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Synageva Biopharma Makes New Moves with Sebelipase Alfa for LAL Deficiency

October 29, 2014

Synageva BioPharma Starts Rolling Submission Of A Biologics License Application To The FDA For Sebelipase Alfa

First-Ever Product Submission for LAL Deficiency
Plans to Complete the BLA and MAA by the End of January 2015

 Synageva BioPharma Corp. (Synageva) (NASDAQ: GEVA), a biopharmaceutical company developing therapeutic products for rare disorders, announced today the start of a rolling submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for sebelipase alfa as a treatment for patients with lysosomal acid lipase deficiency (LAL Deficiency), a rare genetic disease with significant morbidity and early mortality.  A rolling submission allows completed portions of the application to be submitted and reviewed by the FDAon an ongoing basis.  The company anticipates completing the rolling submission of the BLA to the FDA and submitting the Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) by the end of January 2015.  The BLA and MAA will include data from the global, randomized, double-blind, placebo controlled Phase 3 trial of sebelipase alfa in children and adults with LAL Deficiency, and the Phase 2/3 trial of sebelipase alfa in infants with LAL Deficiency.

Sebelipase Alfa for LAL Deficiency

LAL Deficiency is a serious, underdiagnosed disease that manifests with significant morbidity and early mortality.  LAL Deficiency causes progressive and multisystemic organ damage including hepatic 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 with a simple blood test.  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.

Sebelipase alfa is a recombinant form of the human LAL enzyme being developed by Synageva as an enzyme replacement therapy for LAL Deficiency.  Sebelipase alfa has been granted orphan designation by the FDA, the EMA, and the Japanese Ministry of Health, Labour and Welfare.  Additionally, sebelipase alfa received fast track designation by the FDA, and Breakthrough Therapy designation by the FDA for LAL Deficiency presenting in infants.

Sebelipase Alfa Phase 3 Data In Children And Adults With LAL Deficiency Selected For Oral Presentation During The Late-Breaking Session At The Liver Meeting®

Synageva BioPharma Corp. (Synageva) (NASDAQ: GEVA), a biopharmaceutical company developing therapeutic products for rare disorders, announced today that data from its Phase 3, global clinical study evaluating sebelipase alfa in children and adults with LAL Deficiency has been selected for oral presentation during the late-breaking session at The Liver Meeting®, the 65th Annual Meeting of theAmerican Association for the Study of Liver Diseases (AASLD), being held November 7-11 in Boston, MA.

The data will be presented during the late-breaking oral session on Monday, November 10, 4:00-4:15 p.m. EST:

  • “Results of a Global Phase 3, Randomized, Double-Blind, Placebo-Controlled Trial Evaluating the Efficacy and Safety of Sebelipase Alfa as an Enzyme Replacement Therapy in Children and Adults with Lysosomal Acid Lipase Deficiency”.  Publication number: LB-5
  • Presenting Author: Manisha Balwani, M.D., Mount Sinai School of Medicine, New York, NY

The abstract supporting the late-breaking oral presentation is now available on the AASLD website at: https://aasld2014.abstractcentral.com/planner.jsp.

About Sebelipase Alfa for LAL Deficiency

Lysosomal Acid Lipase Deficiency (LAL D) is a life-threatening, underdiagnosed disease that manifests with significant morbidity and early mortality.  LAL D causes progressive and multisystemic organ damage including hepatic cirrhosis and accelerated atherosclerosis that can lead to sudden and unpredictable clinical complications.  LAL D often manifests in childhood but can be diagnosed at all ages with a simple blood test.  LAL D 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.  LAL D was historically called Cholesteryl Ester Storage Disease in children and adults, and Wolman disease in infants.

 

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

 

 

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