PARAMUS, NJ: June 26, 2026 – NS Pharma, Inc. (NS Pharma; Headquarters: Paramus, NJ; President: Yukiteru Sugiyama) announced today that its parent company, Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; Headquarters: Kyoto, Japan; President: Toru Nakai) has entered into an option agreement with Elixirgen Therapeutics, Inc. (Elixirgen; Headquarters, Baltimore, MD; CEO: Aki Ko) under which Nippon Shinyaku may obtain the exclusive worldwide rights to develop and commercialize EXG-7001 for the treatment of Duchenne muscular dystrophy (DMD). The agreement was facilitated by the NS Pharma Innovation Research Partnering (IRP) team, located in Cambridge, MA.
“Collaboration is essential in furthering scientific innovation, and we are proud to partner with Elixirgen to expand treatment options for the Duchenne community,” said NS Pharma President Yukiteru Sugiyama. “This partnership represents our long-term commitment to supporting our community and the necessary scientific exploration needed to combat rare disease.”
EXG-7001 is an investigational, locally administered, mRNA based therapeutic drug that expresses full-length human dystrophin protein. Regardless of genetic mutation, it is intended to suppress local muscle dysfunction by expressing the full-length dystrophin protein, which is deficient within the muscle cells of patients with DMD. EXG-7001 is in preclinical development for the treatment of DMD. Elixirgen is currently preparing for clinical trials in the United States. Following the exercise of the option rights by Nippon Shinyaku and regulatory approval in the United States, NS Pharma intends to commercialize EXG-7001.
About Duchenne Muscular Dystrophy (Duchenne/DMD)
Duchenne is a form of muscular dystrophy that occurs primarily in males. It causes progressive weakness and loss of skeletal, cardiac, and respiratory muscles. Early signs of Duchenne may include delayed ability to sit, stand or walk. There is a progressive loss of mobility, and by adolescence, patients with Duchenne may require the use of a wheelchair. Cardiac and respiratory muscle problems begin in the teenage years and lead to serious, life-threatening complications. For more information about Duchenne, please visit wespeakduchenne.com.
About Nippon Shinyaku
Based on Nippon Shinyaku’s business philosophy, “Helping people lead healthier, happier lives,” we aim to be an organization trusted by the community through creating unique medicines that will bring hope to patients and families suffering from illness. For more information, please visit nippon-shinyaku.co.jp/english.
About Elixirgen Therapeutics, Inc.
Elixirgen is a clinical stage biotechnology company focused on the treatment of rare and aging-associated diseases using its cutting-edge technologies. For more information, visit ElixirgenTx.com and follow on LinkedIn.
About NS Pharma, Inc.
NS Pharma, Inc., is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. NS Pharma is a registered trademark of the Nippon Shinyaku Co., Ltd. For more information, please visit nspharma.com.
US Media Contact: [email protected]
PARAMUS, NJ: June 23, 2026 – NS Pharma, Inc. (NS Pharma; Headquarters: Paramus, New Jersey, USA; President: Yukiteru Sugiyama) announced today that its parent company, Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; Headquarters: Kyoto, Japan; President: Toru Nakai), has entered into a Sponsored Research Agreement (SRA) with Boston Children’s Hospital (Headquarters: Boston, Massachusetts, USA; Chief Executive Officer: Kevin B. Churchwell) for the purpose of developing innovative therapeutic drugs in the field of neurology. The agreement was facilitated by the NS Pharma Innovation Research Partnering (IRP) team, located in Cambridge, MA.
Following the initiation of the strategic collaboration announced in July 2025, Nippon Shinyaku has selected key research themes as proposed by Boston Children’s Hospital as joint research programs. Through co-creation, Nippon Shinyaku will integrate Boston Children’s Hospital’s world class research capabilities with the company’s drug discovery expertise to accelerate the creation of new neurologic therapeutic options for patients with unmet medical needs.
“This new agreement demonstrates meaningful collaborative progress towards furthering rare disease research and the benefits of our combined expertise,” said Dr. Yukiteru Sugiyama, NS Pharma President. “We look forward to continuing to deepen our alliance with Boston Children’s Hospital and setting a positive example of impactful partnership for our rare disease communities.”
About NS Pharma, Inc.
NS Pharma, Inc., is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. NS Pharma is a registered trademark of the Nippon Shinyaku Co., Ltd. For more information, please visit nspharma.com.
