Global Development Pipeline
Octapharma’s immunotherapy franchise will continue to work on multiple sclerosis (MS) and test the validity of a diagnostic tool to predict responses of relapsing-remitting MS (RR-MS) patients to octagam®. The validity of this proprietary tool may revolutionize the way RR-MS patients are treated with immunoglobulin. Other neurodegenerative disorders, notably Alzheimer’s disease (AD), are also a major clinical focus for our team. The role of immunoglobulin therapy for managing the symptoms of AD and MCI is being explored in clinical trials with octagam®.
Post-marketing studies are ongoing to further strengthen the body of evidence on the tolerability of octagam®. New high-yield intravenous and subcutaneous IG formulations are also under advanced stages of development and registrations are expected soon.
A new fibrinogen product is currently under development, addressing yet another essential area in critical care. Fibrinogen (Factor I) is a soluble plasma glycoprotein that is converted into fibrin and helps stop bleeding by promoting blood clot formation. Fibrinogen is used to treat bleeding episodes in people with congenital fibrinogen deficiency. Although it is a rare disease, the prevalence of congenital fibrinogen deficiency should not be underestimated. It varies from silent forms with almost no symptoms over a long period of time to moderate bleeding to severe events such as intracerebral bleeding.
We have also developed and commercialized universal plasma that can be used for any patient regardless of blood group. The uinvesal profile of this product is ensured by mixing plasma of A, B and AB types in predetermined ratios for the neutralization of anti-A and anti-B by free A and B substance.
Lyophilized universal plasma is in clinical development for immediate reconstitution and infusion without the need for cold storage, thawing or blood typing. This will be a key advantage in emergency infusions when time is of the essence.
Our understanding of hemophilia and other bleeding disorders is leveraged in our pioneering products for the treatment of hemophilia A, hemophilia B and von Willebrand disease (vWD). Today, we are leading the way in developing the first human recombinant coagulation Factor VIII, which is designed to reduce the overall immunogenic challenge (and resulting inhibitor formation) in patients with hemophilia A.
Recombinant coagulation FVIII from a human cell line (Human-cl rhFVIII):
Formation of neutralizing FVIII-antibodies is presently the most devastating complication of FVIII replacement therapy in patients with hemophilia A. Human-cl rhFVIII is a newly developed recombinant FVIII (rFVIII) concentrate produced in genetically modified human embryonic kidney (HEK) 293F cells. Thus, Human-cl rhFVIII is the first rFVIII with human-like post-translational modifications (PTM) and does not contain antigenic residues (e.g. NeuGc or Gal-alpha1,3-Gal) that are present in rFVIII from non-human but hamster cell-lines like CHO or BHK. Furthermore, the manufacture of Human-cl rhFVIII is free of human or animal derived added materials, including albumin, which is in line with the most recent recommendations from regulatory and physician`s organizations. Human-cl rhFVIII has successfully entered the global clinical development programme and completed 1 of 2 clinical studies.
With the development of the HEK293F cell-line, Octapharma has the necessary resources and superior biotechnology platform in place to realize further treatment opportunities not only in our core area of hemophilia and other bleeding disorders, but also in the broader field of hematology. Our “Growth through innovations” pipeline strategy will enable us to meet the needs of even more patient groups by strengthening our portfolio with new products and by expanding our product portfolio into new therapeutic areas of hematology. For the latter, the development of recombinant G-CSF (Granulocyte colony-stimulating factor) from the HEK293F cell-line is thus representing another key R&D project in our research activities. G-CSF is a glycoprotein hormone, which stimulates the bone marrow to produce and release granulocytes and stem cells into the circulation. Current G-CSF products are all recombinant and manufactured in E. coli or hamster (CHO) cell-lines.
Established G-CSF indications are the reduction of the duration and incidence of neutropenia and infections in patients receiving chemotherapy after bone marrow transplantation and peripheral blood progenitor cell mobilization.
Making good great
Enhancing existing therapies is also a crucial mandate of our team. Improving the ease of use, convenience and flexibility in dosing are all important aspects of patient care, and we continually invest in making these enhancements possible across our entire product range.
R&D Pipeline Area
Human growth factor
H rFactor VIII
Human cell line based recombinant FVIII
New-high yield intravenous IG
New-high yield subcutaneous IG
Octanate 1000 5ml
1000 IU vials
Universal SD-Plasma for transfusion
Incl. dedicated prion removal step
Lyophilised SD plasma in bags
IV concentrate for inherited/ acquired defficiency
Recombinant granulocyte colony-stimulating factor