FDA approves non-invasive risdiplam tablet for spinal muscular atrophy

FDA approves non-invasive risdiplam tablet for spinal muscular atrophy | Image Credit: © Calin - © Calin - stock.adobe.com.

Genentech has received FDA approval for the risdiplam (Evrysdi) tablet new drug application, indicated for those living with spinal muscular atrophy (SMA).

Risdiplam is the only non-invasive disease-modifying treatment for SMA, according to a Genentech press release. The newly-approved tablet, 5 mg, can be swallowed whole or dispersed in water.

Leaders at Genentech expect the new tablet, indicated for individuals aged 2 years and older who weigh more than 44 lbs (20 kgs), to be available in the "coming weeks."

Approval of the tablet was based on data from a bioequivalence study that demonstrated the 5 mg tablet and original oral solution provided comparable exposure to risiplam, whether the tablet was swallowed whole or dispersed in non-chlorinated drinking water.

"[Risdiplam] 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," stated Levi Garraway, MD, PhD, chief medical officer and head of Global Product Development, Genentech, in a press release.

"The [risdiplam] tablet combines established efficacy with convenience, providing an additional flexible option for SMA management.”

Overall, risdiplam is designed to treat SMA by increasing and sustaining SMN protein production in the central nervous system (CNS) and peripheral tissues. SMN is crucial for maintaining healthy motor neurons and core functions.

Risdiplam is being, or has been, evaluated in several global multicenter trials among individuals with SMA.

In the FIREFISH (NCT02913482) open-label, 2-part clinical trial among infants with Type 1 SMA, infants were approximately 5.5 months of age (median) at the time of enrollment. Of the 58 infants that completed the first year of treatment, 52 entered an open-label extension study, which met primary endpoints and concluded after 5 years of follow-up.

The active JEWELFISH (NCT03032172) study, an open-label exploratory trial, is designed to assess safety, tolerability, pharmacokinetics, and pharmacodynamics among individuals with SMA aged 6 months to 60 years. Patients in this trial received other investigational or approved SMA therapies prior to receiving risdiplam.

A pair of phase 4 clinical studies, HINALEA 1 (NCT05861986) and HINALEA 2 (NCT05861999), are in the active recruiting phase to evaluate the effectiveness and safety of risdiplam in patients under 2 years of age at enrollment, who received onasemnogene abeparvovec gene therapy either pre-symptomatically or post-symptomatically, following a genetically confirmed diagnosis of 5q–autosomal recessive SMA.

The PUPFISH (NCT05808764) phase 2 open-label study is also actively recruiting babies with SMA who are under 20 days of age to investigate the pharmacokinetics and safety risdiplam in this population.

“We cannot underestimate the value that comes with simplifying treatment administration and disease management for people who are living with SMA or those caring for them,” said Kenneth Hobby, president of Cure SMA, in a statement.

“This new room temperature stable formulation option offers an additional choice that may more conveniently fit into daily living activities such as working, traveling, and education," said Hobby.

Reference:

FDA Approves Genentech’s Evrysdi Tablet as First and Only Tablet for Spinal Muscular Atrophy (SMA). Genentech. Press release. February 12, 2025. Accessed February 12, 2025. https://www.businesswire.com/news/home/20250210728175/en/FDA-Approves-Genentech%E2%80%99s-Evrysdi-Tablet-as-First-and-Only-Tablet-for-Spinal-Muscular-Atrophy-SMA