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The U.S. Food and Drug Administration (FDA) granted rare pediatric disease designation to IMR-687, Imara’s drug candidate for the treatment of sickle cell disease (SCD). The investigational therapy has also been granted orphan drug status by the FDA.
Rare pediatric disease designation is granted to drugs that show promise for rare and serious or life-threatening diseases in U.S. patients, primarily age 18 years or younger. The designation provides incentives to advance the development of rare disease drugs, including access to the FDA’s expedited review and approval programs.
“People living with sickle cell disease have limited treatment options for its devastating effects,” James McArthur, PhD, Imara’s president and CEO, said in a news release. “The FDA’s decision reflects its commitment to working with innovators and the patient community toward advancing safe and effective treatments for children suffering from the rare and damaging pediatric manifestations of the disease.”
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This platform is made possible through a partnership with the Sickle Cell Disease Association of America, Inc. (SCDAA) and its member organizations. SCDAA's mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure.