In a significant milestone for Belgian biopharma company Ablynx, the U.S. Food and Drug Administration (FDA) has granted approval for Cablivi (caplacizumab-yhdp), a nanobody designed to treat acquired thrombotic thrombocytopenic purpura (aTTP). The FDA’s decision enables the drug to be used in combination with plasma exchange and immunosuppressive therapy, providing a much-needed treatment option for patients suffering from this rare and life-threatening condition.

Understanding aTTP and Its Impact

Acquired thrombotic thrombocytopenic purpura (aTTP) is an exceptionally rare and dangerous blood clotting disorder where patients experience widespread blood clots in small blood vessels throughout the body. These clots can block blood and oxygen flow to vital organs, potentially causing strokes, heart attacks, and, in severe cases, brain damage or death. The condition can be triggered by underlying conditions such as cancer, HIV, lupus, pregnancy, or infections, and it can also arise after procedures like surgery, chemotherapy, or bone marrow transplantation.

Cablivi’s Innovative Mechanism

Cablivi targets von Willebrand factor (vWF), a protein critical to hemostasis, by preventing its interaction with platelets, which are key players in clot formation. This approach aims to stop blood clots from forming, addressing the core issue in aTTP and offering a novel solution to a condition that has been notoriously challenging to manage.

Clinical Trial Success: Cablivi Outperforms Placebo

The FDA’s approval of Cablivi was based on robust clinical trial data, which showed the drug’s superiority over a placebo in treating aTTP. The trial, involving 145 patients, demonstrated that Cablivi not only improved platelet counts more quickly than a placebo but also reduced the frequency of aTTP-related deaths and recurrences. A key result from the study was the 13% recurrence rate of aTTP in patients treated with Cablivi, significantly lower than the 38% recurrence rate in the placebo group.

The clinical trial further confirmed that Cablivi reduced the occurrence of major thrombotic events, further solidifying its potential as a life-saving treatment option for those battling aTTP.

Expert Commentary: FDA Approval’s Game-Changing Impact

Richard Pazdur, acting director of the Office of Hematology and Oncology Products at the FDA, commented on the approval, emphasizing that aTTP patients often endure painful and lengthy treatments, including daily plasma exchanges. Despite these measures, many still face relapses. He highlighted that Cablivi is the first targeted therapy to inhibit blood clot formation, offering a new option that may reduce recurrences and improve patient outcomes.

Sanofi’s Acquisition and Strategic Move

The FDA approval comes after Ablynx was acquired by Sanofi in a €3.9 billion deal last year. This approval also represents a significant achievement for Sanofi’s newly formed rare blood disorders franchise. Olivier Brandicourt, CEO of Sanofi, underscored the importance of Cablivi’s approval, noting that it marks a major milestone in expanding treatment options for aTTP patients, a group historically underserved by available therapies.

Conclusion: A Major Step Forward for Rare Disease Treatment

The approval of Cablivi by the FDA signals a much-needed breakthrough in the treatment of aTTP, providing hope for patients suffering from this rare and serious condition. With Sanofi now backing Ablynx, there is optimism that Cablivi will continue to drive advancements in rare disease therapies, offering better outcomes for patients who previously had limited treatment options.

  Ablynx, aTTP, Cablivi, FDA, Richard Pazdur, Sanofi