In an incredible move on Friday, the U.S. Food and Drug Administration (FDA) approved Endari for the treatment of patients age five and older with sickle cell disease to reduce severe complications that are common with the blood disorder. What makes this such an incredible decision is that Endari is the first treatment approved for patients with sickle cell disease in almost 20 years. It seems it was due for an update.
The FDA granted the approval of Endari to private biotech firm, Emmaus Medical. Previously, Endari received Orphan Drug designation for this use.
Additionally, development of this drug was in part supported by the FDA Orphan Products Grants Program, which provides grants for clinical studies on safety and effectiveness of products for use in rare diseases or conditions.
Sickle cell disease is an inherited blood disorder in which the red blood cells are abnormally shaped. This restricts the flow in blood vessels and limits oxygen delivery to the body’s tissues, leading to severe pain and organ damage. According to the National Institutes of Health, approximately 100,000 people in the United States have sickle cell disease. The disease occurs most often in African Americans, Latinos and other minority groups. The average life expectancy for patients with sickle cell disease in the United States is approximately 40 to 60 years.
Emmaus Medical is not the only firm pioneering a new treatment in this blood disorder, however. GlycoMimetics Inc. (NASDAQ: GLYC), in conjunction with Pfizer Inc. (NYSE: PFE), is in Phase 3 for Rivipansel, which has also received Orphan Drug status and Fast Track status from the FDA.
Shares of GlycoMimetics were last seen trading up nearly 1% at $11.22, with a consensus analyst price target of $14.25 and a 52-week range of $3.82 to $16.94.
Pfizer shares were trading at $33.20, in a 52-week range of $29.83 to $37.39. The consensus price target is $37.35.