According to a story from Pharmacy Times, the US Food and Drug Administration (FDA) have recently approved the first ever therapy for the rare disorder acquired thrombotic thrombocytopenic purpura (aTTP), a disorder which affects the blood. The drug that was approved is called caplacizumab-yhdp. This drug is a nanobody based therapy that will be combined with immune system suppressing drugs and plasma exchange.
About Acquired Thrombotic Thrombocytopenic Purpura
Acquired thrombotic thrombocytopenic 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.
Caplacizumab-yhdp is the first targeted therapy that is capable of inhibiting the creation of the microscopic blood clots that characterize the disease. The drug targets von Willebrand factor (vWF). This protein interacts with blood platelets and plays a critical role in the formation of blood clots.
The approval of this new therapy for aTTP comes as a result of encouraging Phase 3 clinical trial data in which the treatment was able to initiate changes in platelet count more quickly than immunosuppression and plasma exchange alone. Patients who used the drug also saw substantially reduced risk of thromboembolic events, lower aTTP recurrence, and an overall decreased risk of death from the disease.
Sanofi, the developer of caplaciuzmab-yhdp, says that treatment in combination with plasma exchange should begin soon after aTTP is diagnosed.
