First Treatment of the Rare Disease aTTP Gains FDA Approval

FDA approval was awarded to the first treatment for the rare disease acquired Thrombotic Thrombocytopenic Purpura or aTTP for short.

This rare disease occurs in less than .001% of the population, and before had no cure.

Thrombotic Thrombocytopenic Purpura (TTP)

TTP is extremely rare and only occurs in between 3-4 people per million every year. 10-20% of patients will die from the disease and anywhere from 50-70% will only experience the symptoms once.

The symptoms are characterized by the clotting in small blood cells which cuts off blood and oxygen supplies throughout the body. These clots can lead to strokes, heart attacks, brain damage, and even death.

There are two forms of TTP:

1. Inherited TTP is when a patient is born with the disease due to genetical mutations which don’t properly regulate the production of the enzyme ADAMTS13. Patients will continue to have reoccurrences throughout their lifetime. This form is abnormaly rare and only accounts for 5% of all cases of the disease. There is currently no form of long term treatment, only preventative measures during high-risk symptoms.
2. The second type of TTP is acquired TTP (aTTP) which accounts for 95% of patients. aTTP is a nongenetic occurrence of the disease where the immune system temporarily produces antibodies that prevent the production of a healthy amount of ADAMTS13. The cause is unknown but is seen in patients who have lupus, infections, or HIV. Other risk factors include strenuous bodily anomalies such as giving birth, cancer, or surgery. Cablivi is the first long term treatment for aTTP. It effectively prevents relapses and decreases deaths in patients.

Treatment for aTTP

Typically, patients receive constant plasma transplants for days or even weeks to treat each episode. Cablivi prevents blood clots and reduces reoccurrences.

Cablivi (caplacizumab-yhdp) is injected before and after each session of plasma exchange therapy. After plasma therapy, the patient will continue receiving injections of Cablivi for 30 days which helps to regulate the production of the enzyme which would otherwise cause reoccurrences.

In a longitudinal pharmaceutical study to test the effectiveness of Cablivi – patients who received the treatment only had reoccurring symptoms 13% of the time. This rate is one-third of the number of reoccurrences in patients without the drug. (38%)

From the U.N. to the U.S.

Cablivi was developed by Ablynx in the U.K. and was approved for use in the European Union in August of 2018. The drug did not receive approval in the United States from the FDA until February of 2019 (five months later).

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See the initial research for Cablivi in the New England Journal of Medicine, here.