Positive results for fibryga® in the treatment of bleeding and surgical prophylaxis in patients with congenital fibrinogen deficiency (FORMA-02) published in the Journal of Thrombosis and Haemostasis

The final results of the Phase 3 clinical trial investigating fibryga® for the treatment of bleeding and surgical prophylaxis in patients with congenital fibrinogen deficiency (FORMA-02) has been published in the Journal of Thrombosis and Haemostasis (JTH).

Although fibrinogen replacement therapy is long established in treating congenital fibrinogen deficiencies (CFD), proper clinical trials were lacking for the safe and effective use of fibrinogen concentrates in patients for on demand and prophylactic treatment.

FORMA-02 (NCT02267226) was a prospective, multi-centre, open label, uncontrolled Phase 3 efficacy and safety study in patients with CFD. Twelve centres in nine countries participated in this study between October 2014 and February 2018.

The newly published final data largely confirms the interim results, as reported earlier, and represents the largest dataset from a prospective interventional study in CFD to date.

With the publication of the final results of FORMA-02, Octapharma strongly contributes to the sparse clinical evidence on fibrinogen replacement in CFD and also underlines the company’s commitment to the rare disorder community.

Main results:

The study included 25 patients with hypo- and afibrinogenemia, whilst 24 patients were treated for on-demand treatment of 89 bleeding episodes (BEs), and 9 patients were treated for perioperative prophylaxis in 12 surgeries. Treatment success in on demand treatment was found in 98.9% of BEs. The increase in maximum clot firmness (MCF) using Rotem from pre infusion (0.0 mm), was 5.8±2.5 mm one hour after the first fibryga® infusion (fibryga® mean dose 61.9 mg/kg). For the 12 surgeries (fibryga® mean dose/surgery, 85.8 mg/kg), intraoperative and postoperative treatment success were both rated 100%. Three adverse events were possibly treatment-related, including a moderate case of thrombosis. There were no deaths, no severe allergic or hypersensitivity reactions and no clinical evidence of neutralising anti-fibrinogen antibodies.

To date, FORMA-02 is the largest prospective, multinational, Phase 3 efficacy and safety study in adult and adolescent patients with congenital hypo- and afibrinogenemia. The results confirm an excellent efficacy and safety profile for fibryga® in treating acute bleeding as well as in surgical prophylaxis.

The publication is pre-published online. Open access will be granted in the next weeks.

Reference

1. Lissitchkov, T. and Madan, B. et al. (2019) ‘Fibrinogen concentrate for treatment of bleeding and surgical prophylaxis in congenital fibrinogen deficiency patients’, J Thromb Haemost. 2019 Dec 29. doi: 10.1111/jth.14727. [Epub ahead of print]. 

About Octapharma

The vision of Octapharma is: “Our passion drives us to provide new health solutions advancing human life”. Headquartered in Lachen, Switzerland, Octapharma is one of the largest human protein manufacturers in the world, developing and producing human proteins from human plasma and human cell lines. As a family-owned company, Octapharma believes in investing to make a difference in people’s lives and has been doing so since 1983; because it’s in our blood. Our company values are Ownership, Integrity, Leadership, Sustainability and Entrepreneurship.

In 2018, the Group achieved €1.8 billion in revenue, an operating income of €346 million and invested €240 million into R&D and in capital expenditures in order to ensure future prosperity. Octapharma employs 8,314 people worldwide to support the treatment of patients in 115 countries with products across three therapeutic areas:

• Haematology (coagulation disorders)

• Immunotherapy (immune disorders)

• Critical care (bleeding management and functional volume replacement)

Octapharma has seven R&D sites and six state-of-the-art manufacturing facilities in Austria, France, Germany, Mexico and Sweden.

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