FDA approves Evrysdi tablets for spinal muscular atrophy

Key takeaways:

• Evrysdi is the only FDA-approved, noninvasive, disease-modifying treatment for spinal muscular atrophy.
• The tablets offer similar safety and efficacy as the original oral solution.

The FDA has approved Evrysdi in tablet form for patients with spinal muscular atrophy aged 2 years and older who weigh more than 44 pounds, according to the manufacturer.

Evrysdi (risdiplam, Genentech), a survival motor neuron 2 pre-mRNA splicing modifier, will be available in a 5 mg tablet that can be either swallowed whole or dissolved in water, the release said.

The FDA approval was based on results of a bioequivalence study that demonstrated the 5 mg tablet possessed similar safety and efficacy as the drug in its original oral solution.

Genentech said the oral solution will remain available for individuals who prefer it, and for patients prescribed other doses of the medication.

As part of the label extension, Evrysdi’s prescribing information has been updated to include guidance on tablet administration and storage, the company said in the release.

Evrysdi was granted orphan drug designation by the FDA in 2017, as well as PRIME designation by the European Medicines Agency in 2018.

“Evrysdi has robust potential to modify the SMA disease trajectory and has already been used to treat thousands of patients to date. This approval marks another significant step forward,” Levi Garraway, MD, PhD, Genentech’s chief medical officer and head of global product development, said in the release. “The Evrysdi tablet combines established efficacy with convenience, providing an additional flexible option for SMA management.”