Synageva BioPharma Submits Kanuma™ (Sebelipase Alfa) Application For LAL Deficiency In Japan

LEXINGTON, Mass., May 26, 2015 /PRNewswire/ -- Synageva BioPharma Corp. (NASDAQ: GEVA), a biopharmaceutical company developing therapeutic products for rare disorders, today announced the submission of a New Drug Application (NDA) to the Ministry of Health, Labour and Welfare (MHLW) in Japan for Kanuma™ (sebelipase alfa) as a treatment for patients with lysosomal acid lipase deficiency (LAL Deficiency). Upon approval, Kanuma would be the first approved therapy for these patients. 

The NDA for Kanuma included previously reported data from the global, randomized, double-blind, placebo controlled Phase 3 trial in children and adults with LAL Deficiency, which included enrollment of Japanese patients, and the Phase 2/3 trial of Kanuma in infants with LAL Deficiency.  The MHLW previously granted Orphan Drug Designation (ODD) to sebelipase alfa for the treatment of patients with LAL Deficiency. As a result, this NDA will receive priority review for marketing authorization, and, if approved, sebelipase alfa would have 10 years of market exclusivity for the designated indication.

About LAL Deficiency

LAL Deficiency is a serious, life-threatening disease associated with early mortality and significant morbidity. LAL Deficiency is a chronic disease in which genetic mutations result in decreased activity of the LAL enzyme; this leads to marked accumulation of lipids in vital organs, blood vessels, and other tissues, resulting in progressive and multi-systemic organ damage including fibrosis, cirrhosis, liver failure, accelerated atherosclerosis, cardiovascular disease, and other devastating consequences. LAL Deficiency affects patients of all ages with sudden and unpredictable clinical complications manifesting from infancy through adulthood.  The decreased LAL enzyme activity can be diagnosed with a simple blood test. 

About Kanuma

Kanuma is a recombinant form of the human LAL enzyme being developed by Synageva as an enzyme replacement therapy for patients with LAL Deficiency.  Kanuma has been granted orphan drug designation by the FDA, the European Medicines Agency (EMA), and the Japanese Ministry of Health, Labour and Welfare. Additionally, Kanuma received fast track designation by the FDA, and Breakthrough Therapy designation by the FDA for LAL Deficiency presenting in infants. The FDA accepted for review the Biologics License Application (BLA) for Kanuma, granted the company's request for Priority Review, and established a target action date of September 8, 2015 under the Prescription Drug User Fee Act (PDUFA). The EMA validated the Marketing Authorization Application (MAA) for Kanuma and granted the company's request for accelerated assessment.  

About Synageva

Synageva is a biopharmaceutical company focused on the discovery, development, and commercialization of therapeutic products for patients with rare diseases. The company's pipeline consists of protein therapeutic programs for rare diseases with unmet medical need which are currently at various stages of development.  The company is planning for a global launch of Kanuma for the treatment of LAL Deficiency and is dosing patients in a Phase 1/2 trial with its second, first-mover program, SBC-103 for MPS IIIB.  The company's third, first-mover program, SBC-105, is an enzyme replacement therapy in preclinical development for disorders of calcification.  In addition to these first-mover programs, the pipeline also consists of opportunities that leverage the company's manufacturing platform and other capabilities to create potentially bio-superior treatments for patient populations where there is still unmet medical need.  The company has recently produced enzymes targeting Hunter syndrome, Fabry disease and Pompe disease with expression levels and activity that support further preclinical development.

Synageva routinely posts information that may be important to investors in the Investor Relations section of the company's website at www.synageva.com.  Synageva encourages investors and potential investors to consult this website regularly for important information about the company.

Medical information regarding Kanuma and LAL Deficiency is available by email at [email protected].

Forward-Looking Statements

This news release contains "forward-looking statements".  Such statements generally can be identified by the use of words such as "anticipate," "expect," "plan," "could," "intend," "believe," "may," "will," "estimate," "forecast," "project," or words of similar meaning. These forward-looking statements address, among other matters, potential approval and plans for the potential launch of Kanuma, the ability for the company's manufacturing platform to create potentially bio-superior treatments, and plans to further support preclinical development for a potentially bio-superior program. Many factors may cause actual results to differ materially from forward-looking statements, including inaccurate assumptions and a broad variety of risks and uncertainties, some of which are known, such as, unanticipated costs or delays in the company's research and development programs, risk of delays in completing the company's preclinical and clinical trials, risk that the outcomes of the preclinical or clinical trials may not support registration or further development of the company's product candidates due to safety, efficacy or other reasons, the timing for initiating and completing potential preclinical and clinical studies, the timing for reporting data from potential preclinical and clinical studies, the content and timing of decisions by the FDA, EMA and other regulatory authorities, our ability to prepare for potential commercial launch of Kanuma, the ability to continue to produce favorable product attributes and characteristics expressed using the company's proprietary platform, and the risks identified under the heading "Risk Factors" in the company's annual report on Form 10-Q for the fiscal quarter ending March 31, 2015 filed with the Securities and Exchange Commission (SEC) and other filings Synageva periodically makes with the SEC, and others of which are not known.  Preclinical and clinical trial data are subject to differing interpretations, and regulatory agencies, as well as medical and scientific experts, may not share Synageva's views regarding these data or its implications.  Synageva may encounter problems or delays in preclinical and clinical development and the regulatory process.  No forward-looking statement is a guarantee of future results or events, and investors should avoid placing undue reliance on such statements.  Synageva undertakes no obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise.  Our business is subject to substantial risks and uncertainties, including those referenced above. Investors, potential investors, and others should give careful consideration to these risks and uncertainties.

"Synageva BioPharma™" and "Kanuma™" are trademarks, and "Dedicated to Rare Diseases®" is a registered trademark, of Synageva BioPharma Corp.

SOURCE Synageva BioPharma Corp.

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