US Media Contact:
[email protected]
PARAMUS, NJ: March 9, 2026 – NS Pharma, Inc. (NS Pharma, New Jersey, USA; President, Yukiteru Sugiyama ), a biopharmaceutical leader in rare diseases and subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku), announced today that the National Center of Neurology and Psychiatry (NCNP, Kodaira City; President, Kazuyuki Nakagome) presented 4.5-year safety and efficacy data based on the open-label extension study, including an investigator-initiated clinical trial of brogidirsen (NS-089/NCNP-02) for the treatment of Duchenne muscular dystrophy (DMD) at the 2026 MDA Clinical & Scientific Conference from March 8-11, 2026.
Brogidirsen is an antisense oligonucleotide co-discovered by Nippon Shinyaku and NCNP as an investigational therapy for DMD patients with dystrophin gene mutations that are amenable to exon 44 skipping.
“We are thrilled to see the data continue to demonstrate long-term efficacy in DMD patients amenable to exon 44 skipping and while maintaining patient safety,” said NS Pharma President, Yukiteru Sugiyama, Ph.D. “We are committed to expanding available treatment options to the DMD community and we look forward to realizing that ambition.”
The presented data are based on the first-in-human Phase 1/2 open-label investigator-initiated clinical trial conducted by NCNP and the subsequent Phase 2 open-label extension trial conducted by Nippon Shinyaku. These studies evaluate the safety and efficacy of brogidirsen in six participants who received weekly IV dosing of brogidirsen.
Findings include:
These findings support the potential of brogidirsen to modify the progression of DMD. This extension trial is ongoing to investigate the safety and efficacy of longer-term administration.
A second, ongoing global Phase II study of brogidirsen is being conducted by Nippon Shinyaku and NS Pharma to further evaluate the safety and efficacy of brogidirsen. Learn more at ClinicalTrials.gov.
About Duchenne Muscular Dystrophy (DMD)
Duchenne is a form of muscular dystrophy that occurs primarily in males. It causes progressive weakness and loss of skeletal, cardiac, and respiratory muscles. Early signs of DMD may include delayed ability to sit, stand or walk. There is a progressive loss of mobility, and by adolescence, patients with Duchenne may require the use of a wheelchair. Cardiac and respiratory muscle problems begin in the teenage years and lead to serious, life-threatening complications. For more information, please visit wespeakduchenne.com.
About NS Pharma, Inc.
NS Pharma, Inc., is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. NS Pharma is a registered trademark of the Nippon Shinyaku Co., Ltd. For more information, please visit nspharma.com.
US Media Contact:
[email protected]
PARAMUS, NJ: October 14, 2025 – NS Pharma, Inc. (NS Pharma), a biopharmaceutical leader in rare disease and subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku), announced today that the National Center of Neurology and Psychiatry (NCNP, Kodaira City; President, Kazuyuki Nakagome) presented 3.5-year efficacy and safety data from the open-label extension of an investigator-initiated clinical trial of brogidirsen (NS-089/NCNP-02) for the treatment of Duchenne muscular dystrophy (DMD)at the 30th annual International Congress of the World Muscle Society from October 7 to 11, 2025.
Brogidirsen is an antisense oligonucleotide co-discovered by Nippon Shinyaku and NCNP as an investigational therapy for DMD patients with dystrophin gene mutations that are amenable to exon 44 skipping.
“These long-term motor function data suggest the potential for brogidirsen to slow disease progression in DMD patients amenable to exon 44 skipping and support the robust increases in dystrophin production seen earlier in the trial,” said NS Pharma President, Yukiteru Sugiyama, Ph.D. “We are proud to offer hope to families and continue to demonstrate our commitment to the DMD community
The presented data are based on the investigator-initiated clinical trial conducted by NCNP and its extension study conducted by Nippon Shinyaku. These studies evaluated the efficacy and safety of brogidirsen in 6 participants who received weekly IV dosing of brogidirsen.
Findings include:
• Consistent functional benefits – High exon 44 skipping efficiency and dystrophin expression levels were observed in biopsied muscles at week 25/26 and week 99/100.
• Maintenance of motor function – Participants who remain ambulant after long-term brogidirsen administration maintained their motor function in evaluations including North Star Ambulatory Assessment.
