Grifols seeks US approval for innovative drug to treat rare bleeding disorder

SPAIN – Grifols, a Spanish company specializing in plasma-based therapies, has submitted a regulatory application in the United States for its new drug aimed at treating acquired fibrinogen deficiency (AFD), a rare bleeding disorder.

This new treatment, known as BT524, offers a more reliable and convenient option for patients compared to existing therapies.

The US submission follows an earlier marketing application filed in the European Union. BT524, originally developed by German biotech firm Biotest—which Grifols acquired in 2022 for US $1.9 billion—is expected to launch in Europe during the second half of this year.

A US rollout could follow in early 2026, pending regulatory approval.

BT524 demonstrated its effectiveness in the phase 3 AdFirst trial, with results showing it matched or exceeded the performance of current treatments like fresh frozen plasma (FFP) and cryoprecipitate in reducing blood loss during spinal and abdominal surgeries in AFD patients.

AFD occurs when fibrinogen—a liver-produced protein essential for blood clotting and wound healing—becomes deficient.

This condition can arise during surgeries due to significant blood loss and plasma replacement, resulting in impaired clotting and excessive bleeding.

While existing treatments are effective, they present challenges. Therapies like FFP and cryoprecipitate contain inconsistent fibrinogen levels, require thawing before use, and involve longer preparation times with larger volumes.

BT524, in contrast, is a concentrated preparation that offers a precise dosage in a small volume, allowing for quicker administration directly from a vial.

Dr. Jörg Schüttrumpf, Grifols’ Chief Scientific Innovation Officer, emphasized the importance of this new therapy.

“A fibrinogen deficit impedes the body’s ability to stop bleeding, which can lead to life-threatening situations. Our new fibrinogen is faster to prepare, easier to store at room temperature, and more convenient than current options like cryoprecipitate or FFP,” he stated.

If approved by the FDA, BT524 would become the first concentrated fibrinogen product available in the US market.

This presents a significant growth opportunity for Grifols, as the global AFD therapy market is valued at around US $800 million, with the US representing the largest share. In 2023, Grifols reported revenues of €6.6 billion (approximately US $6.8 billion).

In Europe, however, the competitive landscape is more challenging, as similar concentrated fibrinogen products—such as RiaSTAP by CSL Behring and Fibryga by Octapharma—are already available in certain countries.

This filing comes shortly after Grifols abandoned takeover discussions with private equity firm Brookfield in November, following an inability to agree on the group’s valuation.

Despite this, the company remains focused on expanding its offerings and delivering innovative solutions to address unmet medical needs.