FDA grants breakthrough designation for Duchenne muscular dystrophy treatment

The FDA has granted breakthrough therapy designation to an investigational antisense nucleotide therapeutic to treat individuals with Duchenne muscular dystrophy amenable to exon 44 skipping therapy.

According to a press release from NS Pharma Inc., the FDA’s ruling for NS-089/NCNP-02 was based on results from a first-in-human, investigator-initiated clinical trial conducted in Japan.

Earlier in July, the FDA granted NS-089/NCNP-02 rare pediatric disease designation.

The FDA issues the breakthrough designation to expedite the development and review of treatments for serious or life-threatening diseases.

Further clinical development of the novel therapy is expected to include a pair of phase 2 studies — one conducted in the U.S. by NS Pharma and the other commenced in Japan by Nippon Shinyaku — per the release.

NS-089/NCNP-02 was discovered by a joint research effort between NS Pharma’s parent company, Nippon Shinyaku, and the National Center of Neurology and Psychiatry.