KIACTA™ Granted Orphan Drug Status in Japan

- Japanese drug development grant also received for KIACTA™ -

LAVAL, QC, July 23, 2013 /CNW/ - BELLUS Health Inc. (TSX: BLU) ("BELLUS Health" or the "Company") announced today that the Ministry of Health, Labour and Welfare of Japan has granted Orphan Disease Drug Status in Japan for KIACTA™, a novel drug candidate currently in a Phase III Confirmatory Study for the treatment of AA amyloidosis.

The Company also announced today that the National Institute of Biomedical Innovation (NIBIO), a Japanese government research organization, has granted up to the equivalent of C$500,000 towards KIACTA™ clinical development expenses incurred in Japan. Five clinical sites in Japan are currently participating in the global Phase III Confirmatory Study for KIACTA™.

"Obtaining orphan drug status in Japan is an important milestone in advancing the clinical and commercial strategy for KIACTA™," said Roberto Bellini, President and Chief Executive Officer of BELLUS Health. "The orphan designation and grant highlight KIACTA™'s potential as an effective treatment for AA amyloidosis. Japan becomes the fourth jurisdiction where KIACTA™ has attained orphan drug designation, underscoring the unmet medical need for such a product and validating the development plan of the ongoing Phase III Confirmatory Study."

KIACTA™ has previously received Orphan Disease Drug Status designation in the United States and Orphan Medicinal Product designation in Europe.  In addition, KIACTA™ received Orphan Drug Designation in Switzerland.

KIACTA™ is a novel drug candidate currently in development for the treatment of AA amyloidosis, a condition resulting in renal dysfunction that often rapidly leads to dialysis and death. KIACTA™ is partnered with global private equity firm Auven Therapeutics. AA amyloidosis affects approximately 35,000 to 50,000 individuals in the United States, Europe and Japan.

As a result of the designation in Japan, KIACTA™ will receive priority review from Japanese regulatory authorities as a New Drug Application, once it is submitted. In addition, the drug will be granted 10 years of market exclusivity as a treatment for patients with AA amyloidosis.

In order to attain Orphan Disease Drug Status in Japan, a product has to meet specific criteria, namely:

  • There are fewer than 50,000 patients suffering from the disease
  • There is no equivalent drug or therapy on the market or, if similar therapies exist, the efficacy of the new drug must offer significant benefits in terms of safety and/ or efficacy
  • There must be a logical scientific basis for use of the drug
  • There must be an appropriate development plan in place with a reasonable likelihood of success

About BELLUS Health (www.bellushealth.com)

BELLUS Health is a development-focused healthcare company concentrating on products that provide innovative health solutions and address critical unmet medical needs. The Company's lead program is KIACTA™, a novel drug candidate currently in a Phase III Confirmatory Study for the treatment of AA amyloidosis, an orphan indication resulting in renal dysfunction that often rapidly leads to dialysis and death. KIACTA™ is partnered with global private equity firm Auven Therapeutics. AA amyloidosis affects approximately 35,000 to 50,000 individuals in the United States, Europe and Japan.

SOURCE: BELLUS Health Inc.

For further information:

Adam Peeler
TMX Equicom
416-815-0700 ext. 225 | apeeler@tmxequicom.com


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