Grifols files drug for rare bleeding disorder in the US

Grifols has filed for US approval of a new drug for rare bleeding disorder acquired fibrinogen deficiency (AFD), which promises to provide a more reliable and easier treatment for patients.

The US filing comes after the Spanish specialist in plasma-based therapies submitted a marketing application in the EU for the new concentrated preparation of fibrinogen – codenamed BT624 – that was originally developed by Biotest, a German biotech acquired by Grifols for $1.9 billion in 2022.

It says it expects to launch in Europe in the second half of this year, with a US rollout happening in early 2026, assuming the regulatory reviews result in approval.

The results of the phase 3 AdFirst trial of BT524 reported last year showed it was at least as effective as current therapies based on fresh frozen plasma (FFP) or cryoprecipitate at reducing blood loss in AFD patients undergoing spinal and abdominal surgeries.

Fibrinogen is produced in the liver and is involved in blood clotting and wound healing. In patients with AFD – which can occur during surgery as a result of blood loss and replacement with plasma – clotting is impaired, leading to excessive bleeding.

While effective at treating AFD, the current standard therapies contain variable amounts of fibrinogen and need to be thawed before administration, leading to longer administration times and larger product volume.

In contrast, BT524 offers a defined concentration in a low volume, which means it can be administered quickly from a small vial, according to Grifols.

"A fibrinogen deficit impedes the body's ability to arrest bleeding, which can lead to death in severe situations," said Dr Jörg Schüttrumpf, Grifols' chief scientific innovation officer, in a statement on the US filing.

"Grifols developed its new fibrinogen to be more convenient, faster to prepare and storable at room temperature, an advantage over alternatives such as cryoprecipitate or fresh frozen plasma when time is of the essence," he added. "We're excited to get this innovative solution to patients."

If approved by the FDA, BT524 could become the first concentrated fibrinogen product to reach the US market, making it a major new growth opportunity for Grifols, which has estimated that the global market for AFD therapies is worth approximately $800 million, with the US accounting for the largest share. The company's total revenues were €6.6 billion (around $6.8 billion) in 2023.

In Europe, the market is more competitive, as concentrated fibrinogen preparations from CSL Behring (RiaSTAP) and Octapharma (Fibryga) are already available in some countries.

The filing comes a few weeks after Grifols was in takeover talks involving private equity firm Brookfield, which were abandoned in November after failing to agree a value for the group.