New form of SMA treatment Evrysdi may give patients ‘freedom’
A tablet formulation of Evrysdi (risdiplam) for spinal muscular atrophy (SMA) recently approved in the U.S. is designed to give patients a new option, a Genentech official said.
“Evrysdi is the only oral, non-invasive disease-modifying SMA treatment, and the new tablet formulation may provide greater freedom and independence for people with SMA, thanks to simplified dose administration,” said Gregory Rippon, MD, vice president and chief medical partner of neurology, ophthalmology, and internal medicine at Genentech, the company that markets Evrysdi.
The innovation represents “Genentech’s commitment to support the SMA community by developing formulations that bring more flexibility and simplicity into everyday management of this rare, progressive disease,” Rippon said in a written Q&A with SMA News Today.
The tablet is authorized for people with SMA ages 2 and older who weigh at least 44 pounds (20 kg). The tablets may be swallowed whole or dissolved in a small amount of water that can then be swallowed. They should not be administered via feeding tube.
The new formulation “is intended for patients who are able to swallow a tablet without difficulty or who would prefer to disperse the tablet in drinking water instead of taking the oral solution,” Rippon said, noting that the decision of whether to take the tablet whole or dissolved in water “should be discussed with the treating doctor and care team.”
Simplifying SMA treatment
The tablet contains 5 mg of the active ingredient, the recommended daily dosage for SMA patients ages 2 and older who weigh at least 20 kg. T7he tablets do not need to be stored under refrigeration.
“The new tablet is room temperature stable and does not require dose measurement, which may simplify and provide more administration options for people living with SMA,” Rippon said.
Evrysdi was developed as an oral solution that requires refrigeration for long-term storage. The oral solution has been approved as an SMA treatment since 2020. The original approval covered the therapy’s use in patients ages 2 months and older, and was extended in 2022 to cover patients of all ages. The therapy works to boost levels of the SMN protein. SMA is mainly caused by mutations that lead to irregularly low levels of this protein.
Rippon said the original version of Evrysdi “will remain available for those who may prefer it, those who have swallowing difficulties and those who require administration through a feeding (nasogastric/gastrostomy) tube.”
“Anyone considering switching from oral solution Evrysdi to the tablet should have a discussion with their treating doctor and care team,” he added.
Clinical trials have shown that Evrysdi can help patients retain or improve motor function. Approval of the new tablet version was based on data showing that it delivered the same amount of drug to the body as the oral solution formulation.
Evrysdi is approved in more than 100 countries, and according to Rippon, more than 18,000 people with SMA have received the treatment.
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