• Acceptable safety profile – After 3.5-year of receiving brogidirsen, no serious or severe adverse events, or anaphylaxis related to long-term brogidirsen administration, were reported, and there were no discontinuations.
These findings support the potential of brogidirsen to modify the progression of DMD. This long-term extension trial is ongoing to investigate the efficacy and safety of longer-term administration.
Further, a global Phase II study of brogidirsen is being conducted by Nippon Shinyaku and NS Pharma, Inc. (Headquarters: New Jersey, USA; President: Yukiteru Sugiyama). Learn more at ClinicalTrials.gov.
About Duchenne Muscular Dystrophy (DMD)
Duchenne is a form of muscular dystrophy that occurs primarily in males. It causes progressive weakness and loss of skeletal, cardiac, and respiratory muscles. Early signs of DMD may include delayed ability to sit, stand or walk. There is a progressive loss of mobility, and by adolescence, patients with Duchenne may require the use of a wheelchair. Cardiac and respiratory muscle problems begin in the teenage years and lead to serious, life-threatening complications. For more information, please visit wespeakduchenne.com.
About NS Pharma, Inc.
NS Pharma, Inc., is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. NS Pharma is a registered trademark of the Nippon Shinyaku Co., Ltd. For more information, please visit nspharma.com.
US Media Contact:
[email protected]
PARAMUS, NJ: September 19, 2025 – NS Pharma, Inc. announced today that the U.S. Food & Drug Administration (FDA) has granted Orphan Drug Designation to NS-051/NCNP-04 which is being developed for the treatment of Duchenne muscular dystrophy (Duchenne) in patients amenable to exon 51 skipping. The FDA issues Orphan Drug Designations to support the development and evaluation of new treatments to prevent, diagnose, or treat a rare disease or condition.
NS-051/NCNP-04 previously received Rare Pediatric Disease Designation from the FDA in January 2025. For more information, see the press release here.
Duchenne is a progressive muscle wasting disease caused by a deficiency of the dystrophin protein. It leads to weakness of skeletal, cardiac, and respiratory muscles. There are many types of genetic mutations that can cause Duchenne, and NS-051/NCNP-04 is being developed to treat patients with confirmed gene mutations amenable to exon 51 skipping therapy.
NS-051/NCNP-04 is an antisense oligonucleotide co-discovered by the National Center of Neurology and Psychiatry (NCNP) and Nippon Shinyaku. NS-051/NCNP-04 promotes the skipping of exon 51 within the dystrophin gene, causing the production of a shortened dystrophin protein containing essential functional portions. This is expected to have the effect of stabilizing or improving muscle function.
About Duchenne Muscular Dystrophy (Duchenne)
Duchenne is a form of muscular dystrophy that occurs primarily in males. It causes progressive weakness and loss of skeletal, cardiac, and respiratory muscles. Early signs of Duchenne may include delayed ability to sit, stand or walk. There is a progressive loss of mobility, and by adolescence, patients with Duchenne may require the use of a wheelchair. Cardiac and respiratory muscle problems begin in the teenage years and lead to serious, life-threatening complications. For more information about Duchenne, please visit wespeakduchenne.com.
About NS Pharma, Inc.
NS Pharma, Inc., is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. NS Pharma is a registered trademark of the Nippon Shinyaku Co., Ltd. For more information, please visit nspharma.com.
US Media Contact:
[email protected]
PARAMUS, NJ: September 9, 2025 – NS Pharma, Inc. (NS Pharma) a subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku), announced today that the U.S Food & Drug Administration (FDA) has granted Fast Track designation to NS-229, which is being developed for the treatment of the rare disease eosinophilic granulomatosis with polyangiitis (EGPA). NS-229 is being investigated as a selective Janus kinase 1 (JAK1) inhibitor to help regulate immune cell function and prevent the immune system from causing tissue damage.
FDA Fast Track designation status is granted to treatments of serious medical conditions that fulfil an unmet medical need. This designation allows for expedited FDA review, including more frequent collaboration with the FDA throughout the application process, to facilitate the delivery of important new therapies more quickly. NS-229 was granted Orphan Drug Designation by the FDA in April 2025.
