virtual Clinical and Scientific Conference, held online. The findings showed that Evrysdi significantly improved or stabilized motor function among children and young adults, ages 2 to 25, with SMA type 2 or 3. The improvements were either maintained or increased in the second year of treatment.

Two-year data from the FIREFISH trial, evaluating Evrysdi in infants ages 1–7 months with type 1 SMA, indicated that gains made in the first year were maintained in the second year.

For instance, 59% of the infants — 10 of 17 — could sit without support for five seconds in year two, as compared with 29% in the first year. A further 65% of the babies maintained upright head control, while 29% turned over on their own. A total of 30% could stand with either support or supporting weight.

The Health Canada decision follows others made in the U.S., Europe, Brazil, Chile, Ukraine, South Korea, Georgia, and Russia. More than 30 other countries continue to consider Evrysdi’s approval.

SMA patient advocates celebrated the decision.

“Cure SMA enthusiastically welcomes the arrival of Evrysdi to the Canadian market,” said Amy Loignon, co-president of Cure SMA Quebec.

“Despite the treatments for SMA currently available, a number of Canadians remain without treatment and a large percentage do not have access to existing ones due to their age, state of health, or due to the complexity of administering treatment in a hospital setting,” Loignon said.

According to Loignon, treatment administration has been “especially exacerbated by COVID-19 and the burden caused by it.”

“We are pleased to see this first oral treatment for SMA, giving these patients hope that their condition may stabilize and even improve,” Loignon added.

Forest Ray received his PhD in systems biology from Columbia University, where he developed tools to match drug side effects to other diseases. He has since worked as a journalist and science writer, covering topics from rare diseases to the intersection between environmental science and social justice. He currently lives in Long Beach, California.
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Ana holds a PhD in Immunology from the University of Lisbon and worked as a postdoctoral researcher at Instituto de Medicina Molecular (iMM) in Lisbon, Portugal. She graduated with a BSc in Genetics from the University of Newcastle and received a Masters in Biomolecular Archaeology from the University of Manchester, England. After leaving the lab to pursue a career in Science Communication, she served as the Director of Science Communication at iMM.

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Forest Ray received his PhD in systems biology from Columbia University, where he developed tools to match drug side effects to other diseases. He has since worked as a journalist and science writer, covering topics from rare diseases to the intersection between environmental science and social justice. He currently lives in Long Beach, California.
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