Rare Daily Staff

The U.S. Food and Drug Administration awarded Rare Pediatric Disease Designation to Sancilio Pharmaceuticals for the company’s Altemia, an experimental therapy for the treatment of sickle cell disease in children.

Sickle cell disease is a group of genetic disorders that results in dysfunctional hemoglobin 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.

Many children with sickle develop organ damage and strokes. There are approximately 100,000 cases of sickle cell disease in the United States. Treatment options are limited. The cost of care for this group may exceed $5 billion a year.

Altemia is being developed as an oral, once-a-day formulation for children between the ages of 5 to 17 with sickle cell disease. It consists of a complex mixture of lipids formulated to address the treatment of the disease.

The drug is designed to reduce vaso-occlusive crisis, a common painful complication of sickle cell disease episodes, as well as anemia, organ damage, and other complications. Altemia is designed to replenish the specific lipids destroyed by the variant of hemoglobin in sickle cell disease patients.

The FDA grants Rare Pediatric Disease designation to therapeutics intended to treat serious or life-threatening rare diseases that primarily affect individuals under the age of 18. If Sancilio is successful in winning marketing approval for Altemia, it will receive a rare pediatric disease priority review voucher.

The voucher is potentially lucrative because it is transferrable and can be used to accelerate the approval of any drug. That could give the holder faster access to mutibillion markets. In the past, such vouchers have sold for as much as $350 million.

September 20, 2017

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