Hemophilia is an inherited genetic disorder where the body loses control to stop bleeding caused by stress or any trauma. Deficiency of clotting factor VIII leads to Hemophilia A and the basic treatment prevailing in the market is to replenish the body with factor VIII. Replenishment is done by either preparation of recombinant factor VIII or from plasma derived factor VIII. The global market of Hemophilia is expected to grow by 5.6% annually between 2013 and 2024 and the competition is getting intense with various new players emerging in with better strategies for the treatment.
Key players treating Hemophilia:
There are some traditional players who have been leading the race and competing head on in the Hemophilia market. Top players in this list include: Bayer (Kovaltry, Bay- 94-9027), Novo Nordisk (Turoctocog alfa), and Baxalta (Adynovate). Bayer secured FDA and European commission’s approval for Kovaltry in 2016 to treat Hemophilia patients of all ages. Kovaltry replaced Bayer’s own Kogenate by having slightly extended half-life. Baxter’s FEIBA developed by Shire has a history of almost 30 years to control bleeding in Hemophilia patients with inhibitory antibodies against Factor VIII. Another player in line is Octapharma with Simoctocog alfa which has been given market authorisation in Canada and is approved in US, Australia, and Europe.
Bayer’s Kovaltry Vs Other drugs:
Kovaltry is closely competed by Novo Nordisk, another drug giant, generating around 10% of revenues from hemophilia therapies. Novo Nordisk was in competition already with its FDA & EMA approved Eptacog alpha (Novoseven) and its Turoctocog alfa is running into phase III clinical trial. Novo Nordisk has come up N8-GP which is a pegylated form of Turoctocog alfa. Pegylation improved the half-life of N8-GP by 1.5-fold of the other treatments. N8-GP is running into phase III clinical trials and Novo Nordisk is planning to file for its approval close next year. Pegylation seems to be a promising strategy acquired by many other top players and hence adding up to the competition for Novo Nordisk. For instance, Bayer and Baxalta have come up with pegylated products like Bay 94-9027 and Bax 855 respectively which are long acting recombinant factor VIII and are running into phase III clinical trials.
Basic limitation of the conventional regimen is higher dosing frequency requiring alternate day drug administration and the goal is to extend the half-life of administered product and lower the frequency of infusions.
Even though traditional players are stressing efforts on improving the treatment strategies, there is tough head-on competition in the market from new emerging players like Bioverativ’s Eloctate which promises reduced intake frequency of single dose in three to five days and half-life almost 1.5 times than of other regimens. Eloctate is developed from FC fusion technology where in factor VIII binds to human Immunoglobulin molecule which helps in extending half-life. Eloctate has already been approved by EMA for all ages and is ready to hit the market.
Alternative treatment options
Taking a different turn from conventional treatment regime is Chugai pharmaceutical’s and Roche’s collaborative product ACE910. It’s a bispecific antibody mimicking coagulation factor VIII even in the presence of factor VIII inhibitors. However, a couple of serious adverse events associated with ACE910 lifted the shares of its close competitors like Novo Nordisk and Shire (Baxalta). But with the risk mitigation strategies acquired by Roche, ACE910 is still regarded as a blockbuster candidate in Hemophilia space. Going with the culture of breaking old regimes, BioMarin is progressing with BMN 270, a gene therapy treatment for Hemophilia which will restore patient’s natural clotting capability. EMA regulatory committees viz. CAT and CHNP have agreed to give BMN 270 access to PRIME – an EMA’s initiative for Priority medicines.
All the new developments and initiatives have raised new hopes in Hemophilia space plus the recent rapid advances in the treatment strategies have led to the better understanding of the disease. The main aim is to enable precise and effective treatment strategy, irrespective of who wins the competition.
By Dr. Esha Pandita
Research Analyst, Science and Research @ Innoplexus