FDA Accepts BLA Filing For Synageva's Kanuma™ (Sebelipase Alfa); Grants Priority Review And Assigns PDUFA Date

Feb 23, 2015, 08:00 ET from Synageva BioPharma Corp.

LEXINGTON, Mass., Feb. 23, 2015 /PRNewswire/ -- Synageva BioPharma Corp. (NASDAQ: GEVA), a biopharmaceutical company developing therapeutic products for rare disorders, announced today that the U.S. Food and Drug Administration (FDA) has accepted for review the Biologics License Application (BLA) for Kanuma™ (sebelipase alfa) for the treatment of lysosomal acid lipase deficiency (LAL Deficiency).  The FDA granted the company's request for Priority Review, which shortens the regulatory review period and is reserved for investigational therapies that treat serious conditions and, if approved, would provide a significant improvement in safety or effectiveness compared to available therapies.  The FDA established a target action date of September 8, 2015 under the Prescription Drug User Fee Act (PDUFA). 

The BLA for Kanuma included previously reported data from the global, randomized, double-blind, placebo controlled Phase 3 trial in children and adults with LAL Deficiency, and the Phase 2/3 trial of Kanuma in infants with LAL Deficiency.  Patients in these trials, combined with patients in other ongoing clinical trials with Kanuma, represent the largest patient population studied to date for this rare, devastating disease.

Kanuma and LAL Deficiency

LAL Deficiency is a serious and life-threatening disease that can be diagnosed with a simple blood test.  LAL Deficiency causes progressive and multisystemic organ damage including cirrhosis and accelerated atherosclerosis that can lead to sudden and unpredictable clinical complications.  LAL Deficiency often manifests in childhood but can be diagnosed at all ages.  LAL Deficiency is caused by genetic mutations that result in decreased LAL enzyme activity in the lysosomes across multiple body tissues, leading to the buildup of fatty material in the liver, blood vessel walls and other tissues.

Kanuma is a recombinant form of the human LAL enzyme being developed by Synageva as an enzyme replacement therapy for LAL Deficiency.  Kanuma has been granted orphan 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 BLA for Kanuma and granted the company's request for Priority Review.  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 initiated a Phase 1/2 trial with its second, first-mover program, SBC-103, an enzyme replacement therapy for patients with mucopolysaccharidosis IIIB (MPS IIIB, also known as Sanfilippo B syndrome).  The company's third, first-mover program, SBC-105, is an enzyme replacement therapy in preclinical development for rare disorders of calcification, including generalized calcification in infants (GACI). 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.

Further information regarding Synageva is available at www.synageva.com.

Medical information regarding Kanuma and LAL Deficiency is available by email at medinfo@synageva.com.

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.   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, risk that the outcomes of our clinical trials may not support registration or further development of our product candidates due to safety, efficacy or other reasons, the content and timing of decisions by the FDA and other regulatory authorities, and the risks identified under the heading "Risk Factors" in the Company's prospectus supplement filed with the Securities and Exchange Commission (SEC) on January 7, 2015 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 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.

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SOURCE Synageva BioPharma Corp.



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