Experimental Drug for Sickle Cell Disease Earns Fast Track Designation

According to a story from BioSpace, the biopharmaceutical company Imara, Inc. has recently announced that its experimental product candidate IMR-687 has earned Fast Track designation from the US Food and Drug Administration (FDA). This investigational drug is in development as a treatment for sickle cell disease. IMR-687 is also in the midst of testing in a phase 2a clinical trial with adult patients.

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 become 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. Life expectancy is between 40 and 60. To learn more about sickle cell disease, click here.

About Fast Track Designation

Fast Track designation is an FDA program that is designed with the explicit goal of expediting the development process for drugs that are intended to treat serious diseases or that could fulfill an unmet medical need. In instances where the drug is not satisfying an unmet need, the therapy must display advantages in safety or effectiveness in comparison to currently available therapies. A drug that earns Fast Track designation gets a number of advantages, such as more frequent meetings with the FDA, potential eligibility for Priority Review and Accelerated Approval (if other criteria are met), and Rolling Review, which allows the company to submit its application by section instead of having to wait for completion.

IMR-687 has also earned Orphan Drug designation from the FDA.

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