NEWS RELEASE
March 25, 2020

Marketing authorization in Japan of VILTEPSO® Intravenous Infusion 250 mg
for the treatment of Duchenne muscular dystrophy
KYOTO, Japan Mar. 25, 2020 – Nippon Shinyaku Co., Ltd. (President, Shigenobu
Maekawa) announced today that the Ministry of Health, Labour and Welfare (MHLW) has
approved VILTEPSO® Intravenous Infusion 250 mg (viltolarsen, previously NS-065/NCNP01) for the treatment of patients with Duchenne Muscular Dystrophy (DMD) who areamenable to exon 53 skipping therapy. This represents the first regulatory approval forviltolarsen in the world and the first approved DMD treatment other than steroids in Japan.
DMD is a progressive form of muscular dystrophy that occurs primarily in males. DMD causes
progressive weakness and loss (atrophy) of skeletal, cardiac, and pulmonary muscles due
to a deficiency of normal dystrophin, a protein involved in constructing the framework ofmuscle cells.
VILTEPSO® Intravenous Infusion 250 mg is a morpholino antisense oligonucleotide, which
was co-discovered by Nippon Shinyaku and National Center of Neurology and Psychiatry(NCNP: Kodaira City, Tokyo; President, Hidehiro Mizusawa, Executive Director, Shin’ichi
Takeda). It is designed to increase dystrophin production by binding to exon 53, an exon
next to the dystrophin gene deletion which results in the translation of the mRNA into a
shortened dystrophin protein that contains essential functional portions. In Japan,
VILTEPSO® Intravenous Infusion 250 mg was granted SAKIGAKE designation of MHLW inOctober 2015, Orphan drug designation in August in 2019, and designation of Conditional
Early Approval System in October in 2019. In the U.S, viltolarsen was granted a Rare
Pediatric Disease Designation, Orphan Drug Designation, and a Fast Track Designation.
Viltolarsen has not yet been approved in the U.S.
Nippon Shinyaku continues to focus their efforts on developing treatments for intractable andrare diseases, including DMD.

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Summary of VILTEPSO
Brand Name

VILTEPSO® Intravenous Infusion 250 mg

Generic Name

Viltolarsen

Dosage Forms and Strengths

Viltolarsen 250 mg/5 mL (50mg/mL) in a single-dose vial

Indications and Usage

DMD patients who have a confirmed mutation of the DMD
gene that is amenable to exon 53 skipping

Dosage and administration

80 mg/kg of viltolarsen is administered intravenously oncea week over 1 hour

<Viltolarsen>
Nippon Shinyaku conducted a phase 1/2 study and submitted NDA in September 2019. TheNorth America P2 clinical study was conducted by NS Pharma, Inc. (President, Tsugio
Tanaka), a wholly owned subsidiary of Nippon Shinyaku. Viltolarsen was granted Rare
Pediatric Disease Designation, Orphan Drug Designation, and Fast Track designation in theU.S. Its New Drug Application (NDA) was granted Priority Review by the FDA with ananticipated action date in the third quarter of 2020. While viltolarsen remains under review
by the FDA, NS Pharma continues to study the efficacy and safety of viltolarsen in the Phase
3 RACER53 trial initiated. To meet our commitment to the FDA and the DMD community, we
initiated our confirmatory Phase 3 trial shortly after the completion of our NDA submission tothe FDA.
Below are designations in Japan only.
<SAKIGAKE designation> (Japanese version of Breakthrough Therapy designation)
The designation is to promote Research and Development in Japan aiming at early practicalapplication for world’s first, domestically-produced and innovative pharmaceutical products
for serious and life-threatening diseases. This is aiming to shorten reviewing time for approval,
facilitating a prioritized consultation and review for regulatory approval.

<Orphan Disease designation>
Orphan drug is defined as the medicine of which medical needs are particularly high for adisease of which patient number is less than 50,000 in Japan.

<Conditional Early Approval System>“Conditional Early Approval System” is a system to put highly useful and effective drugs fortreating serious diseases into practical use as early as possible.
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Approval of applicable pharmaceutical products should be granted under a condition
requiring the applicant to conduct post-marketing surveys or other studies that arenecessary to re-confirm the efficacy and safety of the product. The system must be
subjected to the following conditions.
1. For severe diseases
2. Apparent improvement of medical care
3. Confirmatory clinical trials is difficult to conduct, or don’t have sufficient feasibility.
4. Confirmation of a certain degree of efficacy and safety through clinical trials otherthan confirmatory clinical trials.

Contact (Japan):Corporate Communications Dept., Nippon Shinyaku Co., Ltd.
FAX: +81-75-321-9128
U.S. Media Contact:
media@nspharma.com

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