NEWS RELEASE
August 20, 2020

VILTEPSO™ (viltolarsen) injection
Now Commercially Available in the U.S.
KYOTO, Japan August 20th, 2020 – Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; Kyoto:President, Shigenobu Maekawa) announced today that NS Pharma, Inc. (Paramus, NJ;
President, Tsugio Tanaka), a wholly owned subsidiary of Nippon Shinyaku madeVILTEPSO™ (viltolarsen) now available for commercial sales in the United States market asof August 19 (EST).
NS Pharma received marketing authorization for VILTEPSO under an accelerated approval
pathway from the U.S. Food & Drug Administration (FDA) on August 12 (EST) for thetreatment of Duchenne Muscular Dystrophy (DMD) amenable to exon 53 skipping therapy.
To help navigate access to and reimbursement of VILTEPSO, NS Pharma is offering services
and resources to patients, caregivers and their healthcare providers through the NS
Support™ program.
Nippon Shinyaku and NS Pharma will contribute to the treatment of patients with DMD byassisting each patient in obtaining the access to VILTEPSO.
<VILTEPSO>VILTEPSO is an antisense oligonucleotide 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 basedon an increase in dystrophin production in skeletal muscle observed in patients treated with
VILTEPSO. Continued approval for this indication may be contingent upon verification anddescription of clinical benefit in a confirmatory trial. VILTEPSO received marketing
authorization under an accelerated approval pathway in Japan in March 2020 and becamecommercially available in Japan in May this year. Currently, a phase 3 study (RACER53study) is ongoing in approximately 30 centers worldwide as a confirmatory study.
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< 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. There are many types of genetic mutations that can cause DMD. VILTEPSO
is indicated for patients with DMD mutations that are amenable to exon 53 skipping.
< 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. Yourdoctor 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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