Japan Approves Evrysdi as First At-home, Oral SMA Treatment

Evrysdi (risdiplam) has been approved in Japan as an oral, at-home treatment of spinal muscular atrophy (SMA).

The flavored liquid therapy is also approved in the U.S., Europe, and Canada to treat all types of SMA in patients ages 2 months and older, and its use was favored by regulators in 44 other countries. Details on which patient or age groups in Japan would be eligible for Evrysdi were not provided in a press release announcing the decision.

“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,” said Stuart W. Peltz, Ph.D., CEO of PTC Therapeutics, which helped to develop the treatment.

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

Evrysdi is administered daily at home by mouth or via a feeding tube. It works by increasing the levels of SMN, a protein essential to the health of motor neurons, or nerve cells that control muscle movement and whose production is impaired in people with SMA.

Evrysdi is developed by Roche and its subsidiary Genentech, in collaboration with PTC Therapeutics and the SMA Foundation.

The approval was based on data from two pivotal Phase 2/3 trials — FIREFISH (NCT02913482) and SUNFISH (NCT02908685) — currently evaluating Evrysdi’s safety and efficacy in nearly 300 people, ages 2 months to 25 years, with SMA types 1, 2, and 3. The trials are underway across clinical sites around the world, including Japan.

Top-line results from SUNFISH showed that treatment with Evrysdi significantly improved or stabilized motor function among children and young adults, ages 2 to 25, with SMA type 2 or 3.

This was also seen in the FIREFISH trial evaluating Evrysdi in infants 1–7 months old with type 1 SMA. Two-year data showed that motor milestones achieved in the first year were maintained in the second year.

Evrysdi’s therapeutic benefits are also being tested in the Phase 2 JEWELFISH (NCT03032172) and RAINBOWFISH (NCT03779334) trials.

The JEWELFISH study, which has completed enrollment, covers patients 6 months to 60 years old and includes those previously treated with other SMA-targeting therapies.

RAINBOWFISH is still recruiting newborns up to 6 weeks old with a genetic diagnosis of SMA but no evidence of symptoms. More information on this global trial is available here.

Under a license and collaboration agreement, Roche is to make a $10 million milestone payment to PTC once Evrysdi begins to be commercialized in Japan.