Grifols achieves positive results from phase 4 study of Fanhdi® (double-inactivated human anti-hemophilic factor) in patients with von Willebrand Disease

Barcelona, Spain, April 22, 2024 – Grifols (MCE: GRF, MCE: GRF.P NASDAQ: GRFS), one of the world’s leading producers of plasma-derived medicines and a pioneer in rare disease treatment, today announced that its recently concluded observational phase 4 study of Fanhdi^®, Grifols’ double-inactivated human anti-hemophilic factor, showed positive results in the product’s safety and efficacy as prophylaxis and treatment in patients with von Willebrand Disease (VWD). Grifols will present the data today at the World Hemophilia Federation 2024 World Congress, in Madrid.

VWD is an incurable bleeding disorder that affects the ability of blood to clot properly. It's the most common hereditary blood-clotting disorder and in Spain alone, this rare disease is estimated to impact 122 patients per million².

Grifols conducted this phase 4, observational, multi-center, prospective cohort study to evaluate the safety and clinical efficacy of long-term use of Fanhdi in subjects with VWD. The overall clinical efficacy of treatment with Fanhdi (including on-demand and prophylaxis) achieved 100% excellent and/or good (n=15 patients) rated by the investigator, with no safety concerns raised throughout the study.

“This observational study further reinforces the potential long-term effectiveness of Fanhdi in the treatment of bleeding episodes and as prophylaxis before surgical procedures,” said Dr. Jörg Schüttrumpf, Grifols Chief Scientific Innovation Officer. “Grifols continues to develop and provide advanced lifesaving therapies for patients in need.”

A total of 17 participants were enrolled in the 12-month study, 15 of them receiving at least one dose of Fanhdi^®. The efficacy was assessed during 46 bleeding episodes reported for nine (60%) subjects, and six surgical/invasive procedures reported for six (33.3%) subjects.

About Fanhdi^®

In the EU and other countries – though not in the United States – Antihemophilic Factor/von Willebrand Factor Complex (Human), Fanhdi^®, is indicated for the prevention and control of bleeding in patients with Factor VIII deficiency due to hemophilia A. Fanhdi^® is also indicated for surgical and/or invasive procedures in adult and pediatric patients with von Willebrand Disease, except Type III undergoing major surgery, in whom desmopressin (DDAVP^®) is either ineffective or contraindicated. Antihemophilic factor potency (Factor VIII:C activity) is expressed in International Units (IU) on the product label. Additionally, each vial of Fanhdi^® also contains VWF:RCo activity in IU for the treatment of VWD. Fanhdi^® is contraindicated in patients who have manifested life-threatening immediate hypersensitivity reactions, including anaphylaxis, to the product or its components. Anaphylaxis and severe hypersensitivity reactions are possible. Development of activity-neutralizing antibodies has been detected in patients receiving FVIII containing products. Development of alloantibodies to VWF in Type 3 VWD patients has been occasionally reported in the literature. Fanhdi labeling may vary depending on country.