Altemia Granted Orphan Drug Designation for Treatment of Sickle Cell Disease

The European Commission has granted Sancilio Pharmaceuticals Company, Inc. (SPCI) orphan drug designation for its product, Altemia, a novel oral formulation in development for the treatment of sickle cell disease (SCD) in pediatric patients. The designation follows a positive opinion given by the European Medicine Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) in February 2018. Altemia also received Orphan Drug Designation from the US Food and Drug Administration (FDA) in 2015.

Altemia is a mixture of lipids encased in a small, soft gelatin capsule that is intended to be taken once daily to reduce anemia, organ damage, vaso-occlusive crises (VOC) episodes, and other disease complications in SCD patients. Altemia is engineered to replace the specific lipids HbS destroys in an effort to prevent the initiation of the cascade effect that occurs in VOC episodes.

In reference to the research conducted by SPCI and others, it is believed the specific lipids contained in Altemia have the potential to restore fluidity and balance to red blood cells and other cells affected by the disease. Altemia purports to treat SCD by decreasing chronic inflammation, blood cell adhesion, red blood cell hemolysis, VOCs and organ damage, and the factors that lead to pain episodes.

According to a company statement, SPCI and others have good reason to believe this drug will be an effective treatment since it is well positioned to administer specific lipids directly to the membrane of red blood cells of SCD patients. The combination of the drug delivery and highly purified lipids have a high potential of significantly reducing VOCs, sickle cell crisis events, and related mortality as well as addressing the inflammatory symptoms of SCD.

Positive topline data results from SPCI’s Phase 2 study in November 2017 add to the drug’s promise. “The European Commission designating Altemia as an Orphan Medicine for the EU continues to advance the pathway for our lead clinical compound,” said Geoffrey Glass, CEO of SPCI. “The pediatric population of Sickle Cell Disease patients needs, and deserves, more therapeutic options, and we are excited about the opportunity to gain advice from the European Commission on advancing Altemia in the EU markets.”¹

Sickle Cell Disease (SCD) is a genetic disorder in which there is dysfunctional hemoglobin (HbS) and a depletion of certain lipids in the walls of blood cells. These dysfunctions create an increase in the red and white blood cells’ tendency to adhere to each other, which create an inflammatory state that results in reperfusion damage, episodic occlusions of blood vessels, and excruciating pain. Approximately 100,000 cases of SCD exist in the United States.

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References:

1. “Sancilio Pharmaceuticals Company Announces Altemia™ Receives Orphan Drug Designation from the European Medicines Agency for the Treatment of Pediatric Patients with Sickle Cell Disease (SCD).” Business Wire. 3 Apr. 2018