Roche’s SMA treatment Evrysdi demonstrates continued improvements in infants

Pharmaceutical News

Roche’s spinal muscular atrophy (SMA) treatment Evrysdi has continued to demonstrated improvements in motor function and survival in babies, according to new long-term data.

Two-year data from part 2 of the FIREFISH study, evaluating Evrysdi (risdiplam) in infants aged one to seven months at enrolment with Type 1 SMA, found that the drug continued to improve motor function between 12 and 24 months.

This included the ability to sit without support for at least five seconds, with 61% in the Evrysdi arm hitting this goal versus 29% in the placebo group.

In addition, the study found that Evrysdi continued to improve survival in these patients, as well as the ability to feed orally and reduce the need for permanent ventilation.

Specifically, 93% of infants were alive after 24 months of treatment with Evrysdi, and 83% were alive and free from permanent ventilation at the same timepoint.

Further exploratory data suggested that Roche’s drug also continued to improve patients’ ability to swallow and reduce hospitalisation compared to the natural course of Type 1 SMA.

“[This] data highlight[s] the real-world impact of this transformative medicine in babies with the most severe form of SMA. For example, all infants alive after 24 months of treatment were able to swallow which can help them to feed orally rather than through a tube,” said Levi Garraway, chief medical officer and head of global product development at Roche.

“These results increase our understanding of how this first-of-its-kind treatment can extend the lives of babies with Type 1 SMA, providing much needed hope for their families,” he added.

In March, the European Commission (EC) approved Evrysdi for use in in patients aged two months and older with 5q SMA, with a clinical diagnosis of Type 1, Type 2 or Type 3 SMA with one to four SMN2 copies.

Roche said that it is working with reimbursement and assessment bodies across Europe to ensure ‘broad and rapid access to patients in need’.

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