NS089/NCNP-02 is an investigational candidate for patients with Duchenne muscular dystrophy amenable to exon 44 skipping therapy
A microscopic image of calf muscle from a person with Duchenne muscular dystrophy. (Credit: Dr. Edwin P. Ewing, Jr. from Wikimedia Commons)
NS Pharma has secured rare paediatric disease designation from the US Food & Drug Administration (FDA) for its NS089/NCNP-02 (brogidirsen).
NS089/NCNP-02 is an investigational candidate for patients with Duchenne muscular dystrophy amenable to exon 44 skipping therapy.
Primarily occurring in males, Duchenne is a progressive form of muscular dystrophy. It causes progressive weakness and loss of skeletal, cardiac, and respiratory muscles.
Brogidirsen is an antisense nucleotide discovered through joint research between the National Centre for Psychiatry and Neurological Medicine and NS Pharma’s parent company, Nippon Shinyaku.
A Phase 2 study will be carried out in the US by NS Pharma and a Phase 2 study will be carried out in Japan by Nippon Shinyaku as part of the clinical development of NS089/NCNP-02.
The pharmaceutical firm said that more information is expected once the trials are ready to start enrolling participants.
The FDA’s rare paediatric disease status is given for therapies that are intended for serious or life-threatening diseases affecting 200,000 US patients who are 18 years or younger.
In August 2020, the US health regulator granted approval to NS Pharma for Viltepso (viltolarsen) injection to treat Duchenne muscular dystrophy in patients who are eligible for exon 53 skipping therapy.
The approval was granted based on its activity to boost dystrophin, a protein that supports muscle health.