NS Pharma gains EC orphan drug designation to treat EGPA

2024-01-23

孤儿药突破性疗法临床2期快速通道

NS Pharma gains EC orphan drug designation to treat EGPA

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来源: Pharmaceutical Technology

NS-229 is being developed as a treatment for the rare autoimmune disease eosinophilic granulomatosis with polyangiitis. Credit: Mongkolchon Akesin / Shutterstock.com.

NS Pharma has received orphan drug designation (ODD) from the European Commission (EC) for NS-229, which is being developed to treat eosinophilic granulomatosis with polyangiitis (EGPA), a rare autoimmune disease.

A selective inhibitor of Janus kinase (JAK) 1, NS-229 is designed to suppress the excessive T cell, B cell and specific white blood cell activation caused by the condition.

The drug reduces damage to tissues and alleviates symptoms associated with EGPA.

The company plans to commence a Phase II global clinical trial of NS-229 shortly.

EGPA is characterised by inflammation in small blood vessels, leading to tissue and organ damage, particularly affecting the lungs, sinuses, peripheral nerves, skin and kidneys.

The cause of EGPA remains unidentified.

NS Pharma research and development vice-president Takeshi Seita stated: “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.”

The EC’s ODD designation is reserved for therapies aimed at treating conditions affecting fewer than five in 10,000 individuals within the European Union and that are considered life-threatening or debilitating.

ODD status provides the company with a marketing exclusivity period of ten years within the European Union, supporting the ongoing development and assessment of NS-229.

In a related development, the EC granted ODD to NS Pharma’s NS-089/NCNP-02 in December 2023.

This drug is in development for Duchenne muscular dystrophy, a rare and fatal genetic ailment primarily affecting men.

NS-089/NCNP-02 is designed to act on a gene mutation amenable to exon 44 skipping.

The drug previously received rare paediatric disease designation in June 2023, breakthrough therapy designation in July and orphan drug designation from the US Food and Drug Administration (FDA) at around the same time.

The company focuses on research in rare diseases, including exon-skipping technology, to provide new therapy options.

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