Synageva BioPharma Corp.
) recently announced that it has received breakthrough therapy
designation from the US Food and Drug Administration (FDA) for
Synageva is developing sebelipase alfa for the treatment of
early onset lysosomal acid lipase deficiency (LAL deficiency),
also known as Wolman disease. The candidate is currently in a
phase II/III study.
The designation, which was enacted as part of the 2012 Food
and Drug Administration Safety and Innovation Act, is granted to
potential new treatments for serious or life-threatening diseases
or conditions where the initial clinical data shows that the
treatment has the potential to demonstrate substantial
improvement on one or more clinically significant endpoints
compared to existing treatments. The designation should help
fasten the development and review process for the candidate.
Apart from Synageva, companies like
) have also recently received breakthrough therapy designation
for their candidates.
Pharmacyclicswas granted breakthrough therapy designation for
ibrutinib for treating chronic lymphocytic leukemia / small
lymphocytic lymphoma patients with a deletion of the short arm of
chromosome 17p. Earlier in the year, the US regulatory body had
granted breakthrough therapy designation to ibrutinib for
treating mantle cell lymphoma patients as a monotherapy. Novartis
was granted breakthrough therapy designation for its pipeline
candidate LDK378, which is being developed for the treatment of
patients with anaplastic lymphoma kinase positive (ALK+)
metastatic non-small cell lung cancer (NSCLC).
Synageva carries a Zacks Rank #3 (Hold). Currently,
) in the pharma space is well positioned with aZacks Rank #1
SYNAGEVA BIOPHM (GEVA): Free Stock Analysis
NOVARTIS AG-ADR (NVS): Free Stock Analysis
PHARMACYCLICS (PCYC): Free Stock Analysis
SANTARUS INC (SNTS): Free Stock Analysis
To read this article on Zacks.com click here.