Sickle Cell

Altemia, a new treatment for sickle cell disease (SCD), was granted orphan drug designation.

The U.S. FDA has approved hydroxycarbamide/hydroxyurea tablets in pediatric patients suffering from sickle cell anemia, ages two years and older, France-based Addmedica has announced.

Imara has announced that it has dosed the first patient in its Phase 2a clinical trial of IMR-687 in adult patients with sickle cell disease.

Emmaus Life Sciences has announced that it has a Medicaid Drug Rebate Agreement with the Centers for Medicare and Medicaid Services, allowing coverage of Endari (L-glutamine oral powder).

Emmaus Life Sciences has announced that Endari, a medication developed to reduce acute complications in patients with sickle cell disease, is now available by prescription in the United States.

BioMarin Chief Executive Officer Jean-Jacques Bienaime spoke about the company’s development of valoctocogene roxaparvovec, a gene therapy in development for the treatment of Hemophilia A.

Global Blood Therapeutics, Inc. was granted Breakthrough Therapy Designation from the U.S. Food and Drug Administration for voxelotor for the treatment of sickle cell disease.

A study published in the Journal of Clinical Sleep Medicine found 44 percent of adults with sickle cell disease also experience sleeping difficulties.