Roche’s Evrysdi becomes first pill Cleared for spinal muscular atrophy

Roche’s Evrysdi becomes first pill cleared for spinal muscular atrophy

The FDA on Wednesday approved the industry’s first-ever tablet treatment for spinal muscular atrophy—Roche’s SMN2 splicing modifier Evrysdi—offering patients a more convenient and flexible dosing option.

The approval, granted to Roche subsidiary Genentech, makes Evrysdi the only noninvasive disease-modifying therapy for spinal muscular atrophy (SMA), according to the company’s news release. The tablet, which can be taken whole orally or dissolved in water, will be available in the coming weeks, the company said, and can be given to patients aged 2 years and up who weigh over 20 kgs (44 lbs).

Genentech chief medical officer Levi Garraway called the approval a “significant step forward” for SMA patients. “Evrysdi has robust potential to modify the SMA disease trajectory, and has already been used to treat thousands of patients to date,” Garraway said, adding that the tablet formulation “combines established efficacy with convenience.”

Everysdi’s active ingredient is risdiplam, a small-molecule drug that promotes the production of full-length and functional SMN protein in the brain. SMN—also known as survival of motor neuron—is involved in repairing DNA, managing cellular stress and preserving overall neuromuscular function. In SMA, genes encoding for SMN are typically mutated, leading to dysfunctional protein expression.

The drug was first approved in 2020, but as an oral solution that must be constituted by a healthcare provider before being dispensed to patients, according to its label. The drug is administered once daily after a meal through an oral syringe.

Reference:
https://www.pharmalive.com/roches-evrysdi-becomes-first-pill-cleared-for-spinal-muscular-atrophy/

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