Evrysdi approved in Japan for the treatment of spinal muscular atrophy


PTC Therapeutics has announced that Evrysdi (risdiplam) has been approved in Japan by the Japanese Ministry of Health, Labor and Welfare for the treatment of spinal muscular atrophy (SMA).

The approval has been granted based on data from two pivotal studies, FIREFISH and SUNFISH, evaluating Evrysdi in infants and adults living with type one, two or three SMA.

The first commercial sale will trigger a USD$10million milestone payment to PTC from Roche under its license and collaboration agreement.

Stuart Peltz, CEO of PTC Therapeutics, said: “We are thrilled to see the Evrysdi approval in Japan and are excited that SMA patients have an effective and convenient at home, oral treatment option that avoids the need for in-hospital administration

“The rapid adoption of Evrysdi globally underscores the importance of this drug and the profound benefit this therapy provides to SMA patients.”

Evrysdi is designed to treat SMA by increasing and sustaining the production of SMN protein, which is found throughout the body and is critical for maintaining healthy motor neurons and movement.

Evrysdi, which is now available in 48 countries, has been approved for the treatment of patients with SMA, aged two months and older by the US Food and Drug Administration and the European Medicines Agency. Roche leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics.


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