NS Pharma, a unit of Japan-based Nippon Shinyaku, has received orphan drug designation from the European Commission (EC) for NS-229 to treat eosinophilic granulomatosis with polyangiitis (EGPA).
Developed in-house by NS Pharma, NS-229 is a potent and selective Janus kinase (JAK) 1 inhibitor, being developed to treat EGPA.
It is designed to restrain the excessive activation of T cells, B cells and certain white blood cells, said NS Pharma.
EGPA is a rare autoimmune disease that is generally accompanied by symptoms of allergic rhinitis and bronchial asthma.
The lungs, sinuses, skin, kidneys, and peripheral nerves can all sustain tissue and organ damage as a result of this inflammation in the small blood arteries.
Nippon Shinyaku’s subsidiary expects that the candidate can lower tissue damage and control various symptoms of EGPA.
The company has plans to conduct a Phase 2 global trial of NS-229.
NS Pharma research and development vice president Takeshi Seita said: “EGPA is a serious, life-threatening disease with unmet medical need.
“We are encouraged that our innovative therapy will proceed in development for the patients who need treatment.”
EC designates some medications as orphan drugs when they are meant to treat conditions that are either life-threatening or severely incapacitating and impact fewer than five out of every 10,000 individuals in the European Union.
NS Pharma has been granted a 10-year marketing exclusivity period by the orphan drug designation, which facilitates the company’s ongoing research and development of this treatment.
In July last year, the firm received a rare paediatric disease designation from the US Food & Drug Administration (FDA) for its NS089/NCNP-02 (brogidirsen).
NS089/NCNP-02 is an investigational asset to treat patients with Duchenne muscular dystrophy amenable to exon 44 skipping therapy.
The candidate has been developed via joint research between the National Centre for Psychiatry and Neurological Medicine and Nippon Shinyaku.