About EGPA
EGPA, previously known as Churg-Strauss syndrome, is a rare autoimmune disease that causes inflammation in the small-to-medium-sized blood vessels which can cause tissue and organ damage to the lungs, sinuses, peripheral nerves, skin, and kidneys. EGPA is generally preceded by symptoms of bronchial asthma and allergic rhinitis. The cause is unknown. It is estimated that EGPA affects between 5,600 and 14,500 people in the U.S.*
A Phase 2, double-blind, randomized placebo-controlled global study of NS-229 is being conducted by Nippon Shinyaku and NS Pharma to assess the efficacy and safety of the investigational JAK1 inhibitor NS-229, in treating EGPA patients. By inhibiting JAK1, NS-229 may help regulate the overactive immune response characteristic of EGPA, potentially limiting the damage to heathy tissues.
About NS Pharma, Inc.
NS Pharma, Inc., is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. NS Pharma is a registered trademark of the Nippon Shinyaku Co., Ltd. For more information, please visit www.nspharma.com.
US Media Contact:
[email protected]
*Estimated prevalences 1.7 / 100,0001) and 4.4 / 100,0002) were multiplied by a 2023 U.S. population estimate of around 330 million and rounded to nearest hundred.
1) Bell, CF., Lau, M., Shen, Q. Clinical and Economic Characteristics of Patients Diagnosed with Eosinophilic Granulomatosis with Polyangiitis (EGPA, formerly Churg-Strauss Syndrome) in the United States [abstract]. Arthritis Rheumatol. 2018; 70 (suppl 9).
2) Berti A, Cornec D, Crowson CS, Specks U, Matteson EL. The Epidemiology of Antineutrophil Cytoplasmic Autoantibody-Associated Vasculitis in Olmsted County, Minnesota: A Twenty-Year US Population-Based Study. Arthritis Rheumatol. 2017;69:2338-2350.
PARAMUS, NJ: July 1, 2025 – NS Pharma, Inc. (NS Pharma) announced today that its parent company, Nippon Shinyaku Co., Ltd. (Nippon Shinyaku), headquartered in Kyoto, Japan, has finalized a strategic partnership with Boston Children’s Hospital (Boston Children’s) (Headquarters: Boston, Massachusetts, United States), to support the research and development of innovative therapies for rare diseases. Nippon Shinyaku and Boston Children’s will leverage their combined expertise in clinical research and pharmaceutical development to bring more treatment options to patients. The agreement was facilitated by the NS Pharma Innovation Research Partnering (IRP) team, located in Cambridge, MA.
“Through our strategic alliance with Boston Children’s Hospital, the NS Group will continue to apply advanced medical technology in the field of rare diseases,” said NS Pharma President Yukiteru Sugiyama. “We are excited about the extensive opportunities for innovation derived from this collaboration and the ability to deliver new therapies to patients suffering from these conditions as soon as possible.”
Under this alliance, Nippon Shinyaku and Boston Children’s will align their focus areas, aiming to inspire and support research proposals focused on rare diseases. Nippon Shinyaku will assess the Boston Children’s proposals and select those of interest for joint research. Proposals may span a range of developmental stages, from early investigations into the biological mechanisms of rare disease, to the identification of possible therapeutic targets, or the generation and validation of new therapeutic entities.
About NS Pharma, Inc.
NS Pharma, Inc., is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd., headquartered in Paramus, NJ. NS Pharma is a registered trademark of the Nippon Shinyaku Co., Ltd. Nippon Shinyaku is focused on incurable and rare diseases and is currently marketing Viltepso® (nucleic acid drug), a treatment for Duchenne muscular dystrophy developed in-house, in Japan and the United States. For more information, please visit www.nspharma.com.
U.S. Media Contact:
[email protected]
PARAMUS, NJ: January 23, 2025 – NS Pharma, Inc. (NS Pharma), a subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku), announced that Food and Drug Administration (FDA) has granted rare pediatric disease designation to NS-051/NCNP-04 which is being developed for the treatment of Duchenne muscular dystrophy (Duchenne). The FDA’s rare pediatric disease designation is granted for treatments of serious or life-threatening diseases that affect children under the age of 18 and fewer than 200,000 patients in the United States.
Duchenne is a progressive muscle wasting disease caused by a deficiency of the dystrophin protein. It leads to weakness of skeletal, cardiac, and respiratory muscles. There are many types of genetic mutations that can cause Duchenne, and NS-051/NCNP-04 is being developed to treat patients with confirmed gene mutations amenable to exon 51 skipping therapy.
