Roche has announced that the U.S. Food and Drug Administration (FDA) has approved a new tablet formulation of Evrysdi (risdiplam), making it the first and only oral tablet treatment for spinal muscular atrophy (SMA).
This approval introduces a non-invasive, disease-modifying therapy for individuals affected by SMA, expanding treatment options. The newly approved 5 mg Evrysdi tablet can be taken whole or dissolved in water for ease of administration.
Convenient Storage: The tablet formulation can be stored at room temperature, eliminating the need for refrigeration.
Equivalent Efficacy and Safety: Clinical studies have confirmed that the tablet provides the same effectiveness and safety profile as the already available oral solution.
Simplified Dosing: Patients now have an alternative to the liquid form, potentially improving convenience and independence for those undergoing treatment.
Approval Based on Bioequivalence Study
The FDA’s approval was supported by a bioequivalence study, demonstrating that the 5 mg tablet—whether swallowed whole or dissolved in non-chlorinated water—delivers risdiplam at levels comparable to the existing liquid solution. As a result, patients switching to the tablet can expect the same therapeutic outcomes.
Updated Prescribing Information
As part of this approval, prescribing guidelines now include specific instructions on how to take and store the tablet. The new formulation is approved for individuals aged two years and older who weigh at least 20 kg (44 lbs). The tablet is expected to be available for patients in the coming weeks.
Roche leads the clinical development of Evrysdi in collaboration with the SMA Foundation and PTC Therapeutics, advancing research to improve treatment options for SMA patients globally.
About Evrysdi (Risdiplam)
Evrysdi is a treatment designed to enhance the production of survival motor neuron (SMN) protein by modifying the splicing of SMN2 pre-mRNA. It is specifically developed for individuals with SMA caused by mutations in chromosome 5q, which result in a deficiency of the SMN protein. This protein is crucial for maintaining motor neuron function and overall muscle health.
Evrysdi is taken once daily, either as a liquid administered orally or via a feeding tube or in its new tablet form, which can be swallowed whole or dissolved in water. The medication works by increasing and maintaining SMN protein levels in both the central nervous system and peripheral tissues, supporting motor function and overall mobility.
Regulatory and Industry Recognition
Since its development, Evrysdi has received multiple designations and accolades:
PRIME designation from the European Medicines Agency (EMA) in 2018.
Orphan Drug Designation by the FDA in 2017.
Drug Discovery of the Year (2021) by the British Pharmacological Society.
Society for Medicines Research Award for Drug Discovery (2021).
Currently, Evrysdi is approved in over 100 countries, with more than 16,000 individuals with SMA having received the treatment worldwide.
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Ongoing and Completed Clinical Trials
Evrysdi’s effectiveness has been assessed through various global clinical trials, including:
FIREFISH (NCT02913482): A study evaluating Evrysdi in infants with Type 1 SMA, showing positive results after five years of follow-up.
SUNFISH (NCT02908685): A placebo-controlled study for individuals aged 2 to 25 years with Type 2 or 3 SMA, which met its primary endpoint.
JEWELFISH (NCT03032172): A study examining the safety and pharmacokinetics of Evrysdi in patients previously treated with other SMA therapies.
RAINBOWFISH (NCT03779334): A trial evaluating Evrysdi in newborns with a genetic diagnosis of SMA but no symptoms at the start of treatment.
MANATEE (NCT05115110): An ongoing study investigating the combination of Evrysdi with GYM329, an anti-myostatin molecule aimed at enhancing muscle growth in children with SMA.
HINALEA 1 & 2 (NCT05861986, NCT05861999): Phase IV studies assessing the effectiveness of Evrysdi in young children who received gene therapy for SMA.
PUPFISH (NCT05808764): A Phase II trial analyzing Evrysdi’s pharmacokinetics and safety in infants under 20 days old diagnosed with SMA.
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Understanding Spinal Muscular Atrophy (SMA)
SMA is a progressive neuromuscular disorder that severely impacts muscle function, often leading to fatal outcomes if untreated. It is caused by mutations in the SMN1 gene, which result in insufficient SMN protein production. This deficiency affects motor neurons, leading to muscle weakness and atrophy over time.
Approximately 1 in 10,000 infants are affected by SMA, making it the leading genetic cause of infant mortality. Depending on the severity of the condition, individuals may experience difficulties with movement, eating, and even breathing. Treatments such as Evrysdi aim to increase SMN protein levels, helping to preserve motor function and improve quality of life for those affected by SMA.
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