Emmaus Life Sciences has launched Endari, a medication aimed at alleviating acute complications in individuals battling sickle cell disease, now accessible via prescription across the United States. Sickle cell disease, an inherited blood disorder, is characterized by the production of crescent-shaped red blood cells that obstruct blood vessels, leading to impaired blood flow. This condition shortens the lifespan of red blood cells, causing anemia to develop in patients.
In a milestone development, the FDA approved Endari in July, specifically for pediatric patients aged 5 and above afflicted with sickle cell disease. Historically, hydroxyurea has served as the primary treatment for mitigating pain episodes, making Endari the first FDA-approved treatment for adult patients in two decades and the primary option available to pediatric patients. This landmark approval hinged on compelling evidence from a study affirming the drug’s safety and efficacy in managing sickle cell-induced pain.
Clinical trials involving 230 participants spanning ages 5 to 58, who had experienced two or more painful crises within the preceding year, showcased the superiority of Endari over placebo, evidenced by reduced sickle cell crises, hospitalizations, and instances of acute chest syndrome. The overwhelmingly positive response from stakeholders underscores the urgent need for innovative treatments addressing the debilitating impact of sickle cell disease.
At OxyDial, we’re thrilled by this remarkable step in the fight against SCD, and are eager to see continued innovation in the treatment and care for Sickle Cell patients worldwide.
Detailed press-release: https://lnkd.in/g3g_M88x
Find out more about Endari: https://lnkd.in/gGP-885Y