NEWS RELEASE
November 21, 2023
NS Pharma Unveils Duchenne Heroes, a Program to Inspire and Amplify the Voices of People Living with Duchenne Muscular Dystrophy
Paramus, NJ: November 21, 2023 – NS Pharma, Inc. (“NS Pharma”) today announced the launch of Duchenne Heroes a program created to raise awareness and champion the voices of people affected by Duchenne muscular dystrophy (Duchenne). Duchenne Heroes are individuals, caregivers and family members who hope to inspire others by sharing their personal experiences in navigating diagnosis and daily life with Duchenne.
Duchenne Heroes share their authentic insights on a new educational website, WeSpeakDuchenne.com, which provides straightforward information to help families navigate the complexities of the Duchenne journey, and a platform to hear from others who have been down the same path.
“Our goal is to make a positive impact in the lives of people living with Duchenne by providing helpful information and uplifting voices from the community,” said Gilberto Gil, Director of Patient and Caregiver Marketing at NS Pharma. “The Duchenne Heroes’ insights and authentic experiences can inspire families who may be navigating a Duchenne diagnosis. It is my hope that these unique perspectives combined with easy-to-understand information about Duchenne Muscular Dystrophy, will make WeSpeakDuchenne.com a valuable online resource for the community.”
WeSpeakDuchenne.com includes educational resources such as a “Duchenne-opedia,” a glossary of useful terms to know, as well as a primer on the importance of increasing dystrophin for people with Duchenne, and a section where caregivers share their responses to commonly asked questions around diagnosis and treatment. The Duchenne Heroes Stories page shines a light on different families’ path to diagnosis and the learnings that helped as they navigate life with Duchenne.
“It means a lot to our family to participate in the Duchenne Heroes program as we know how life-changing a Duchenne diagnosis can be,” said Elizabeth Cojeen, member of the Duchenne Heroes program. “Our son, Emmett, inspires us every day with his bright smile and big personality to stay positive, and we hope sharing our story helps other families do the same.”
Additional Duchenne Heroes stories will be included over time to showcase diverse perspectives around topics relevant to the broader community.
“The biggest lesson I’ve learned since my son Brantley’s Duchenne diagnosis is that there is no clear-cut path or single solution for a lot of life’s transitions,” said Dianna Marlow, member of the Duchenne Heroes program. “Our family’s journey, like everyone else’s, is unique when it comes to things like schooling, independence and accessibility. As Duchenne Heroes, we’re proud to share our experiences if it helps to bring ideas or encouragement to other families.”
In addition to being featured on the website, Duchenne Heroes will share their stories across NS Pharma’s social media platforms (Facebook: @NS Pharma, Inc.; X: @NSPharmaInc.) and make appearances at advocacy events in the coming year.
To learn more about the Duchenne Heroes program and explore educational resources, please visit WeSpeakDuchenne.com.
About Duchenne Muscular Dystrophy (Duchenne)
Duchenne is a progressive form of muscular dystrophy that occurs primarily in males. Duchenne
causes progressive weakness and loss of skeletal, cardiac and pulmonary 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 use of a wheelchair. Cardiac and respiratory muscle problems begin in the teenage years and can lead to serious, life- threatening complications.
About NS Pharma, Inc.
NS Pharma, Inc. is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. For more information, please visit https://www.nspharma.com. NS Pharma is a registered trademark of the Nippon Shinyaku group of companies.
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NEWS RELEASE
July 7, 2023
FDA Grants Rare Pediatric Disease Designation to NS-089/NCNP-02 for the Treatment of Duchenne Muscular Dystrophy
NS Pharma, Inc. (NS Pharma; President, Tsugio Tanaka) is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; President, Toru Nakai)
PARAMUS, NJ: July 7, 2023 – NS Pharma, Inc. announced today the U.S. Food & Drug Administration (FDA) has granted Rare Pediatric Disease Designation to NS- 089/NCNP-02 (brogidirsen) an investigational candidate for patients with Duchenne muscular dystrophy amenable to exon 44 skipping therapy.
