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First Oral SMA Treatment Gets EMA Panel Thumbs Up

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended marketing authorization for risdiplam (Evrysdi), the first treatment that can be given orally to patients with certain types of spinal muscular atrophy (SMA).

Risdiplam is “expected to increase survival of motor neurons and reduce symptoms of the disease,” the EMA said in a statement.

SMA is a rare and often fatal genetic disease that causes muscle weakness and progressive loss of movement.

Risdiplam is indicated for the treatment of 5q SMA in patients aged 2 months and older who have a clinical diagnosis of type 1, 2, or 3 SMA or who have one to four SMN2 copies.

The recommendation is based on two studies; one investigated the effects of risdiplam in patients with infantile-onset SMA, and the other involved patients with later-onset SMA.

“The results from the trials show beneficial effects in very young patients in terms of their motor development and survival at 12 months, compared to data on the natural course of the disease in these patients,” the EMA said.

“The positive effect in later-onset SMA (Type 2 and 3) has been demonstrated in a double-blind placebo-controlled trial, including patients between 2 and 25 years of age,” the agency noted.

The main adverse reactions observed in trials were headaches; mouth ulcerations; aphthous ulcers; urinary tract infections, including cystitis; arthralgia; nausea; pyrexia; and dizziness/vertigo.

The EMA reviewed the marketing application for risdiplam under an accelerated timetable to allow patients faster access to the medicine.

As part of its recommendation, the committee has asked the company to conduct a postauthorization observational efficacy study to further evaluate disease progression in SMA patients (both presymptomatic and symptomatic) with one to four SMN2 copies treated with risdiplam, in comparison to natural history data in untreated patients.

The CHMP positive opinion will now be sent to the European Commission for the adoption of a decision on an EU-wide marketing authorization.

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  • Posted on March 1, 2021