RARE Daily

FDA Grants Rare Pediatric Disease Designation to NS Pharma’s Treatment for Duchenne Muscular Dystrophy

July 7, 2023

Rare Daily Staff

The U.S. Food and Drug Administration has granted Rare Pediatric Disease designation to NS Pharma’s investigational antisense nucleotide for patients with Duchenne muscular dystrophy amenable to exon 44 skipping therapy.

Duchenne is a progressive form of muscular dystrophy that occurs primarily in males. Duchenne causes progressive weakness and loss of skeletal, cardiac, and respiratory muscles. Early signs of Duchenne may include delayed ability to sit, stand or walk. There is a progressive loss of mobility, and by adolescence, patients with Duchenne may require the use of a wheelchair. Cardiac and respiratory muscle problems begin in the teenage years and lead to serious, life-threatening complications.

NS-089/NCNP-02 (brogidirsen) is an antisense nucleotide discovered through joint research between NS Pharma’s parent company, Nippon Shinyaku, and the National Center for Psychiatry and Neurological Medicine.

Clinical development of NS-089/NCNP-02 includes a planned phase 2 study in the United States conducted by NS Pharma and a phase 2 study conducted in Japan by Nippon Shinyaku.

The FDA grants Rare Pediatric Disease designation for serious and life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the United States. The Rare Pediatric Disease Priority Review Voucher program is intended to address the challenges that drug companies face when developing treatments for these unique patient populations. Under this program, a sponsor who receives an approval for a drug or biologic for a “rare pediatric disease” may be eligible for a voucher that can be redeemed to receive priority review of a subsequent marketing application for a different product or sold to another sponsor for priority review of their marketing application. Recently, Sarepta Therapeutics sold its voucher $102 million.

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