The FDA’s Rare Pediatric Disease Designation is granted for treatments intended for serious or life-threatening diseases that affect children under the age of 18 and less than 200,000 patients in the U.S.
NS-089/NCNP-02 is an antisense nucleotide discovered through joint research between NS Pharma’s parent company, Nippon Shinyaku, and the National Center for Psychiatry and Neurological Medicine (Kodaira City, President: Kazuyuki Nakagome).
Clinical development of NS-089/NCNP-02 includes a planned Phase 2 study in the United States conducted by NS Pharma and a Phase 2 study conducted in Japan by Nippon Shinyaku. Additional details will be provided once the trials are ready to begin enrolling participants.
About Duchenne Muscular Dystrophy (Duchenne)
Duchenne is a progressive form of muscular dystrophy that occurs primarily in males.
Duchenne 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.
About NS Pharma, Inc.
NS Pharma, Inc., is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. For more information, please visit http://www.nspharma.com. NS Pharma is a registered trademark of the Nippon Shinyaku group of companies.
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May 9, 2023
VILTEPSO® (viltolarsen) injection Four-Year Clinical Trial Data Published in the Journal of Neuromuscular Diseases
Data were from a completed four-year study (up to Week 216) of the open-label extension trial of a VILTEPSO Phase 2 study
NS Pharma, Inc. (NS Pharma; President, Tsugio Tanaka), is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; President, Toru Nakai)
Paramus, NJ: May 9, 2023 – NS Pharma is pleased to announce that previously reported long-term efficacy and safety data (final visit up to Week 216) from the open-label extension of a Phase 2 study of VILTEPSO® (viltolarsen) have been published in the May issue of the Journal of Neuromuscular Diseases. The article, “Efficacy and Safety of Viltolarsen in Boys With Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study,” is freely available under open access (click here).
“In this extension study it was encouraging to see that, compared to the CINRG DNHS control group, Viltepso-treated patients showed maintenance of motor function through the first two years of treatment and experienced delay of disease progression over the following two years of treatment, whereas the control group declined over this same four-year time period,” said Leslie Magnus, MD, Vice President, Medical Affairs. “NS Pharma is pleased that these data support the role of VILTEPSO as an important treatment strategy for DMD patients who are amenable to exon 53 skipping.”
The data published in the Journal of Neuromuscular Diseases are from an open-label trial (N=16) that is the extension of a previous 24-week Phase 2 trial in North America. All 16 patients aged 4 to
<10 years with DMD amenable to exon 53 skipping at baseline in the 24-week study elected to enroll in this long-term trial to continue evaluation of motor function and safety. Assessments of timed function tests (Time to Stand, Time to Run/Walk, Time to Climb 4 Stairs) were compared to a group- matched DMD historical control drawn from the Cooperative International Neuromuscular Research Group Duchenne Natural History Study (CINRG DNHS). Both groups received a stable dose of glucocorticoid treatment.
Efficacy results at Week 205 for VILTEPSO compared to the CINRG DNHS control group for the primary endpoint of mean change from baseline for Time to Stand was 2.7 seconds vs. 8.3 seconds, respectively.
The most frequently reported adverse events were mild to moderate and included cough, nasopharyngitis, rash, pyrexia, and vomiting. This safety profile was similar to that seen in the previous short-term study. There were no treatment-related serious adverse events and no treatment discontinuations.
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 DMD who are amenable to exon 53 skipping therapy. Prior to its approval in Japan, VILTEPSO was granted with 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 DMD patients.
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 NS Pharma, Inc.
NS Pharma, Inc., is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. For more information,
please visit http://www.nspharma.com. NS Pharma is a registered trademark of the Nippon Shinyaku group of companies.
