Synageva BioPharma Corp. recently announced that it has received breakthrough therapy designation from the US Food and Drug Administration (FDA) for sebelipase alfa.
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 Pharmacyclics, Inc. and Novartis AG (NVS - Analyst Report) 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, Santarus Inc. in the pharma space is well positioned with aZacks Rank #1 (Strong Buy).