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2023-08-17| Trials & Approvals

The U.S. FDA Grants Orphan Drug Designation to NS-089/NCNP-02 for the Treatment of DMD

by Sinead Huang
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Japanese pharmaceutical company Nippon Shinyaku Co., Ltd. (Nippon Shinyaku) announced in early August that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to NS-089/NCNP-02 (brogidirsen) for the treatment of Duchenne Muscular Dystrophy (DMD). Nippon Shinyaku is a biotech company headquartered in Kyoto, dedicated to developing new drugs and actively engaging in innovations in the medical field, especially in the treatment of rare diseases. This designation recognizes the significance of their efforts to provide improved medical options for patients.

Related article: Entrada Hit With Clinical Hold on IND Application for Muscular Dystrophy Therapy

Orphan Drug Designation Provides Special Advantages for DMD Treatment

NS-089/NCNP-02 has received FDA’s “Orphan Drug Designation.” This designation is specifically intended for drugs addressing rare diseases (affecting fewer than 200 thousand patients in the U.S.), providing seven years of exclusive marketing rights and tax incentives to encourage research and development in this area. 

Orphan drugs are medications developed for rare diseases, where traditional drug development and commercialization models might not be financially viable due to the rarity of the conditions. To address this, governments and pharmaceutical companies offer incentives such as tax breaks, market exclusivity, and patent protection to encourage research and development efforts.

Furthermore, NS-089/NCNP-02 has also been granted “Rare Pediatric Disease Designation” by the FDA. This designation provides further support for the drug’s development, particularly for the treatment of pediatric patients. Finally, NS-089/NCNP-02 also received “Breakthrough Therapy Designation” from the FDA this year. This designation signifies the potential of the drug to make significant breakthroughs in DMD treatment, thus expediting its development and approval process. Collectively, these three designations solidify NS-089/NCNP-02’s pivotal role in DMD treatment, offering hope and improvements for patients with this condition.

DMD: A Progressive Muscle Atrophy Disease

DMD is a rare and severe genetic muscle disease, predominantly affecting males. It typically manifests in early childhood, with initial symptoms including difficulty in walking and standing, as well as muscle enlargement. As the disease progresses, patients might require assistance such as wheelchairs, and cardiac and respiratory issues could arise during adolescence.

DMD is caused by a genetic defect in the dystrophin protein. This defect prevents normal production of the dystrophin protein, which is crucial for maintaining the integrity of muscle structure. Lack of dystrophin leads to muscle damage, resulting in muscle atrophy, loss of function, and potential impact on heart and respiratory muscle function.

NS-089/NCNP-02: A Potential Drug for DMD Treatment

NS-089/NCNP-02 is a drug jointly discovered by Nippon Shinyaku and the National Center of Neurology and Psychiatry (NCNP). It falls under the category of antisense nucleic acids, which influence gene expression to address specific genetic defects.

The mechanism of NS-089/NCNP-02 involves exon skipping to correct the genetic defect in the DMD gene. Since DMD patients lack functional dystrophin protein leading to muscle structure loss, NS-089/NCNP-02 can skip exon 44, allowing the produced dystrophin protein to have partial functionality, thereby aiding in maintaining muscle structure integrity.

Currently, NS-089/NCNP-02 is in clinical trial stages. The drug’s development holds promise for providing new treatment options for DMD patients with specific genetic defects, improving their quality of life and slowing disease progression. This research also signifies significant advancements in gene therapy, offering hope for treating rare diseases.

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