Grifols readies US, EU approval push for blood clot treatment after phase 3 success

Grifols is readying an approval run at U.S. and European regulators on the back of a phase 3 win for the Spanish plasma medicine producer's concentrated form of a blood clotting factor.

The product, dubbed BT524, is a concentrated form of fibrinogen, a glycoprotein that is essential for blood clot formation and stops excessive bleeding resulting from injuries or surgery. Grifols was testing BT524 in a late-stage study of patients with acquired fibrinogen deficiency (AFD), which it described as an “underserved growth market.”

The Barcelona, Spain-based company announced this morning that the product had met the study’s primary endpoint, demonstrating its noninferiority to standard of care when it came to reducing blood loss in patients with AFD who underwent spinal or abdominal surgery, while also “maintaining an excellent safety profile.”

Specifically, mean blood loss was 1,444 mL among the BT524 cohort compared with 1,735 mL among patients who received standard of care. It meant patients receiving Grifols’ product lost an average of 291 mL less blood.

AFD usually occurs during surgery when a patient’s body contains insufficient fibrinogen to arrest bleeding. It is typically treated with a plasma extract called cryoprecipitate or fresh frozen plasma, which both contain fibrinogen.

However, both of these treatment options contain various other unneeded proteins and elements, meaning that large volumes are required to ensure the patient receives sufficient fibrinogen, Grifols explained in the release. They also need to be thawed before use, which is not only time-consuming but can put patients’ lives at risk, the company added.

In contrast, BT524 can be administered to “patients on the operating table immediately,” who will only receive the fibrinogen necessary to prevent hemorrhaging, Grifols explained.

The company’s plan is to kick off the approval process in both Europe and the U.S. in the fourth quarter of the year. If successful, it would mark the first fibrinogen concentrate approved for AFD in the U.S., Grifols noted.

“The positive results for fibrinogen are an important milestone and strengthen a Grifols innovation strategy that’s diversified across plasma and non-plasma, balanced between internal and external investments, and includes both shorter- and longer-term development cycles,” Grifols’ chief operating officer, Victor Grifols Deu, said in the release.

While Grifols has its eye on the first fibrinogen concentrate approval for AFD in the U.S., its competitors have had more luck getting their own versions to market for congenital fibrinogen deficiency. CSL Behring got RiaSTAP approved in that indication as far back as 2009, while Switzerland’s Octapharma followed suit with Fibryga in 2017. Since then, Fibryga has also received approval to treat AFD across Europe off the back of a phase 2 study.

Meanwhile, Grifols is in the midst of an ongoing legal battle with Gotham City Research after the New York hedge fund accused the company of wrongful accounting practices.