Evrysdi Approved in Brazil for SMA, But No Indications Specified

Evrysdi Approved in Brazil for SMA, But No Indications Specified
1
(1)

Evrysdi (risdiplam) has been approved for the treatment of spinal muscular atrophy (SMA) in Brazil by the country’s National Health Surveillance Agency, known as ANVISA.

The Brazilian approval came within seven months of Roche’s original submission, with the country becoming the second to approve the medicine, following the U.S. While the precise indications for which Evrysdi has been approved in Brazil have not been specified, in the U.S. the therapy is currently approved to treat adults and children, ages 2 months and older, with all SMA types.

A request for approval also was recently filed with the Japanese Ministry of Health, Labour and Welfare (MHLW) by Chugai Pharmaceutical, part of the Roche Group. This filing in Japan triggered a $7.5 million milestone payment from Roche to PTC Therapeutics, one of the therapy’s co-developers.

“We are very pleased with both the rapid approval in Brazil as well as the filing of Evrysdi in Japan,” Stuart W. Peltz, PhD, CEO of PTC Therapeutics, said in a press release.

“These important achievements continue to build momentum for Evrysdi globally, and we are working hard alongside our partners in the collaboration to make the medicine available to SMA patients around the world,” Peltz said.

Evrysdi targets the underlying cause of spinal muscular atrophy by helping cells produce a fully functional version of the survival motor neuron (SMN) protein, which is crucial for muscle health and is missing in SMA patients. The therapy does so by boosting the ability of the SMN2 “backup gene” — a gene similar to SMN1 that also encodes the protein and normally remains unaffected in SMA patients — to produce the protein.

In the U.S., Evrysdi is now the third disease-modifying therapy to be approved for SMA, joining Spinraza (nusinersen) and Zolgensma (onasemnogene abeparvovec-xioi).

While Spinraza and Zolgensma are both delivered through injections — intravenously, or into the vein, in the case of Zolgensma, and directly into the spinal canal, for Spinraza — Evrysdi comes as a liquid that patients can either swallow or add to a feeding tube once daily.

This delivery method distributes Evrysdi throughout the body, where it acts on the motor neurons of the central nervous system (composed of the brain and spinal cord), as well as muscles and other tissues. It also allows patients to take the medicine at home, a key consideration during the ongoing COVID-19 pandemic.

“The strong efficacy, excellent safety profile, and convenient oral formulation of Evrysdi are so important to the SMA community. Access to a home administered therapy is particularly important during the COVID-19 pandemic,” Peltz said.

Two ongoing clinical trials — FIREFISH (NCT02913482) and SUNFISH (NCT02908685) — supported the applications filed in both the U.S. and Japan.

The FIREFISH trial is evaluating Evrysdi’s safety, tolerability, efficacy, and pharmacological properties in infants ages 1 to 7 months, with symptomatic type 1 SMA. The study already met its primary goal, with almost 30% of enrolled infants able to sit without support for five seconds after one year of treatment.

A number of infants in the trial have achieved other clinically meaningful milestones, including gaining upright head control and being able to roll from side to side. Two children also were able to stand without support.

SUNFISH is assessing the safety, tolerability, and efficacy of Evrysdi compared with a placebo in people with type 2 or type 3 SMA, ages 2 to 25. One-year data from SUNFISH showed Evrysdi significantly improved or stabilized motor abilities in patients who were unable to walk unaided.

An exploratory efficacy analysis of the study also showed that the therapy continued to lead to significant improvements in motor function in treated patients after two years, compared with data from untreated patients participating in natural history studies.

The Roche group, through its various members, is currently seeking broad global approval for Evrysdi. Beyond the U.S., Brazil, and Japan, applications have been filed in 15 other countries. Health authorities in CanadaChina, Switzerland, and Europe — where the application is under accelerated assessment — are already reviewing these approval requests.

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.
Total Posts: 53
Joana holds a BSc in Biology, a MSc in Evolutionary and Developmental Biology and a PhD in Biomedical Sciences from Universidade de Lisboa, Portugal. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells — cells that made up the lining of blood vessels — found in the umbilical cord of newborns.
×
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.
Latest Posts
  • lung function and SMA
  • SRK-015, muscle-directed therapy
  • Novartis Gene Therapies

How useful was this post?

Click on a star to rate it!

Average rating 1 / 5. Vote count: 1

No votes so far! Be the first to rate this post.

As you found this post useful...

Follow us on social media!

We are sorry that this post was not useful for you!

Let us improve this post!

Tell us how we can improve this post?