Sancilio Pharma awarded rare pediatric status for sickle cell candidate

The US Food and Drug Administration has granted Rare Pediatric Disease designation to Altemia Soft Gelatin Capsules for the treatment of sickle cell disease (SCD) in children. The drug is being developed by USA-based Sancilio Pharmaceuticals (SPCI).

Altemia, a leading development program for SPCI, is being developed as an oral, once-a-day formulation for children between the ages of 5 – 17 with SCD. Altemia consists of a complex proprietary mixture of various fatty acids, primarily in the form of Ethyl Cervonate (SPCI’s proprietary blend of docosahexaenoic acid and other omega-3 fatty acids), and surface active agents formulated using ALT specifically to address the treatment of SCD.

"The FDA's Rare Pediatric Disease designation for Altemia, in addition to the previously granted Orphan Drug designation, further stresses the agency's recognition that the SCD community faces a critical need for new treatments like Altemia," said Dr Frederick Sancilio, president and chief executive of SPCI. "We continue to make important progress in our clinical trial; a randomized, double-blind, placebo-controlled, multicenter study to evaluate the efficacy and safety of Altemia in pediatric patients. We look forward to reporting top-line results from the study early in the fourth quarter, this year," the FDA added.