According to a story from prnewswire.com, the drug Cablivi, also known as caplacizumab, has been granted marketing authorization by the European Commission. Cablivi is meant to treat acquired thrombotic thrombocytopenic purpura (aTTP), a rare disorder that affects blood clotting. This is the first drug to gain approval for the treatment of aTTP.
Acquired thrombotic thrombocytopenia purpura (aTTP) is a rare disorder which is characterized by the appearance of microscopic blood clots that affect the small blood vessels and cause low platelet counts. In the acquired form of the disease, the cause is either the result of autoimmune activity or aTTP may appear as the result of another medical condition, drug use, or disease. People of African descent appear to be at slightly greater risk. These risk factors include cancer, bone marrow transplant, pregnancy, or HIV. Symptoms include high blood pressure, hemolytic anemia, reduced kidney function, fever, thrombocytopenia, and changes in mental function. Treatment of aTTP often include plasmapharesis and the use of steroids and immune suppressants. This disease is often lethal without treatment, but most patients will survive with prompt intervention. This is a very rare disease, with only about four to five cases per million people appearing in a single year. To learn more about aTTP, click here.
Even with the current treatment standard, aTTP still has a mortality rate of around 20 percent, and most deaths can occur during the first month or so after diagnosis. In research trials, Cablivi was utilized as a supplement to current treatment approaches, and it was able to meaningfully improve several endpoints associated with aTTP, such as a reduced number of symptom recurrences, a reduction in disease-related deaths, and a shorter time to changes in platelet count.
Priority Review in the US
In addition to its approval in the EU, the US Food and Drug Administration (FDA) has accepted Cablivi under Priority Review, with a response expected to be announced in February of next year. The drug works by acting on the ability of platelets to stick together and form the clots that appear with aTTP and can result in serious symptoms such as organ damage.