NS-051/NCNP-04 is an antisense oligonucleotide co-discovered by the National Center of Neurology and Psychiatry (NCNP) and Nippon Shinyaku. NS-051/NCNP-04 promotes skipping of exon 51 of the dystrophin gene that produces a shortened dystrophin protein containing essential functional portions, which is expected to have the effect of stabilizing or improving muscle function.
NS Pharma is working to develop products for patients with rare diseases.
About Duchenne Muscular Dystrophy (Duchenne)
Duchenne is a form of muscular dystrophy that occurs primarily in males. It causes progressive weakness and loss of skeletal, cardiac, and respiratory muscles. Early signs of Duchenne may include delayed ability to sit, stand or walk. There is a progressive loss of mobility, and by adolescence, patients with Duchenne may require the use of a wheelchair. Cardiac and respiratory muscle problems begin in the teenage years and lead to serious, life-threatening complications. For more information about Duchenne, please visit wespeakduchenne.com.
About NS Pharma, Inc.
NS Pharma, Inc., is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. NS Pharma is a registered trademark of the Nippon Shinyaku Co., Ltd. For more information, please visit www.nspharma.com.
US Media Contact:
[email protected]
Selective JAK1 Inhibition is a 21st Century science. At NS Pharma, we are investigating this therapeutic approach for the treatment of rare diseases like eosinophilic granulomatosis with polyangiitis (EGPA).
With JAK1 inhibition, we consider two important variables: potency and target selectivity. These aspects are crucial when developing safe and effective therapies. Higher potency means a lower dose of the drug is needed for it to have its intended effect. High selectivity helps to ensure that the drug interacts only with its intended target, minimizing the risk of side effects.
The main challenge of working with JAK1 inhibitors to treat EPGA is that different therapies are needed to treat distinct stages of EGPA – no single therapy has been found to treat all stages of this condition.1 Scientists are still working to understand the causes of EGPA fully. This is complex, as they believe several factors are involved.
On the plus side, if JAK1 inhibition is effective, researchers hope it could reduce symptoms of EGPA regardless of disease stage. This would present a treatment opportunity for many patients with EGPA, rather than just a select few. Researchers are currently conducting a Phase 2 clinical trial to evaluate its safety and effectiveness in treating EGPA.
EGPA is extremely rare, affecting just about 0.0017% of the population.2 This makes accessing diagnosis and effective treatment challenging for patients. If JAK1 inhibition for EGPA is successful, this therapy could support healthier and happier futures for people with EGPA.
1 Eosinophilic Granulomatosis with Polyangiitis – Vasculitis Foundation. Accessed on July 19, 2024.https://www.vasculitisfoundation.org/education/vasculitis-types/eosinophilic-granulomatosis-with-polyangiitis/
1 Eosinophilic Granulomatosis with Polyangiitis (EGPA) – American Lung Association. Accessed on July 19, 2024. https://www.lung.org/lung-health-diseases/lung-disease-lookup/egpa
2 Vaglio A, Buzio C, Zwerina J. Eosinophilic granulomatosis with polyangiitis (Churg-Strauss): state of the art. Allergy. 2013 Mar;68(3):261-73. doi: 10.1111/all.12088
May 27, 2024
NS Pharma Shares Preliminary Results of Viltolarsen (NS-065 / NCNP-01)
Phase 3 Clinical Trial (RACER53 Study)
PARAMUS, NJ: May 27, 2024 – NS Pharma, Inc. (NS Pharma), a subsidiary of Nippon Shinyaku Co., Ltd., announced today that it has received preliminary analysis results from the global Phase 3 clinical trial (RACER53 study, NCT04060199) of NS-065/NCNP-01 (generic name: viltolarsen).
Viltolarsen was approved by the United States (US) Food and Drug Administration (FDA) in 2020 under the brand name VILTEPSO® – for the treatment of Duchenne muscular dystrophy (Duchenne) in patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping – under the FDA accelerated approval pathway based on an increase in dystrophin production in skeletal muscle observed in treated patients. In the US, continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
The RACER53 Study is a randomized, double-blind, placebo-controlled, comparative study of 77 ambulatory boys with Duchenne. The study evaluated the efficacy and safety of an 80 mg/kg once weekly dosing of the treatment – versus placebo – for 48 weeks and was intended to serve as a confirmatory study.
