Evrysdi

risdiplam

Table of contents

Opinion

On 25 February 2021, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product Evrysdi1, intended for the treatment of spinal muscular atrophy (SMA). Evrysdi was reviewed under EMA’s accelerated assessment programme. The applicant for this medicinal product is Roche Registration GmbH.

Evrysdi will be available as 0.75 mg/ml powder for oral solution. The active substance of Evrysdi, risdiplam, an SMN2 mRNA splicing modifier, is an orally administered small molecule with systemic distribution (ATC code: M09AX10).

The benefits with Evrysdi are its ability to alter the clinical course of the disease, allowing patients to achieve developmental milestones which would have been otherwise impossible to achieve. The most common side effects are diarrhoea, rash and headache.

The full indication is:

Evrysdi is indicated for the treatment of 5q spinal muscular atrophy (SMA) in patients 2 months of age and older, with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies.

Treatment with Evrysdi should be initiated by a physician with experience in the management of SMA.

Detailed recommendations for the use of this product will be described in the summary of product characteristics (SmPC), which will be published in the European public assessment report (EPAR) and made available in all official European Union languages after the marketing authorisation has been granted by the European Commission.


1 This product was designated as orphan medicine during its development. EMA will now review the information available to date to determine if the orphan designation can be maintained

 

Key facts

Name
Evrysdi
Agency product number
EMEA/H/C/005145
International non-proprietary name (INN) or common name
  • risdiplam
Active substance
  • Risdiplam
Therapeutic area
Muscular Atrophy, Spinal
Accelerated assessment

This medicine had an accelerated assessment. This means that it is a medicine of major interest for public health, so its timeframe for review was 150 evaluation days rather than 210. For more information, see Accelerated assessment.

Orphan

This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation.

Date opinion adopted
25/02/2021
Company name
Roche Registration GmbH 
Status
Positive
Application type
Initial authorisation

Related content

How useful was this page?

Add your rating
Average
1 rating
1 rating