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NEWS RELEASE
April 14, 2023
NS Pharma Announces FDA Clearance to
Initiate Phase II Study for NS-089/NCNP-02, an Exon 44 Skipping Candidate for the Treatment of Duchenne Muscular Dystrophy
NS Pharma, Inc. (NS Pharma; President, Tsugio Tanaka) is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; President, Toru Nakai)
PARAMUS, NJ: April 14, 2023, – NS Pharma, Inc. announced today the U.S. Food & Drug Administration (FDA) has agreed to the planned Phase II study of NS-089/NCNP- 02 for Duchenne muscular dystrophy. NS-089/NCNP-02 is an investigational candidate for patients with Duchenne muscular dystrophy amenable to exon 44 skipping therapy.
“Progress has been made in the treatment of Duchenne, but patients and families need new and more treatment options,” said Vamshi Rao, MD, Ann & Robert H. Lurie Children’s Hospital of Chicago. “There are currently no available antisense treatments that target Duchenne patients amenable to exon 44 skipping therapy, which is why I am excited about this program and the potential advance of effective treatments for Duchenne muscular dystrophy.”
Study efficacy measures will include the expression of dystrophin protein and motor function. Trial details will be made available through ClinicalTrials.gov. Additional information will be provided once the trial is ready to begin enrolling.
“We are pleased to announce FDA’s clearance to proceed with our Phase II clinical trial in our endeavor to help patients with Duchenne amenable to exon 44 skipping therapy,” said Takeshi Seita, Vice President, R&D at NS Pharma, Inc. “We are confident in our exon skipping drug discovery platform and excited about the future potential of our development program.”
In addition to NS-089/NCNP-02, NS Pharma’s parent company, Nippon Shinyaku, has four investigational exon skipping candidates in various stages of preclinical development.
About Duchenne Muscular Dystrophy (Duchenne)
Duchenne is a progressive form of muscular dystrophy that occurs primarily in males.
Duchenne causes progressive weakness and loss of skeletal, cardiac, and pulmonary 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.
About NS Pharma, Inc.
NS Pharma, Inc., is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. For more information, please visit http://www.nspharma.com. NS Pharma is a registered trademark of the Nippon Shinyaku group of companies.
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Results of a 4-Year Viltolarsen Extension Study of Functional and Safety Outcomes
March 19, 2023
VILTEPSO® (viltolarsen) injection Open Label Extension Clinical Trial Data Scheduled for
Presentation at the MDA Clinical & Scientific Conference 2023
Data were from a final four-year analysis (up to Week 216) of the open-label extension trial of a VILTEPSO Phase 2 study
NS Pharma, Inc. (NS Pharma; President, Tsugio Tanaka), is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; President, Dr. Toru Nakai)
Paramus, NJ: March 19, 2023 – NS Pharma is pleased to announce participation in the Muscular Dystrophy Association’s (MDA) Clinical & Scientific Congress 2023 being held in Dallas, Texas.
The company will be presenting previously reported long-term efficacy and safety data (final analysis up to Week 216) from the open-label extension of a Phase 2 study of VILTEPSO® (viltolarsen).
The presentation will be given by Edward Smith, MD, Duke University School of Medicine. For more information, please visit the MDA Congress website to view the abstract: https://www.mdaconference.org/abstract-library/results-of-a-4-year-viltolarsen-extension-study-of- functional-and-safety-outcomes/
“There have been amazing scientific advances in the treatment of Duchenne, which is why it is important to keep the community informed about existing therapeutic options and the long-term evidence these treatments have generated,” said Dr. Smith. “The Muscular Dystrophy Association is one of the premier advocacy groups supporting the Duchenne community and I am pleased to take part in this year’s knowledge sharing at the organization’s Clinical & Scientific Congress on behalf of NS Pharma.”
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 DMD who are amenable to exon 53 skipping therapy. Prior to its approval in Japan, VILTEPSO was granted with 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 DMD patients.
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 NS Pharma, Inc.
NS Pharma, Inc., is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. For more information, please visit http://www.nspharma.com. NS Pharma is a registered trademark of the Nippon Shinyaku group of companies.
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NEWS RELEASE
October 24, 2022
NS Pharma Reports on Results of VILTEPSO® (viltolarsen) injection After Four Years of Treatment in Open-Label Extension Trial in Duchenne Muscular Dystrophy
Results were from a final analysis (up to Week 216) of the open-label extension trial of a VILTEPSO Phase 2 study.
In the study, the primary endpoint of Time to Stand and secondary endpoints, including Time to Run/Walk and Time to Climb 4 Stairs, were evaluated in comparison to a group-matched DMD historical control.
PARAMUS, NJ: October 24, 2022 – NS Pharma, Inc. (NS Pharma; President, Tsugio Tanaka), a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; President, Toru Nakai), announced today that long-term efficacy and safety data (final analysis up to Week 216) from the open-label extension of a Phase 2 study of VILTEPSO® (viltolarsen) injection were presented at the 27th International Hybrid Annual Congress of the World Muscle Society (WMS 2022) in Halifax, Nova Scotia, Canada.
“These data represent the longest clinical experience of an exon 53 skipping therapy for the treatment of Duchenne muscular dystrophy. In this four-year study, Viltepso- treated patients maintained motor function over the first two years of treatment and experienced significant delay of disease progression over the following two years, compared with the CINRG DNHS control group which declined over this same time period,” said Leslie Magnus, MD, Vice President, Medical Affairs. “These data are encouraging as NS Pharma continues to evaluate the effect of Viltepso on improving or stabilizing motor function in a Phase 3 study.”
Data presented at WMS 2022 are from an open-label trial (N=16) that is the extension of a previous 24-week Phase 2 trial in North America. All 16 patients aged 4 to <10 years with DMD amenable to exon 53 skipping in the 24-week study elected to enroll in this long-term trial to continue evaluation of motor function and safety. Assessments of timed function tests (Time to Stand, Time to Run/Walk, Time to Climb 4 Stairs) were compared to a group-matched DMD historical control drawn from the Cooperative International Neuromuscular Research Group Duchenne Natural History Study (CINRG DNHS). Both groups received a stable dose of glucocorticoid treatment. Efficacy results at Week 205 significantly favored viltolarsen compared to the CINRG DNHS control group for the primary endpoint of mean change from baseline for Time to Stand (viltolarsen 2.7 seconds vs. 8.3 seconds for CINRG DNHS, p=0.0040), and secondary endpoints including Time to Run/Walk 10 meters (viltolarsen 2.0 seconds vs. 6.0 seconds for CINRG DNHS, p=0.0002), and Time to Climb 4 Stairs (viltolarsen -0.01 m/s vs. -0.13 m/s for CINRG DNHS, p=0.0088).
The most frequently reported adverse events were mild to moderate in this 216-week open-label extension period and included cough, nasopharyngitis, rash, pyrexia, and vomiting. This safety profile was similar to that seen in the previous short-term study. There were no treatment-related serious adverse events and no treatment discontinuations.
“This long-term study gives physicians who treat patients with DMD important information about the long-term impact of VILTEPSO on ameliorating the disease course,” said Paula Clemens, MD, from the University of Pittsburgh School of Medicine. “Further study is ongoing, but these four-year data give confidence that VILTEPSO can be considered an important part of the treatment strategy for patients with DMD whose dystrophin mutations are amenable to exon 53 skipping therapy.”
In addition to this Phase 2 open-label extension study, NS Pharma continues to investigate the efficacy and safety of VILTEPSO in the confirmatory Phase 3 RACER53 trial. This study was initiated in October 2019 and is currently enrolling patients. The purpose of this Phase 3 randomized, double-blind, placebo-controlled trial is to evaluate the efficacy of viltolarsen on functional motor endpoints compared to placebo in patients with DMD amenable to exon 53 skipping.
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 DMD who are amenable to exon 53 skipping therapy. Prior to its approval in Japan, VILTEPSO was granted with 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 DMD patients.
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 NS Pharma, Inc.
NS Pharma, Inc., is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. For more information, please visit http://www.nspharma.com. NS Pharma is a registered trademark of the Nippon Shinyaku group of companies.
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NEWS RELEASE
September 17, 2021
VILTEPSO® (viltolarsen) injection Interim Long-Term Clinical Trial Data Scheduled for Presentation at the World Muscle Society 2021 Virtual Conference
PARAMUS, NJ: September 17, 2021 – NS Pharma, Inc. (NS Pharma; President, Tsugio Tanaka), a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; President, Toru Nakai), announced today an electronic poster presentation on long-term efficacy and safety data (interim analysis at 109 weeks) from the open-label extension (up to 192 weeks) of a Phase 2 study of VILTEPSO® (viltolarsen) injection at the World Muscle Society 2021 Virtual Conference being held September 20–24.
The electronic poster presentation will be given by Paula Clemens, MD from the University of Pittsburgh School of Medicine on Thursday, September 23rd from 16:30 to 18:30 BST. For more information, please visit the World Muscle Society Virtual Conference website to view the full program: https://www.wms2021.com/page/programme.
About VILTEPSO® (viltolarsen) injection
Prior to its approval in the U.S., 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 DMD who are amenable to exon 53 skipping therapy. Prior to its approval in Japan, VILTEPSO was granted with 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
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.
About NS Pharma, Inc.
NS Pharma, Inc., is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. For more information, please visit http://www.nspharma.com. NS Pharma is a registered trademark of the Nippon Shinyaku group of companies.
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NEWS RELEASE
April 1, 2021
NS Pharma’s VILTEPSO® (viltolarsen) Receives Permanent J-code from Centers for Medicare and Medicaid Services
PARAMUS, NJ: April 1, 2021 – NS Pharma, Inc. (NS Pharma; President, Tsugio Tanaka), a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; President, Shigenobu Maekawa), announced that the J-code assigned by the Centers for Medicare and Medicaid Services (CMS) for VILTEPSO® (viltolarsen) injection is effective today.
As of April 1, 2021, healthcare providers may use the Level II HCPCS code: J1427 (Injection, Viltolarsen, 10 mg) when submitting claims for VILTEPSO. J-codes are unique identifiers used by commercial insurance plans, Medicare, Medicaid and other government insurance programs. The assignment of a permanent J-code for VILTEPSO will help to streamline billing and reimbursement for home infusion providers, hospitals, outpatient treatment centers and physicians’ offices.
“The designation of a permanent J-code for VILTEPSO is good news for families because simplifying billing and payment enables improved patient access,” said Tsugio Tanaka, President, NS Pharma, Inc. “We encourage families to contact our NS Support team who can help guide you through the access process and advocate on your behalf.”
VILTEPSO is commercially available through a network of specialty distributors and specialty pharmacy providers. The distribution partners in this limited network were selected for their experience providing responsive, reliable service to healthcare providers and families.
About NS Support
NS Support provides comprehensive, personalized access services and care coordination for providers and patients. By enrolling in NS Support, patients and providers receive customized resources and ongoing, highly-responsive assistance every step of the way.
About NS Pharma, Inc.
NS Pharma, Inc., is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. For more information, please visit http://www.nspharma.com. NS Pharma is a registered trademark of the Nippon Shinyaku group of companies.
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NEWS RELEASE
August 12, 2020
NS Pharma’s VILTEPSO™ (viltolarsen) injection Now FDA-Approved in the U.S. for the Treatment of Duchenne Muscular Dystrophy in Patients Amenable to Exon 53 Skipping Therapy
Patients taking VILTEPSO showed an increase in dystrophin expression to an average of 5.9% of normal after 20-24 weeks of treatment.
Overall, in a pivotal study of VILTEPSO 100% of patients showed an increase in dystrophin levels after treatment and 88% of patients showed dystrophin levels of 3% of normal or greater.
PARAMUS, NJ: August 12, 2020 – NS Pharma, Inc. (NS Pharma; President, Tsugio Tanaka), a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; President Shigenobu Maekawa), announced today that the U.S. Food & Drug Administration (FDA) has approved VILTEPSO™ (viltolarsen) injection for patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 53 skipping therapy. VILTEPSO received an Accelerated Approval by the FDA based on an increase in dystrophin, a key protein for supporting muscle health. Since a lack of dystrophin is the underlying cause of DMD, increasing dystrophin as much and as early as possible is a key goal in the treatment of DMD. VILTEPSO is the first and only exon 53 skipping therapy to demonstrate an increase in dystrophin in children as young as four years old. The continued approval of VILTEPSO may be contingent on confirmation of a clinical benefit in a Phase 3 confirmatory trial.
DMD is caused by genetic mutations that prevent dystrophin production. Patients with DMD experience progressive and irreversible muscle loss with symptoms appearing as early as two years of age. Cardiac and respiratory muscle problems begin in the teenage years and lead to serious, life-threatening complications.
The VILTEPSO New Drug Application (NDA) submission included results from a Phase 2, two-period study in patients aged four to less than 10 years of age conducted in North America (Study 1, N=16) and a multicenter, open-label study in boys five to less than 18 years of age conducted in Japan (Study 2, N=16).
In Study 1, of those patients who received the recommended dose of 80 mg/kg/wk (N=8), 100% of patients (8/8) showed an increase in dystrophin levels after treatment with VILTEPSO and 88% of patients (7/8) showed dystrophin levels of 3% or greater than normal. Overall, after 20-24 weeks of treatment a mean increase in dystrophin expression to nearly 6% of normal was observed with VILTEPSO (80 mg/kg/wk) versus 0.6% at baseline.
The most common side effects of VILTEPSO included upper respiratory tract infection, injection site reaction, cough and fever.
“For decades, neurologists who treat DMD have hoped for the discovery of therapies capable of significantly improving dystrophin production, and the magnitude of dystrophin increases observed with VILTEPSO are impressive,” said study investigator Vamshi Rao, MD, Ann & Robert H. Lurie Children’s Hospital of Chicago. “The approval of VILTEPSO is an exciting development for DMD patients amenable to exon 53 skipping therapy and may rapidly become a foundational treatment for these patients.”
Patients receiving treatment with VILTEPSO have the option and flexibility to receive infusions at their home or at a hospital or treatment center. VILTEPSO is administered by a trained healthcare professional as an 80 mg per kg of body weight 60-minute weekly intravenous infusion.
NS Pharma will provide families, physicians and healthcare professionals dedicated and individualized resources every step of the way through the NS Support program. NS Pharma will be hosting a series of webinars on the comprehensive care coordination available through NS Support. Follow us on LinkedIn and Twitter for information and registration for upcoming webinars.
“On behalf of NS Pharma and Nippon Shinyaku, I would like to express our deepest gratitude to the families and physicians who participated in our clinical trials and made today’s approval possible,” said Tsugio Tanaka, President, NS Pharma, Inc. “We are proud to now offer an important new treatment option to help address the significant unmet needs caused by this devastating disease.”
NS Pharma continues to study the safety and efficacy of VILTEPSO in the confirmatory Phase 3 RACER53 trial. This study was initiated in October 2019 and is currently enrolling. The purpose of this Phase 3 trial is to confirm the clinical findings that were submitted under the Accelerated Approval pathway.
About Duchenne Muscular Dystrophy (DMD)
DMD is a progressive form of muscular dystrophy that occurs primarily in males. DMD causes progressive weakness and loss of skeletal, cardiac, and pulmonary 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 DMD may require the use of a wheelchair. Cardiac and respiratory muscle problems begin in the teenage years and lead to serious, life-threatening complications.
About VILTEPSO™ (viltolarsen) injection
Prior to its approval in the U.S., 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 DMD who are amenable to exon 53 skipping therapy. Prior to its approval in Japan, VILTEPSO was granted with 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
For additional safety information, please see the full Prescribing Information.
About NS Pharma, Inc.
NS Pharma, Inc., is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. For more information, please visit http://www.nspharma.com. NS Pharma is a registered trademark of the Nippon Shinyaku group of companies.
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