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Synageva
BioPharma Corp. (Synageva)
GEVA, a biopharmaceutical
company developing therapeutic products for rare diseases, today
announced that the U.S. Food and Drug Administration (FDA) granted
Breakthrough Therapy designation to sebelipase alfa for the treatment of
early onset lysosomal acid lipase deficiency (LAL Deficiency), also
known as Wolman disease. The designation was based on data generated
from clinical trials with sebelipase alfa in patients with early onset
LAL Deficiency. The FDA also confirmed that late onset LAL Deficiency is
“a serious and life threatening disease or condition” and that
Breakthrough Therapy designation could be obtained for this aspect of
the disease with additional clinical information.
According to the FDA,
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