Friday, December 22, 2023
NS Pharma, Inc. (NS Pharma), a subsidiary of Nippon Shinyaku Co., Ltd., has been granted orphan drug designation by the European Commission (EC) for NS-089/NCNP-02 on December 13, 2023. This drug is currently in development to treat Duchenne muscular dystrophy (Duchenne), a rare and fatal genetic disorder primarily affecting males. Duchenne results from various genetic mutations, and NS-089/NCNP-02 focuses on addressing a specific gene mutation through exon 44 skipping.
Orphan drug designation is awarded by the EC to drugs designed for diseases affecting fewer than five in 10,000 people in the European Union (EU), which are life-threatening or chronically debilitating. This designation provides NS Pharma with a ten-year marketing exclusivity period, supporting the company's ongoing development and assessment of this therapy.
Before receiving the EC designation, NS-089/NCNP-2 obtained rare pediatric disease designation in June 2023, breakthrough therapy designation in July 2023, and orphan drug designation in July 2023 from the U.S. Food and Drug Administration (FDA).
Takeshi Seita, Vice President of Research & Development at NS Pharma, expressed optimism, stating, "We are making significant strides towards providing life-changing treatment for patients with Duchenne amenable to exon 44 skipping." He added, "Our team is eager to continue advancing the development of this groundbreaking science."
NS-089/NCNP-02, an antisense nucleic acid, was discovered through collaborative research between Nippon Shinyaku and the National Center of Neurology and Psychiatry (NCNP). By skipping a portion of the genetic information in the dystrophin gene, it generates a functional dystrophin protein with a slightly shorter chain length, anticipated to mitigate muscle function deterioration.
NS Pharma remains committed to developing therapies for intractable and rare diseases, with a particular focus on expediting treatments for Duchenne patients.
Duchenne is a progressive form of muscular dystrophy predominantly affecting males, resulting in the gradual weakening and loss of skeletal, cardiac, and respiratory muscles. Early indications may include delayed ability to sit, stand, or walk. Mobility progressively declines, often necessitating wheelchair use by adolescence. Cardiac and respiratory issues emerge in the teenage years, leading to severe, life-threatening complications.
Source: prnewswire.com
