The congenital fibrinogen deficiency (CFD) market is expected to grow at a compound annual growth rate (CAGR) of 1.7% through 2031, reaching over $800m across the three major markets (3MM: US, Germany, and Japan), according to GlobalData’s recent report: Congenital Fibrinogen Deficiency: Opportunity Assessment and Forecast. Major factors driving this growth include the launch of improved human fibrinogen concentrate (HFC) products, the large market potential for HFC in treating acquired fibrinogen deficiency (AFD), and recent increased demand for blood plasma. The Covid-19 pandemic has also led to a decrease in blood donations, which has increased demand for human plasma and facilitated higher usage of HFC.

High interest has sparked across the CFD drug space as more experts are becoming aware of the clinical efficacy and large market opportunity of using HFC products to treat various AFD indications, including surgeries involving massive blood loss and unexpected trauma. Since HFC replacement therapy is the mainstay of treatment for CFD, increased development of more innovative fibrinogen-based therapies will benefit both the CFD population and patients experiencing AFD.

Current treatment options for CFD are limited to the use of four HFC products, which serve as intravenous (IV) replacement therapy for fibrinogen (factor I) to control and prevent bleeding episodes in symptomatic patients. Two HFCs are marketed in the US (RiaSTAP and Fibryga) while three products are available in Germany (RiaSTAP/Haemocomplettan P, Fibryga, and FibCLOT), with only one marketed product available for use in Japan (Fibrinogen HT).

There are two pipeline HFCs in late-stage development expected to launch during the forecast period, Biotest/Grifols‘s BT-524 and Grifols’s FIB Grifols. Both pipeline products use Biotest’s newly developed production facility, which enables improved manufacturing of fibrinogen with higher purity, defined concentrations, viral safety, and good solubility. BT-524 is currently being studied in a Phase III trial (ADFIRST) in AFD patients undergoing elective spinal and/or abdominal surgery and has high potential to gain approval for both indications. This provides a large advantage over currently marketed products and may allow BT-524 to capture a significant share of the market at launch.

HFCs are derived from human plasma and carry risks of infection and blood clots. An urgent unmet need exists among the CFD population for differentiated therapies with lower risks of thrombosis, guaranteed viral safety, and more convenient routes of administration. CFD is an extremely rare disease in which many patients are often asymptomatic and do not require treatment. Since the drug treated CFD population is very small, drug developers are not heavily invested in the space and clinical trials frequently struggle with recruitment, especially in areas with low diagnosed prevalence. The large, untapped AFD market will lead to increased drug development of more innovative fibrinogen replacement therapies, advancing the HFC drug space for both CFD and AFD patients.

HFC products must be administered intravenously in a healthcare setting and carry risks of thrombosis. While they treat the symptoms of CFD, they are not curative. Therefore, there is also a significant need for new therapies that target the underlying cause of CFD that may offer a cure for patients living with severe disease, or for patients with genotypes such as dysfibrinogenemia and hypodysfibrinogenemia, who are often ineligible for treatment with HFCs.

Attempts have been made to develop treatments that are not derived from human plasma, in order to eliminate the risk of transmitting viral infections, decrease the risk of blood clots, and to provide options that patients can self-administer at home. However, the large size of the fibrinogen molecule has prevented the production of a recombinant fibrinogen protein and will delay potential development of gene therapies in the future. Key opinion leaders (KOLs) interviewed by GlobalData have noted that the development of products not made using human blood is possible but will require more time and research.

Given the huge market potential for HFC use in AFD patients, the development of fibrinogen-based therapies is expected to increase dramatically, fulfilling many unmet needs for CFD patients. Other indications also remain largely unexplored in the HFC drug space, leaving ample opportunities for companies to invest in this area for a variety of blood disorders and surgical complications. As the field becomes more understood and demand for human plasma continues to increase, GlobalData anticipates a dynamic market with a variety of fibrinogen-based treatment options for CFD patients in the near future.