Synageva BioPharma Receives 14th FDA Breakthrough Therapy Designation

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Synagea BioPharma, a Lexington, Massachusetts, biopharmaceutical company developing therapies for rare diseases, announces on May 20, 2013, that orphan drug Sebelipase Alfa receives FDA Breakthrough Therapy Designation #14 for the treatment of early onset Lysosomal Acid Lapase Deficiency (LAL Deficiency), also known as Wolman Disease. The FDA also said that late onset LAL Deficiency, a “serious and life threatening disease or condition”, could obtain the Breakthrough Therapy Designation for this aspect of the disease with additional clinical information.

The early onset LAL Deficiency has a devastating impact on infants, who often die within the first six months of life. LAL Deficiency  is a rare autosomal recessive Lysosomal Storage Disorder (LSD), caused by a decrease in LAL enzyme activity. As a result, cells cannot metabolize lipids, leading to fat buildup in the liver and gut, as well as malnutrition. Early onset LAL Deficiency results in infants developing severe malabsorption, growth failure, and liver failure. There are currently no approved therapies for LAL Deficiency. LAL Deficiency affects only 1 out of every 500,000 people.

Sebelipase Alfa (SBC-102) is a recombinant form of the human LAL enzyme  being developed as an Enzyme Replacement Therapy (ERT) for LAL Deficiency. Synageva BioPharma is evaluating Sebelipase Alfa in clinical trials for both early and late onset LAL Deficiency.  Sebelipase Alfa has been granted orphan drug status by the :

•   FDA
•   European Medicines Agency (EMA)
•   Japanese Ministry of Health, Labour, and Welfare.

Sebelipase Alfa has also received FDA Fast Track designation. Synageva BioPharma currently has a Phase III study in late-onset patients and a Phase II/III trial for the early onset in infants.

References

Hide and Seek Foundation

LAL SOLACE (Support Organization for Lysosomal Acid Lipase Deficiency – Advocacy, Care, & Expertise)

Synageva BioPharma Rare Disease Pipeline.

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