NS Support will provide patients and clinicians with information on product availability and program enrollment. The hub can be reached by telephone at 833-677-8778, Monday through Friday from 8 a.m. to 8 p.m. ET.
The FDA placed viltolarsen on fast track development, and designated it an orphan drug and rare pediatric disease potential treatment for DMD patients amenable to exon 53 skipping. In Europe, the therapy was also given orphan drug designation for the same indication.
“Based on our conversations with the DMD community, there is a high demand for assistance with navigating the complicated insurance landscape for DMD patients amenable to exon 53 skipping therapy,” Tsugio Tanaka, president of NS Pharma, said in a press release.
“We want to ensure the smoothest patient experience possible and a rapid journey towards access and treatment. That is why we are launching NS Support now, so we can begin the process of engaging with families who want to learn more about what NS Support can offer,” added Tanaka.
DMD is the most common of a group of over 30 muscular dystrophies, all inherited genetic conditions. In Duchenne, a mutation in the DMD gene causes the body to produce little to no functional dystrophin, a protein essential for the health of muscle cells and their connection to surrounding tissues.
Dystrophin production can be restored by skipping over the mutation. Genes store instructions for making proteins on DNA segments called exons. A mutation in a single exon can result in an unstable protein, if it results in a protein at all.
Viltolarsen is designed to conceal exon 53 of the DMD gene, while allowing a shorter but functional dystrophin protein to be made from the remaining exons. Mutations in exon 53 account for approximately 8% of DMD cases.
An ongoing Phase 3 clinical trial (NCT04060199), called RACER 53, is assessing viltolarsen’s safety and efficacy in up to 74 boys ages 4–7 with DMD and able to walk. It is enrolling patients amenable to exon 53 skipping at 26 locations worldwide, including across the U.S., in Canada and Europe.
More information on contacts and study sites is available here.
NS Pharma’s FDA application is supported by data from a Phase 2 trial (NCT02740972) in the U.S. and Canada, and from a Phase 1/2 trial in Japan.
Results of the North American study showed that viltolarsen, injected directly into the bloodstream, restored dystrophin production in the muscles of boys ages 4 to 10. Treatment up to 24 weeks was safe and well tolerated.
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