FDA approves new tablet version of Roche’s Evrysdi for spinal muscular atrophy

USA – The FDA has approved a tablet version of Evrysdi (risdiplam), a treatment for spinal muscular atrophy (SMA) developed by Roche.

This new formulation provides an easier way for patients to take the medication compared to other SMA treatments like Biogen’s Spinraza (nusinersen), which requires intrathecal administration, and Novartis’ Zolgensma (onasemnogene abeparvovec), a one-time gene therapy.

Making treatment more convenient

Unlike the current oral liquid form of Evrysdi, which must be refrigerated, the new tablet formulation offers a more convenient option.

The tablets can be stored at room temperature and either swallowed whole or dissolved in water, making them easier to use for both patients and caregivers.

According to Roche, this change aims to “provide greater freedom and independence for people with SMA thanks to a simplified dose administration.”

Kenneth Hobby, president of the non-profit Cure SMA, emphasized the importance of treatment convenience for those living with the disease.

“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,” he stated.

He added that the new formulation could make it easier for patients to manage their medication while working, traveling, or attending school.

Strong market performance for Evrysdi

Despite being the last SMA treatment to enter the market, Evrysdi has become the top-selling option in its category.

Early 2024 revenue reports showed that patients prefer simpler treatment options, helping Roche’s small-molecule drug surpass its competitors in sales. The new tablet version is expected to further strengthen its market position.

Who can use the new Evrysdi tablets?

The tablet version has been proven to be bioequivalent to the oral solution and will be available in the coming weeks. It is approved for patients aged two and older who weigh more than 44 lbs.

A step forward in SMA treatment

The approval of Evrysdi tablets represents a significant step forward in making SMA treatment more accessible and convenient.

With its room-temperature stability and easy administration, the new formulation is expected to improve the daily lives of both patients and caregivers.