Grifols is gearing up to file for approval of a drug for rare bleeding disorder acquired fibrinogen deficiency (AFD) that could provide a more reliable and easier treatment for patients.
The Spanish company’s Biotest subsidiary has reported top-line results of the phase 3 AdFirst trial of the concentrated preparation of fibrinogen, called BT524, which show it is 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.
In the study, average blood loss was 1,444 mL in the BT524 group compared 1,735 mL for subjects treated with cryoprecipitate/FFP, a reduction of 291 mL.
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.
The phase 3 results will be used to support regulatory filings before the end of the year in the US – where it could be the first product of its kind in the market – as well as in Europe where concentrated fibrinogen preparations from CSL Behring (RiaSTAP) and Octapharma (Fibryga) are available in some markets.
Barcelona-based Grifols has said the global market for AFD therapies is worth approximately $800 million a year, and it is driven by its use in surgical treatments. The US accounts for the largest share of that total.
“This successful clinical trial is a significant stride towards a potential therapy that could contribute to the care of patients who experience severe blood loss during major surgery,” said Jörg Schüttrumpf, the company’s chief scientific innovation officer.
Grifols bought Biotest in 2022 in a deal, worth approximately $1.9 billion, that was aimed at extending its core plasma collection business and adding to the group’s therapeutic pipeline.
Along with BT524, the acquisition added Yimmugo for acquired and congenital immunodeficiencies – which has been approved in Europe and filed in the US – along with trimodulin which is in phase 3 for community-acquired pneumonia.
News of the positive trial will be particularly welcome for Grifols at the moment, as it is locked in a legal dispute with short-seller Gotham City Research over a report which claimed it had fraudulently manipulated financial reporting and led to a share price crash that wiped more than €3 billion of its valuation.
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