Oral inhibitor rilzabrutinib granted FDA orphan drug status for SCD

The U.S. Food and Drug Administration has granted orphan drug status to Sanofi’s rilzabrutinib, an investigational therapy aimed at preventing painful vaso-occlusive crises (VOCs) in people with sickle cell disease (SCD). This designation intends to encourage companies to develop therapies for rare diseases, or those affecting fewer…

Potential Treatment Targeting Nitric Oxide Levels, Olinciguat, Named Orphan Drug by FDA

The U.S. Food and Drug Administration (FDA) recently granted orphan drug status to olinciguat (IW-1701) as a potential treatment of sickle cell disease (SCD), Ironwood Pharmaceuticals announced. Evidence suggests that the symptoms of sickle cell disease may be associated with nitric oxide deficiency in the blood. Nitric oxide…

Altemia Granted Orphan Drug Status in EU as Potential Therapy for Pediatric Sickle Cell Disease

The European Medicines Agency recently granted orphan drug status to Sancilio Pharmaceuticals’ Altemia for the treatment of sickle cell disease (SCD) in pediatric patients, the company announced. Altemia is a specific formulation of lipids designed to replenish the elements needed for healthy red blood cells that are damaged…

Imara’s IMR-687 for SCD Receives FDA Rare Pediatric Disease Designation

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…