NS Pharma’s NS-089/NCNP-02 gains FDA rare paediatric disease status

2023-07-10

孤儿药寡核苷酸临床2期

NS Pharma’s NS-089/NCNP-02 gains FDA rare paediatric disease status

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来源: Pharmaceutical Technology

NS Pharma’s NS-089/NCNP-02 (brogidirsen) is intended for patients with Duchenne muscular dystrophy. Credit: Jose Luis Calvo via Shutterstock.com.

The US Food and Drug Administration (FDA) has awarded rare paediatric disease (RPD) designation to NS Pharma’s investigational therapy, NS-089/NCNP-02 (brogidirsen), to treat Duchenne muscular dystrophy.

The condition is responsive to exon 44-skipping therapy.

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NS-089/NCNP-02 is an antisense nucleotide discovered in collaborative research between the National Center for Psychiatry and Neurological Medicine and Nippon Shinyaku, the parent firm of NS Pharma.

The drug candidate’s clinical development comprises a Phase II clinical trial to be carried out in the US by NS Pharma and a Phase II study to be conducted by Nippon Shinyaku in Japan.

NS Pharma will provide further trial data once subject enrolment has commenced.

The regulatory agency granted RPD status for therapies that could potentially treat serious or life-threatening ailments affecting 200,000 US patients who are 18 years or younger.

Duchenne is a progressive type of muscular dystrophy seen primarily in men. It leads to gradual weakness and skeletal, cardiac and respiratory muscle loss.

In December 2022, NS Pharma received orphan drug status from the US FDA for an investigational candidate, NS-018 (ilginatinib), to treat myelofibrosis, a rare type of blood cancer.

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