The US Food and Drug Administration (FDA) has granted NS Pharma’s NS-089/NCNP-02 breakthrough therapy designation in a move that will help ramp up the future treatment options for Duchenne Muscular Dystrophy (DMD).

The news follows another recent FDA win for NS Pharma, which is owned by Japanese pharmaceutical company Nippon Shinyaku, after its therapy was awarded a rare paediatric disease (RPD) designation earlier in July.

A first-in-human clinical trial (NCT04129294) in six Japanese patients with DMD amenable to exon 44 skipping showed that the therapy increased dystrophin protein expression, and improved the motor function of patients as measured by The North Star Ambulatory Assessment. NS-089/NCNP-02 is an antisense nucleotide that helps cells skip over a particular exon. In the case of DMD patients, this is the mutated – and therefore dysfunctional – exon 44 in the DMD gene.

Nippon Shinyaku discovered NS-089/NCNP-02 in a joint research project with the National Center of Neurology and Psychiatry in Tokyo. NS Pharma is aiming to make NS-089/NCNP-02 the first DMD therapy for exon 44 skipping. Sarepta already has marketed antisense oligonucleotide therapies like Exondys 51 (eteplirsen), Vyondys 53 (golodirsen) and Amondys 45 (casimersen) that target exon 51, exon 53, and exon 45 respectively. NS Pharma itself has an approved exon 53 skipping treatment too – Viltepso (viltolarsen).

Sarepta was recently in the news after the FDA approved the much-anticipated gene therapy Elevidys (delandistrogene moxeparvovec-rokl). The treatment, which joined an exclusive club of the 32 FDA-approved cell and gene therapies, is for children with DMD who have a confirmed mutation in the DMD gene. The Muscular Dystrophy Association hailed the therapy as “ground-breaking”.

Reference：https://www.pharmaceutical-technology.com/news/ns-pharma-scores-breakthrough-therapy-status-for-dmd-therapy/