Genentech, a member of the Roche Group, announced through a press release on February 12, 2025, that the U.S. Food and Drug Administration (FDA) has approved a new tablet formulation of Evrysdi® (risdiplam) as a noninvasive, disease-modifying treatment for spinal muscular atrophy (SMA). This approval expands patient access, offering greater convenience and flexibility in disease management.
Evrysdi is the only disease treatment for SMA available worldwide. The treatment is available in more than 100 countries and has benefited approximately 16,000 people diagnosed with SMA. The newly approved 5 mg tablet allows the patients to take it either in a whole form or to disperse it in non-chlorinated drinking water while maintaining the same efficacy and safety as the oral solution.
According to Levi Garraway, M.D., Ph.D., Chief Medical Officer and Head of Global Product Development at Genentech, data suggest that Evrysdi demonstrates strong potential to change the course of SMA and support treatment for thousands of patients. The tablet approval represents major progress in SMA treatment because it offers flexible new options for medication delivery.
The FDA approved the Evrysdi tablet through a bioequivalence study that demonstrated equal drug exposure when patients took either whole 5 mg tablets or dispersed tablets in water compared to the existing oral solution. The oral solution continues to be accessible to patients who need different dosing options or prefer liquid form.
Evrysdi tablet users now have the advantage of storing their medication under room temperature conditions, which enhances the portability for patients on the go. Kenneth Hobby from Cure SMA explained that this advancement significantly benefits patients by reducing the complexity of treatment.
The prescribed tablet form is available for individuals who are two years old or older with a weight minimum of 44 lbs (20 kgs). Patient access to the new formulation will become available during the following weeks.
Evrysdi is an approved SMA treatment because it functions as a survival motor neuron 2 (SMN2) pre-mRNA splicing modifier to help patients with this severe neuromuscular condition, which results from chromosome 5q mutations and deficient SMN protein levels. When motor neurons lack adequate SMN protein, they degenerate, which leads to progressive muscle weakness that can eventually impact swallowing and breathing.
Through sustained delivery of SMN protein throughout the body, Evrysdi functions to protect healthy motor neurons and enhance muscle functioning. The treatment requires daily administration through either liquid form by mouth or a feeding tube or tablet form taken by mouth.
Ongoing Research and Clinical Trials
Evrysdi undergoes multiple worldwide clinical trials at present.
The FIREFISH research examined that the Type 1 SMA infants demonstrated positive results during five years of study. SUNFISH studied the individuals between ages 2 and 25 who had Types 2 or 3 SMA and met its main study objective. JEWELFISH has tested the safety measures and pharmacodynamics functions along with the drug distribution patterns of patients who received other SMA treatments.
The RAINBOWFISH study demonstrated its main objective while evaluating therapy safety and performance in infants before the onset of symptoms below six weeks of age. The MANATEE clinical trial combines Evrysdi with the muscle therapy GYM329 as an ongoing Phase II/III trial for children aged between 2 and 10 years. The HINALEA 1 and 2 under Phase IV trials continue as these trials examine how Evrysdi affects young children who had received gene therapy previously.
The PUPFISH research team conducts Phase II assessments on newborns under 20 days of age to determine Evrysdi’s pharmacologic behavior and safety characteristics. It actively searches for new participants in its current recruitment cycle.
The most common side effects from Evrysdi treatment include fever, diarrhea, rash, and upper respiratory tract infections. The assessment of drug interactions alongside staff consultation with patients should be a standard practice for doctors. The Evrysdi tablet approval from Genentech enhances the treatment accessibility for patients with SMA. The new tablet provides patients with increased flexibility as it carries the same effectiveness as the oral solution. SMA treatment has advanced through development, which allows improved disease management and enhanced patient quality of life.
References: Genentech. FDA Approves Genentech’s Evrysdi Tablet as First and Only Tablet for Spinal Muscular Atrophy (SMA). Published February 12, 2025. Accessed February 14, 2025. https://www.gene.com/media/press-releases/15052/2025-02-12/fda-approves-genentechs-evrysdi-tablet-a
