RIVIERA BEACH, Fla., Sept. 19, 2017 /PRNewswire/ -- Sancilio Pharmaceuticals Company, Inc. (SPCI) today 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 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.
"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. The cost of care for this group may exceed $5BB.
Altemia™ is our 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.
Altemia™ is designed to replenish the specific lipids destroyed by HbS creating a cascade that culminates in episodes. Based on research performed by Sancilio Pharmaceuticals Company, Inc. (SPCI) and others, the specific lipids contained in Altemia™, may restore balance and fluidity to red blood cells and other cells impacted by the disease. We believe that Altemia™ will treat sickle cell disease by decreasing blood cell adhesion, chronic inflammation and red blood cell hemolysis, the factors that lead to reduction in pain episodes, VOC crises and organ damage. Based on its formulation and mechanism of action, we believe that Altemia™ is well-positioned to deliver a narrow, therapeutic dose of certain lipids directly to the membrane of red blood cells of sickle cell patients. The combination of ALT® drug delivery technology and highly purified lipids may reduce VOC significantly. We also believe that 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 sickle cell crisis events and related mortality.
About Sancilio Pharmaceuticals Company, Inc.
Sancilio Pharmaceuticals Company, Inc. (SPCI) is a fully integrated, specialty pharmaceutical company focused on developing, manufacturing and commercializing pharmaceutical products, including those based on our proprietary Advanced Lipid Technologies® (ALT®) platform. SPCI is pursuing treatments for sickle cell disease, short bowel syndrome and severe hypertriglyceridemia. We utilize our cGMP compliant facility to develop and manufacture our products. Our ALT® platform is designed to enhance the bioavailability, reduce the food effect and improve the efficacy of lipids and lipophilic active pharmaceutical ingredients (APIs). Lipids are hydrophobic or amphipathic molecules, including fatty acids, steroids (including hormones) and fat-soluble vitamins (such as vitamins A, D, E and K). Our business model is to apply our ALT® platform to lipids or lipophilic APIs to create unique product candidates that address the disorders and diseases resulting from imbalances of lipids in the body. In addition to our primary focus of developing our proprietary products using the ALT® platform, we make use of, and license rights to, our proprietary ALT® platform and other technologies to third parties, providing both development and subsequent soft gelatin encapsulation services. More information is available at www.sancilio.com.
Contacts Sancilio Pharmaceuticals Company, Inc. Marc Wolff, 561-847-2302 Executive Vice President & Chief Financial Officer