Sancilio Pharmaceuticals Company, Inc. (SPCI) receives rare pediatric disease designation from the U.S. Food and Drug Administration for Altemia, a treatment of sickle cell disease (SCD) in children.
Sancilio Pharmaceuticals Company, Inc. (SPCI) announced 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. Altemia, a development program for SPCI, is being developed as an oral, once-a-day formulation for children between the ages of 5 – 17 with 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 Officer 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 defines a rare pediatric disease as a serious or life-threatening disease in which the serious or life-threatening manifestations primarily affect individuals age 18 years or younger, and is a rare disease that impacts fewer than 200,000 individuals in the United States. The Rare Pediatric Disease designation provides incentives to advance the development of rare disease drugs and biologics.
Additionally, the FDA’s Rare Pediatric Disease Priority Review Voucher Program states that a sponsor with a Rare Pediatric Disease designation who receives a New Drug Application (NDA) or Biologic License Application (BLA) approval for a rare pediatric disease may be eligible for a voucher that can be redeemed to obtain priority review for any subsequent marketing application.
About Sickle Cell Disease (SCD)
Sickle Cell Disease (SCD) is a group of genetic disorders that results in dysfunctional hemoglobin (HbS) and a depletion of certain lipids in the walls of blood cells. These abnormalities create an inflammatory state and an increase in the red and white blood cell’s tendency to adhere to each other, resulting in episodic occlusions of blood vessels, reperfusion damage, and excruciating pain.
Ultimately, many children develop end organ damage and strokes. There are approximately 100,000 cases of SCD in the United States. Treatment options are limited.
About Altemia
Altemia is a proprietary product candidate that is being developed for the treatment of SCD. Altemia consists of a complex mixture of lipids formulated using Advanced Lipid Technologies (ALT) specifically to address the treatment of the disease. The drug is encapsulated in a small soft gelatin capsule and intended to be taken once daily to reduce VOC episodes, anemia, organ damage and other disease complications in sickle cell patients.
(Source: PR Newswire)
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