Sancilio Gets Rare Pediatric Disease Designation for Sickle Cell Disease Treatment

Mathew Shanley

Sancilio Pharmaceuticals Company, Inc. (SPCI) announced today that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation to Altemia Soft Gelatin Capsules for the treatment of sickle cell disease (SCD) in children.

The designation comes with a bit of good timing, as September is National Sickle Cell Awareness Month. The awareness effort was first officially recognized by the federal government in 1983.

Altemia, a leading development program for SPCI, is an in-development oral, once-a-day formulation intended for children between the ages of 5-17 with SCD.

SCD is a rare genetic condition, and characterized by red blood cells that are hard, sticky, and C-shaped (like the farm tool the "sickle"). These cells clog smaller blood vessels, and result in pain, increased risk for infection, acute chest syndrome, and stroke.

SCD affects 90,000 to 100,000 Americans, most of whom are African Americans, and current treatment options are limited to reducing painful episodes.

"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 Officer of SPCI 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."

Altemia is intended to be taken once-a-day to reduce vaso-occlussive crisis (VOC) episodes, anemia, organ damage and other disease complications in sickle cell patients. It has the potential to decrease blood cell adhesion, chronic inflammation and red blood cell hemolysis, each of which can lead to reduction in pain and organ damage.

Rare Pediatric Disease designation is granted by the FDA to therapies with the potential to treat serious or life-threatening rare diseases, specifically conditions that affect individuals under 18 years old. With the designation, pending approval, Altemia will be eligible to receive a rare pediatric disease priority review voucher, which can be worth upward of several million dollars.

Earlier this year, Sarepta sold its Priority Review Voucher for $125 million.

The drug appears to be the first for SCD to ever receive a pediatric voucher. Last year, RDR spoke with Bill Cumming, an advocate and special needs parent about the struggles of regulatory bodies not recognizing SCD as a pediatric condition.

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