Altemia is being developed by Sancilio Pharmaceuticals as an oral, once-daily formulation for children aged 5 to 17 years who have SCD. The investigational drug consists of a mixture of lipids encapsulated in a small, soft gelatin capsule created to reduce vaso-occlusion (VOC) episodes, anemia, organ damage and other SCD-related complications.
Altemia was designed to restore balance and fluidity to red blood cells and other cells affected by the disease. According to Sancilio, Altemia has the potential to address the inflammatory symptoms of SCD and to assist in reducing sickle cell events in general. By minimizing damage, Altemia may be able to reduce SCD-related crises and mortality.
The FDA’s Rare Pediatric Disease designation provides incentives to support pharmaceutical companies that are developing drugs and biologics for rare diseases. The federal agency defines a rare pediatric disease as a serious/life-threatening disease that primarily affects individuals younger than 18 years and that impacts fewer than 200,000 individuals in the U.S.
“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,” Frederick Sancilio, president and CEO, said in a press release. “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 SCOT trial was initiated in December 2016 as a double-blind, randomized, placebo-controlled study enrolling about 66 SCD patients aged between 15 and 17 years old. Currently underway in sickle cell medical centers throughout the U.S., the study is designed to confirm a rapid uptake of certain fatty acids into red blood cells depleted as a result of SCD.
The study is measuring replenishment of cell membranes as its primary goal, as well as reduction or elimination of anemia and red cell destruction. Other measures include the level of inflammation and clotting factors linked to VOC crisis.
Preliminary results of the SCOT trial should be announced soon.