Viltepso Commercially Available in the US for DMD Treatment

Viltepso Commercially Available in the US for DMD Treatment
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NS Pharma‘s Viltepso (viltolarsen) is now commercially available in the U.S. for people with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping.

The FDA conditionally approved Viltepso earlier this month, and continued approval remains contingent upon confirmation of clinical benefit in the Phase 3 RACER53 clinical trial (NCT04060199). That trial is currently recruiting nearly 74 boys, ages 4 to 7, in several countries, including the U.S., Japan, Canada, and Europe. More information is available here.

“The response by the DMD community to the FDA approval of Viltepso has been incredibly uplifting, and underscores NS Pharma’s responsibility that goes beyond the development of new and exciting treatment options,” Tsugio Tanaka, president of NS Pharma, said in a press release.

Viltepso is administered via intravenous (into-the-vein) infusion of 80 mg/kg of body weight. The infusion is delivered by a trained healthcare professional over the course of an hour, once per week. Patients may choose to receive Viltepso at home, at a treatment center, or in a hospital.

The treatment is available through a network of specialty distributors and specialty pharmacy providers, selected for their experience in providing responsive and reliable service to healthcare providers and families with DMD, said NS Pharma.

The therapy previously was made available in the U.S. for eligible patients through an expanded access program.

NS Pharma, the U.S. subsidiary of Nippon Shinyaku, launched the NS Support program to help families, physicians, and healthcare professionals gain access to Viltepso and be reimbursed for it. The company also plans to host webinars on how to coordinate care. Webinar information and registration can be found on the the company’s Twitter and LinkedIn accounts.

“Through our NS Support program, we will assist each patient and family obtain access to Viltepso, and help navigate obstacles related to location in the U.S. or financial circumstances,” said Tanaka.

Viltepso also has received regulatory approval in Japan.

People with DMD lack dystrophin, an essential protein for muscle health. Viltepso is an exon-skipping therapy intended to “mask” the mutated exon 53 to enable the production a shorter but functional version of dystrophin. About 8% of all DMD patients have mutations in exon 53. (Exons are the DNA regions containing information to generate proteins.)

The FDA based its approval of the medicine on the results of a Phase 2 trial (NCT02740972) conducted in North America and a second Phase 1/2 study (Japic CTI-163291), held in Japan, both in boys with DMD amenable to exon 53 skipping. In the North American-based study, treatment with weekly 80 mg/kg doses of Viltepso increased dystrophin levels to nearly 6% of normal after 24 weeks of therapy.

Forest Ray received his PhD in systems biology from Columbia University, where he developed tools to match drug side effects to other diseases. He has since worked as a journalist and science writer, covering topics from rare diseases to the intersection between environmental science and social justice. He currently lives in Long Beach, California.
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José is a science news writer with a PhD in Neuroscience from Universidade of Porto, in Portugal. He has also studied Biochemistry at Universidade do Porto and was a postdoctoral associate at Weill Cornell Medicine, in New York, and at The University of Western Ontario in London, Ontario, Canada. His work has ranged from the association of central cardiovascular and pain control to the neurobiological basis of hypertension, and the molecular pathways driving Alzheimer’s disease.
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Forest Ray received his PhD in systems biology from Columbia University, where he developed tools to match drug side effects to other diseases. He has since worked as a journalist and science writer, covering topics from rare diseases to the intersection between environmental science and social justice. He currently lives in Long Beach, California.
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