The primary endpoint of the study was Time to Stand from Supine evaluated as velocity (rise/sec). The viltolarsen group showed a trend of increased velocity from baseline after treatment for 48 weeks. However, the placebo group also showed a trend of increased velocity, and there was no statistically significant difference between the viltolarsen group and the placebo group.
Preliminary safety results indicated that all adverse events that occurred under viltolarsen treatment were mild or moderate. There were no treatment emergent adverse events that led to discontinuation of the drug during the study.
“We are currently conducting further detailed data analyses and identifying factors that may have influenced the results (e.g. age, treatment period, and effect of
NEWS RELEASE
concomitant drugs including glucocorticoid therapy),” said NS Pharma President Tsugio Tanaka, MSc. “Considering the results of prior clinical studies, we have confidence that viltolarsen can be a beneficial treatment for amenable patients with Duchenne.”
Specifically, in addition to the increase in dystrophin production in skeletal muscle that formed the basis of the FDA approval, a previously reported Phase 2, open- label, long-term extension study evaluated viltolarsen in 16 subjects between the ages of four and 10 with Duchenne amenable to exon 53 skipping. The study found that subjects receiving viltolarsen showed statistically significant improvements in the study’s primary endpoint of mean change from baseline for Time to Stand at week 205 as compared to a historical control group that was matched for key factors. In this study, treatment emergent adverse events were primarily mild or moderate. No study participants discontinued the study drug due to adverse events.
NS Pharma is currently conducting further detailed data analyses, including post- hoc data analyses, and plans to work closely with regulatory authorities to determine how to proceed based on the results of this analysis and in the best interests of patients. The company will report on additional analyses and discussions with the regulatory authorities at a later date.
About VILTEPSO® (Viltolarsen) Injection
Prior to its approval in the U.S. in August 2020, VILTEPSO was granted Priority Review as well as Rare Pediatric Disease, Orphan Drug and Fast Track Designations. In March 2020, VILTEPSO was approved in Japan for the treatment of patients with Duchenne who are amenable to exon 53 skipping therapy. Prior to its approval in Japan, VILTEPSO was granted the SAKIGAKE designation, orphan drug designation, and designation of Conditional Early Approval System.
Indication
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
Warnings and Precautions: Kidney toxicity was observed in animals who received viltolarsen. Although kidney toxicity was not observed in the clinical studies with
VILTEPSO, the clinical experience with VILTEPSO is limited, and kidney toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. Kidney function should be monitored in patients taking VILTEPSO.
Serum creatinine may not be a reliable measure of kidney function in patients with Duchenne. Serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio should be measured before starting VILTEPSO. Consider also measuring glomerular filtration rate before starting VILTEPSO. During treatment, monitor urine dipstick every month, and serum cystatin C and urine protein-to-creatinine ratio every three months.
Urine should be free of excreted VILTEPSO for monitoring of urine protein. Obtain urine either prior to VILTEPSO infusion, or at least 48 hours after the most recent infusion. Alternatively, use a laboratory test that does not use the reagent pyrogallol red, which has the potential to generate a false positive result due to cross reaction with any VILTEPSO in the urine. If a persistent increase in serum cystatin C or proteinuria is detected, refer to a pediatric nephrologist for further evaluation.
Adverse Reactions: The most common adverse reactions include upper respiratory tract infection, injection site reaction, cough, and pyrexia.
To report an adverse event, or for general inquiries, please call NS Pharma Medical Information at 1-866-NSPHARM (1-866-677-4276)
For more information about VILTEPSO, see full Prescribing Information.
About Duchenne Muscular Dystrophy (Duchenne)
Duchenne is a progressive form of muscular dystrophy that occurs primarily in males. It causes progressive weakness and loss of skeletal, cardiac, and respiratory muscles. Early signs of Duchenne may include delayed ability to sit, stand or walk. There is a progressive loss of mobility, and by adolescence, patients with Duchenne may require the use of a wheelchair. Cardiac and respiratory muscle problems begin in the teenage years and lead to serious, life-threatening complications. For more information about Duchenne, please visit wespeakduchenne.com.
About NS Pharma, Inc.
NS Pharma, Inc., is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. NS Pharma is a registered trademark of the Nippon Shinyaku Co., Ltd. For more information, please visit www.nspharma.com.
U.S. Media Contact: