NEWS RELEASE
August 13, 2020

VILTEPSOTM (viltolarsen) Now Approved in the U.S.
for the Treatment of Duchenne Muscular Dystrophy
KYOTO, Japan August 13, 2020 – Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; HQ, Kyoto;
President, Shigenobu Maekawa) announced 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.
DMD is a progressive muscle disease that primarily occurs in boys due to a genetic mutationin the dystrophin gene. This prevents the production of normal dystrophin resulting in reduced
muscle strength/function that progressively continues to decline. There are many types ofgenetic mutations that can cause DMD. VILTEPSOTM is indicated for patients with DMD
mutations that are amenable to exon 53 skipping.
A phase 2 clinical study was conducted in North America by NS Pharma, Inc. (Paramus, NJ;
President, Tsugio Tanaka), a wholly owned subsidiary of Nippon Shinyaku. Viltolarsen wasgranted Rare Pediatric Disease Designation, Orphan Drug Designation, and Fast Track
Designation in the US. The FDA reviewed the NDA under an accelerated approval pathway,
based on data from the phase 2 study conducted in North America and the phase 1/2 study
conducted in Japan. Viltolarsen also received Priority Review and has now received
marketing authorization in the US. Currently, a phase 3 study (RACER53 study) is ongoingin approximately 30 centers worldwide as a confirmatory study.
In Japan, VILTEPSO was granted marketing authorization by the Ministry of Health, Labourand Welfare (MHLW) in March 2020 and launched by Nippon Shinyaku in May 2020.
Nippon Shinyaku continues to focus its research efforts on the treatment of DMD in Japanand the U.S. VILTEPSO will be made available in the U.S. and marketed by NS Pharma.

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< Summary of VILTEPSO >Brand Name

VILTEPSOTM (viltolarsen) injection

Generic Name

Viltolarsen

Date of FDA approval

August 12, 2020

Dosage Forms and Strengths

Injection 250 mg/5 mL (50 mg/mL) solution in a single-dosevial

Indications and UsageVILTEPSO is an antisense oligonucleotide indicated for thetreatment of Duchenne muscular dystrophy (DMD) inpatients who have a confirmed mutation of the DMD genethat is amenable to exon 53 skipping.

Dosage and administration

Recommended dosage of VILTEPSO is 80 mg/kg of bodyweight once weekly. Administer as an intravenous infusion
over 60 minutes.

< 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 ofDMD 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 andrespiratory muscle problems begin in the teenage years and lead to serious, life-threatening
complications.
< VILTEPSO (general name: Viltolarsen) >VILTEPSO is antisense oligonucleotide indicated for the treatment of DMD) in patients who
have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping, which
was co-discovered by Nippon Shinyaku and National Center of Neurology and Psychiatry(Kodaira City, Tokyo; President, Hidehiro Mizusawa). VILTEPSO was granted SAKIGAKE
designation, Orphan Drug Designation, and designation of Conditional Early Approval
System in Japan.
< Phase 2 study (201 study) in the U.S. >In this Phase 2 clinical study, that included patients aged four to less than 10 years of age(N=16), 100% of patients (8/8) showed an increase in dystrophin levels after treatment with
VILTEPSO, and 88% of patients (7/8) showed dystrophin level of 3% or greater than normalfor patients taking VILTEPSO 80 mg/kg/wk (n=8).1,2 Overall, after 20-24 weeks of treatmenta mean increase in dystrophin expression to nearly 6% of normal was observed with2/3

VILTEPSO (80 mg/kg/week) versus 0.6% at baseline (primary endpoint).1,2 The mostcommon side effects of VILTEPSO included upper respiratory tract infection, injection site
reaction, cough, and fever.

< References >1. Viltepso [prescribing information]. Paramus, NJ: NS Pharma, Inc.; 2019.
2. Clemens PR, Rao VK, Connolly AM, et al; for the CINRG DNHS Investigators. Safety,tolerability, and efficacy of viltolarsen in boys with Duchenne muscular dystrophy
amenable to exon 53 skipping: a phase 2 randomized clinical trial [published online
ahead of print May 26, 2020. JAMA Neurology. doi:10.1001/jamaneurol.2020.1264.

< 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 trademarkof the Nippon Shinyaku group of companies.

< 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.

The most common side effects of VILTEPSO included upper respiratory tract infection,
injection site reaction, cough and fever.

For more information about VILTEPSO, see Full Prescribing Information.

< Contact >Corporate Communications Dept., Nippon Shinyaku Co., Ltd.: FAX: +81-75-321-9128

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