FDA approves new tablet version of oral SMA treatment Evrysdi
New formulation designed to make treatment easier, more convenient
The U.S. Food and Drug Administration (FDA) has approved a new tablet formulation of Evrysdi (risdiplam), an approved oral treatment for spinal muscular atrophy (SMA).
“Evrysdi has robust potential to modify the SMA disease trajectory, and has already been used to treat thousands of patients to date,” Levi Garraway, MD, PhD, chief medical officer and head of global product development at Genentech, said in a company press release. “This approval marks another significant step forward.”
Genentech leads Evrysdi’s development in collaboration with the SMA Foundation and PTC Therapeutics.
The new tablet is available at a dose of 5 mg, which is the recommended dosage for patients ages 2 and older who weigh at least 20 kg (about 44 pounds). The tablet can be swallowed whole or dissolved in water.
“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. “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.”
New formulation provides ‘flexible option’
The chief cause of SMA is mutations in the gene SMN1, which provides instructions to make a protein called SMN. Lack of the SMN protein causes motor neurons, the nerves that control movement, to sicken and die, ultimately leading to SMA symptoms.
A different gene, SMN2, is able to produce SMN protein as well, but due to a quirk of the genetic sequence it is usually much less active than SMN1. Evrysdi works to boost the activity of SMN2 to facilitate production of more SMN protein, which has been proven to help slow the progression of SMA.
Evrysdi was approved by the FDA in 2020 as to treat SMA patients ages 2 months and older. The agency extended the approval in 2022 to also cover babies younger than 2 months.
The originally approved version of the drug is an oral solution, which remains the only version available for children younger than 2 and SMA patients who take the medication via feeding tube. The new tablet formulation may offer more convenient dosing for older SMA patients who don’t use a feeding tube, according to Genentech.
“The Evrysdi tablet combines established efficacy with convenience, providing an additional flexible option for SMA management,” Garraway said.
The FDA’s approval of the new tablet was based on bioequivalence data showing that the tablet delivered the same amount of the drug’s active ingredient to the body as the older version, which according to Genentech suggests that the two formulations should be identical in terms of their effects.
According to its updated FDA label, the Evrysdi tablet can either be swallowed whole or can be dissolved in a teaspoon of room-temperature, non-chlorinated drinking water. It should not be dissolved in any other liquid.
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