NS Pharma, Inc. has been developing products to change the way patients and doctors fight rare diseases.
VILTEPSO® (viltolarsen), an exon-skipping therapy designed for patients with Duchenne muscular dystrophy (DMD), was granted accelerated approval by the FDA. Preliminary results from a Phase 3 confirmatory study of VILTEPSO have been received and are undergoing analysis and discussion with the FDA.
View indication and ISIVILTEPSO is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with VILTEPSO. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
In clinical studies, no patients experienced kidney toxicity during treatment with VILTEPSO. However, kidney toxicity from drugs like VILTEPSO may be possible. Your doctor may monitor the health of your kidneys before starting and during treatment with VILTEPSO.
Common side effects include upper respiratory tract infection, injection site reaction, cough, and fever.
For more information about VILTEPSO, see full Prescribing Information.
News / February 7, 2025
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