According to a publication from BioSpace, the French biopharmaceutical company Sanofi has successfully secured FDA approval for their acquired thrombotic thrombocytopenic purpura (aTTP) drug Cablivi. This represents the first approved treatment for the rare blood condition.
Acquired Thrombotic Thrombocytopenic Purpura
Say that three times fast.
The condition, known more conveniently as aTTP, causes blood clots to form in small blood vessels throughout the body. These clots can cause serious complications, like low platelet count, red blood cell death, and poor organ oxygenation. If left untreated, it can lead to organ failure – and even death.
The clotting starts when a person’s body mistakenly begins to produce antibodies that inhibit the effectiveness of the blood clotting enzyme ADAMTS13. Episodes can last for days, to weeks, to months – and relapses are known to occur in some 60% of aTTP patients. Prior to Cablivi’s approval, the only course of treatment available to aTTP patients was a physically exhausting daily plasma exchange.
Thrombotic thrombocytopenic purpura (or inherited TTP) is the rarer form of the disease, forming only some 5% of all TTP cases. People with this form of TTP were passed a faulty ADAMTS13 gene from one or both of their parents and are born with the condition.
TTP of some form affects an estimated one person in every 250,000.
Cablivi Secures FDA Approval
Last month, Sanofi successfully concluded a phase 3 clinical trial for Cablivi – at that point still technically an experimental drug.
The final data was impressive – aTTP patients administered the drug showed an average plate count 155% that of those in the placebo group. The Cablivi group also required significantly fewer plasma exchanges, and experienced an impressive 67% reduction in flare-ups.
It was a home run, and the success of the phase 3 trial paved the way for Cablivi’s approval.
It represents a huge shift in the treatability of aTTP. Cablivi is administered as an injection to be given in addition to immunosuppressive therapy and semi-regular plasma exchanges.
These plasma exchanges can sometimes take hours at a time, which is why Cablivi potentially offers such a significant improvement in quality of life. By reducing the need for daily plasma exchanges and lowering the rate of aTTP recurrence, Cablivi offers a real chance for some people in the rare disease community to retain a degree of normalcy in their lives.
Although aTTP is highly rare, hundreds of people could stand to benefit from a drug like Cablivi. How might Cablivi’s approval form the foundations of new investigative drugs? Share your thoughts with Patient Worthy!