Our collective expertise equips us to bring forth unprecedented treatment options to families living with rare diseases, raising the standard of what’s possible through dedicated partnership combined with scientific excellence.
Fulfilling our mission to help people live healthier, happier lives takes an unwavering devotion to scientific research and development. It takes empathy and compassion from patient-focused emissaries to carefully place scientific advancement into the hands of the people who need it the most: patients with high unmet medical need. And, in the field of rare disease science, it takes patience and tenacity.
“At NS Pharma we recognize scientific progress is the culmination of years of collaborative effort between scientists, physicians, patients, and industry. Throughout that journey, we stand committed to meeting our rare disease communities at the intersection of advocacy, education, and support.”
NS Pharma President Yukiteru Sugiyama, Ph.D.
Drug discovery and clinical development at NS Pharma happens in concert with our parent company, Nippon Shinyaku. We work in the fields of intractable and rare diseases, developing small molecule, nucleic acid, gene, and cell therapies. Our science is nurtured through global clinical trials and partnerships with best-in-class organizations and medical facilities.
NS Pharma, Inc. is 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.
We go beyond symptom management, providing patients with disease-modifying treatment options that improve and extend quality time with loved ones.
Exon-skipping has the potential to treat Duchenne muscular dystrophy.
JAK1 inhibition is being developed to treat EGPA, a type of vasculitis.
Gene therapy has the potential to address critical genetic mutations, like those causing Hunter syndrome.
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.
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.
