According to an article from PM360, the French biopharmaceutical company Sanofi has announced successful results at the end of a phase 3 study of their hopeful acquired thrombotic thrombocytopenic purpura (or aTTP) drug, Cablivi.
About Acquired Thrombotic Thrombocytopenic Purpura
Thrombotic thrombocytopenic purpura is a condition that causes tiny blood clots to form in the body’s small blood vessels. These clots can create a lot of serious problems, including low platelet (blood clotting cell) count, red blood cell death, and limited oxygen transfer to major organs. This all leads to serious organ damage, and can be fatal.
Acquired TTP (aTTP) is the non-inherited form of the condition. In patients with aTTP, the body mistakenly produces antibodies that inhibit the effectiveness of the enzyme ADAMTS13, which is normally involved in controlling blood clotting. This form of TTP is most often seen in adults, though children can also develop it.
Currently, the condition is treated with a daily plasma exchange – a physically demanding therapy that has many living with the condition hoping for a promising replacement as soon as possible.
Cablivi Succeeds in Phase 3 Trials, Waits for US Market
Enter Cablivi. In the wake of their successful phase 3 trial for the drug (dubbed HERCULES), Sanofi is now waiting for the FDA to act on their Biologics License Application. The BLA is a request for permission to introduce a biologic (a pharmaceutical made in or from biological sources) from overseas. Cablivi was approved for aTTP by the European Council back in 2018, and received orphan drug designation on both continents back in 2009.
Most pharmaceuticals don’t make it to phase 3 testing in the first place. Only drugs that have proven their safety and efficacy in earlier phase 1 and 2 trials can proceed on to stage 3, which is kind of like the fact-checking trial. Phase 3 studies are often multinational and involve hundreds, if not thousands of subjects over a lengthy period of time that can extend for years. As such, they’re a big investment. Hopes were high, but Cablivi seems to have met all expectations.
Over the course of the trial, patients taking Cablivi were 1.55 times more likely to have a normal platelet count than patients who had received the placebo. Cablivi recipients also enjoyed statistically significant reductions in the frequency of their plasma exchanges, hospitalizations, and flare ups – including an impressive 67% reduction in aTTP recurrences.
The target action for the BLA is February 6, which means Cablivi might be treating American aTTP patients within the next few months.
Do you or does someone you know live with thrombotic thrombocytopenic purpura? Are you excited for these promising developments? Let Patient Worthy know!