Nippon Shinyaku Co., Ltd. has been developing compounds in several therapeutic fields, including hematologic malignancies and intractable/orphan diseases. NS Pharma, Inc. has been developing NS-018 (for myelofibrosis) and NS-065/NCNP-01 (for Duchenne muscular dystrophy) on behalf of Nippon Shinyaku Co., Ltd.
US: Phase I/II
US: Phase III
*Viltepso (viltolarsen) injection was approved in the United States on August 12, 2020 (accelerated approval); approved in Japan (conditional approval).
NCNP=National Center of Neurology and Psychiatry.
VILTEPSO 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.
Important Safety Information
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.
full Prescribing Information.