Experimental Treatment for Sickle Cell Disease Earns Orphan Drug Designation in the EU

According to a story from Street Insider, the biopharmaceutical company Forma Therapeutics Holdings, Inc., recently announced that its investigational therapy candidate FT-4202 has earned Orphan Drug designation from the European Commission. The drug has also previously received Orphan Drug, Rare Pediatric Disease, and Fast Track designation from the US Food and Drug Administration (FDA). It is being developed as a treatment for sickle cell disease. Forma Therapeutics is focused on developing treatments for rare blood diseases and cancers.

About Sickle Cell Disease

Sickle cell disease is a genetic, inherited disorder of the blood. This disorder is characterized by an abnormality affecting hemoglobin, which is found in red blood cells and is responsible for carrying oxygen. The abnormality causes blood cells to lose their typical circular shape and instead take on an elongated, sickle-like appearance. This is caused by a genetic mutation that may have arisen as a defense against malaria, although this benefit only occurs in people with sickle cell trait, not the disease. Symptoms begin to appear at around six months old and include swelling of the hands and feet, stroke, bacterial infections, and acute episodes of severe pain termed sickle cell crisis. Severity of disease varies, but these attacks can result in serious declines in health and organ damage. Treatment is mostly symptomatic, but bone marrow transplant has been curative in children. The disease most frequently affects people of African ancestry. Life expectancy is between 40 and 60. To learn more about sickle cell disease, click here.

About Orphan Designation in the EU

Orphan designation in the EU is similar, but not identical to, Orphan Drug designation in the US. It is generally reserved for therapies being developed for diseases that are considered rare, which is defined as any illness that affects less than 5 in 10,000 people in the EU. The disease must be chronic, debilitating, and/or life threatening. The medicine must have the potential to meet an unmet medical need or have major advantages over current treatments. The designation confers several benefits to the recipient company, such as certain fee reductions, protocol assistance, and a ten year period of market exclusivity if the drug is approved.

A phase 1 trial is currently accepting enrollment in order to investigate the pharmacological and safety properties of FT-4202. Forma also aims to initiate a phase 2/3 trial early next year.

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