July 27, 2018

Mitsubishi Tanabe Pharma’s MT-7117 Receives
U.S. FDA Fast Track Designation
for the Investigational Treatment of Patients with theUltra-Rare Disease, Erythropoietic Protoporphyria
Mitsubishi Tanabe Pharma Corporation (MTPC) (Head Office: Osaka; President &Representative Director, CEO: Masayuki Mitsuka), announces that the U.S. Food andDrug Administration (FDA) has granted Fast Track designation for its investigational
drug coded MT-7117 for the prevention of phototoxicity in patients witherythropoietic protoporphyria (EPP1). Mitsubishi Tanabe Pharma Development
America, Inc. (MTDA) and MTPC Group companies have initiated the enrollment ofthe Phase 2 “ENDEAVOR” trial for MT-7117 in the United States.
“The FDA has established significant challenging criteria in order to meet thestandard required for the Fast Track designation2. This designation allows theMT-7117 Development Team to have increased dialogue with the FDA regarding theclinical program at key points in time, which could lead to a priority review of theNew Drug Application depending on the outcome of our clinical trials,” said Douglas
N. Dobak, Vice President and Head of Regulatory Affairs, MTDA.
Erythropoietic Protophyria (EPP) is an inherited disorder of the heme metabolic
pathway that results from mutations of the ferrochelatase (FECH) gene or, lesscommonly, the aminolevulinic acid synthase-2 (ALAS2) gene. EPP is characterized
by accumulation of protoporphyrin in blood, erythrocytes and tissues and cutaneous
photosensitivity that usually manifests in early infancy during initial exposure to sun.
Photosensitivity is the main symptom of EPP and exposure of skin to sunlight resultsin acute cutaneous symptoms such as stinging, burning and severe pain leading tothe development of erythema, cutaneous edema and petechiae with prolonged
exposure to sunlight. Photosensitivity has a significant deleterious impact on thequality of life of EPP patients, leading to chronic avoidance of both long-wave
radiation and visible light and a resultant profound decrease of social activity. EPP isa lifelong disorder whose prognosis depends on the evolution of the hepatic disease;in addition, approximately 3 percent of all patients with EPP develop liver pathology.
MTDA has started the “ENDEAVOR” trial in the United State. The ENDEAVOR trial isa Phase 2, multicenter, randomized, double-blind, placebo-controlled study to1/2

evaluate efficacy, safety, and tolerability of MT-7117 in subjects with EPP; The study
will enroll approximately 100 subjects and consists of a 2-week screening period, a16-week double-blind treatment period, and a 6-week follow up period.
MTPC Group will work hard to ensure that our research will substantially contributeto delivering the best possible pharmaceutical products as quickly as possible tomany patients fighting against severe diseases all over the world.

Mitsubishi Tanabe Pharma Corporation
Corporate Communications Department
Media contacts: TEL:+81 6 6205 5119
Investor contacts: TEL:+81 6 6205 5110

1.

NIH website: https://rarediseases.info.nih.gov/diseases/4527/erythropoietic-protoporphyria.

2.

US Food and Drug Administration. Fast Track Information Page:
https://www.fda.gov/ForPatients/Approvals/Fast/ucm405399.htm■ Mitsubishi Tanabe Pharma Development America, Inc.
The U.S. headquarters of Mitsubishi Tanabe Pharma Development America, Inc.
(MTDA) is located in Jersey City, New Jersey. MTDA is a wholly-owned subsidiary of
Mitsubishi Tanabe Pharma Corporation’s 100 percent-owned U.S. holding company,
Mitsubishi Tanabe Pharma Holdings America, Inc.
of Radicava®

MTDA has obtained the approval

the new treatment option for ALS in more than 20 years in the ALS

therapeutic area in the United States. MTDA is dedicated to research and develop
innovative pharmaceutical products that address the unmet medical needs ofpatients. http://mt-pharma-development-america.com/2/2