A Few Words on LFB's Pipeline
New treatments for serious and often rare diseases
LFB develops biomedicinal products within its areas of expertise namely Immunology, Hemostasis and Intensive Care, for the treatment of serious and often rare diseases.
LFB USA’s role developing new biotherapies in the US
LFB USA has a key role in developing LFB's pipeline in the US, focusing on research, clinical development and regulatory strategies and filings. For the Factor FVIIa (LR769) program, LFB USA handles the upstream manufacture of the protein and the clinical and regulatory aspects. On April 1st, 2020, LFB's recombinant form of human Factor VIIa was recently approved by the FDA for treatment and control of bleeding episodes occurring in adults and adolescents 12 years of age and older with hemophilia A or B with inhibitors (neutralizing antibodies) (https://www.fda.gov/news-events/press-announcements/fda-approves-additional-treatment-adults-and-adolescents-hemophilia-or-b-and-inhibitors). The Phase III studies in pediatric and surgical patients are now complete and will be filed soon to the FDA for review/approval for these follow-on indications. For more information on Factor VIIa, please visit the Sevenfact®website.
LFB USA is to play a role in the clinical and regulatory development of other high-potential LFB products in the US such as: fibrinogen, high-purity Von Willebrand factor, recombinant anti-D antibody and liquid 10% immunoglobulin. All of these products will undergo advanced clinical and regulatory development in the US through LFB USA, as they are all currently marketed, filed or in phase II/III clinical trials in Europe and other parts of the world.
Fibrinogen Deficiency and Clottafact®
Fibrinogen deficiency (also known as Factor I deficiency) is a rare bleeding disorder caused by absent or defective Factor I blood clotting protein. The deficiency can be congenital (hypo-, dys- or afibrinogenemia) or an acquired hypofibrinogenemia (such as postpartum haemorrhage, trauma or surgical interventions including cardiovascular or digestive operations and hepatic transplantations).
Clottafact® is human plasma-derived factor I concentrate. It is used for congenital and acquired hypofibrinogenemia.
Von Willebrand Disease and Wilfactin®
Von Willebrand disease is an inherited bleeding disorder caused by a defect or deficiency of a blood clotting protein called von Willebrand Factor (VWF). People with this disorder have varying degrees of bleeding tendency but typically bruise easily, have recurrent nosebleeds, or bleed after tooth extraction, tonsillectomy or other surgery. Women can have increased menstrual bleeding or heavy blood loss during childbirth.
Wilfactin® is human plasma-derived von Willebrand factor concentrate and is used for the prevention and treatment of hemorrhage or surgical bleeding in von Willebrand disease when desmopressin (DDAVP) treatment alone is ineffective or contraindicated.
Rh Incompatibility and Anti-D Immunoglobulin
RhD fetomaternal alloimmunization may occur in an RhD-negative mother bearing an RhD-positive fetus, whenever fetomaternal hemorrhage (e.g., during delivery) sensitizes the mother against the RhD antigen. Subsequent hemolytic disease, leading to morbidity and mortality, can affect the fetus or the newborn in current and future pregnancies.
The goal of treatment with Anti-D Immunoglobulin is to prevent RhD-alloimmunization in RhD-negative women carrying an RhD-positive fetus as well as treatment of RhD-negative individuals after incompatible transfusions with blood components containing RhD-positive red blood cells.
Immune Disorders and IVIG
In immune deficient patients who have decreased or inactive antibody production capabilities, IVIG is used as a plasma protein replacement therapy (IgG). It is administered to maintain adequate antibody levels to prevent infections. In patients with autoimmune disease, high dose IVIG may decrease the severity of the autoimmune diseases.
IVIG is a blood product that contains the pooled, polyvalent, IgG antibodies extracted from the plasma of blood donors. It is administered intravenously.