Oxbryta (voxelotor) has become the first approved therapy in the European Union to target the underlying cause of sickle cell disease (SCD) for patients 12 and older. The medication, given as a once-daily oral tablet, suppresses the sickling and destruction of red blood cells that underlies the disease. Eligible…

The U.S. Food and Drug Administration (FDA) has agreed to expand the use of Oxbryta (voxelotor) — the first approved therapy targeting the underlying cause of sickle cell disease (SCD) — to children as young as 4. The therapy previously was approved for SCD patients ages 12 and older.

Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…

The European Medicines Agency (EMA) is reviewing an application from Global Blood Therapeutics (GBT) that seeks full marketing approval for the oral therapy Oxbryta (voxelotor) for people with sickle cell disease (SCD), ages 12 and up. Oxbryta would be the first treatment targeting the root cause of…

Global Blood Therapeutics (GBT) has launched an early access program for voxelotor in Europe and other regions outside the U.S. to allow people with hemolytic anemia associated with sickle cell disease (SCD) to have access to treatment. The early access program will be open to those…

FT-4202, a potential disease-modifying oral treatment for sickle cell disease (SCD) by Forma Therapeutics, has been designated an orphan drug by the European Medicines Agency (EMA). The decision was based on a positive recommendation by EMA’s Committee for Medicinal Products for Human Use. Orphan designation is given to investigative…

Global Blood Therapeutics (GBT) plans to submit a marketing authorization application to the European Medicines Agency (EMA) asking that Oxbryta (voxelotor) be approved for treating hemolytic anemia in people with sickle cell disease (SCD) who are 12 or older. The submission is anticipated by…