This follows the Orphan Drug Designation for Altemia™ granted by the U.S. Food and Drug Administration (FDA) in 2015
RIVIERA BEACH, Fla. – April 3rd, 2018
Micelle BioPharma today announced that the European Commission has granted Orphan Designation in the European Union (EU) to Altemia, the Company’s novel oral formulation being developed for the treatment of SCD in pediatric patients. This approval follows a positive opinion in February 2018 from the European Medicine Agency’s (EMA) Committee for Orphan Medicinal Products (COMP).
Micelle BioPharma reported positive topline data from its Phase 2 study in November 2017. “The pediatric population of Sickle Cell Disease patients need, and deserve, more therapeutic options, and we are excited about the opportunity to gain advice from the European Commission on advancing Altemia in the EU markets.”
Orphan Designation is granted by the European Commission to drugs that are intended for the treatment, prevention or diagnosis of life-threatening or chronically debilitating rare diseases. Rare diseases are those defined as having a prevalence of not more than five per 10,000 population in the European Union. The designation potentially qualifies the sponsor for ten years of marketing exclusivity upon approval, as well as fee reduction for various centralized activities, including applications for marketing authorization, inspections and protocol assistance.
Altemia had previously received Orphan Drug Designation from U.S. Food and Drug Administration (FDA) for the US market.
“We are extremely encouraged to receive this designation, further supporting the significant clinical and regulatory progress made in advancing Altemia’s development program,” said Adrian L. Rabinowicz, MD, Chief Medical Officer of Micelle BioPharma.
Micelle BioPharma plans to present detailed data from the recently completed SCOT Phase 2 study in peer reviewed journals and upcoming scientific conferences. The Company plans to meet with the U.S. FDA as well as European Medicines Agency (EMA) to address next steps for Altemia.
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 cells’ tendency to adhere to each other, resulting in episodic occlusions of blood vessels, reperfusion damage and excruciating pain. Ultimately, many children develop organ damage and strokes. There are approximately 100,000 cases of SCD in the United States and treatment options are limited. The cost of care for this group may exceed $5 billion.
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.
HbS destroys specific lipids, creating a cascade that culminates in VOC episodes. Altemia is designed to replenish those lipids in order to prevent the cascade effect from initiating.
Based on research performed by Micelle Bio Pharma 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, VOCs 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 VOCs 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 Micelle Bio Pharma, Inc.
Micelle Bio Pharma 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. Micelle BioPharma 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.