The Evolution of Hemophilia Treatment—And What It Means for BioMarin
Hemophilia is a condition where blood does not clot, and this condition is normally inherited. The condition is caused due to defects in a gene of the X chromosome, which is a clotting factor. Generally, the diseases are widely seen in males as the X chromosome is inherited from mother to baby boy. The disease is widely treated with replacement therapy and gene therapy. The other treatment which is used is medication. However, there are ways to reduce the risk of the condition, which include regular exercise and others. The condition can be prevented by taking preventive treatment by injection of clotting factor VIII for hemophilia A, or IX for hemophilia B.
Hemophilia is a hereditary genetic disorder which impairs body’s ability to control coagulation or blood clotting. In this disease, clotting factors control bleeding from a broken vessel is deficient; therefore, coagulation does not occur. Of the two types of hemophilia, hemophilia A has higher prevalence compared to the other variant hemophilia B. . Acquired hemophilia is a rare non genetic form of hemophilia in which autoantibodies develop against the plasma coagulation factor
Download PDF Brochure Study Here
In late 1960’s plasma-derived clotting factor was the option for treating hemophilia. The factor VIII and factor IX derived from pooled plasma allowed more accurate dosing. However, it presented a higher viral contamination risk, and the safety of these products became a concern when many people lost lives to infections such as HIV.
Recombinant clotting factors
In the 1990s, the cloning of the factor VIII gene allowed for recombinant human factor VIII production and proved to be a major breakthrough. It eliminated the risk of the transmission of blood-borne pathogens.
Recent advances in hemophilia therapy include the use of inhibitors and prophylaxis. Specifically, EHL (extended half-life) clotting factors extend the time between infusions.
Next generation therapies
Advanced investigational therapies include gene therapy and novel molecules. Hemophilia is a single gene disorder that holds a clear cause-and-effect relationship, and thus, gene therapy could be a real win for the disorder. Currently, Biogen (BIIB) and Sangamo Biosciences (SGMO), Shire (SHPG) through its subsidiary Baxalta, and Pfizer (PFE) are conducting research on gene therapy.
Gene therapy is expected to be a new paradigm for hemophilia patients because it’s one-off therapy and novel therapy might require weekly or monthly dosage. The former has proven its efficacy, whereas novel treatments have yet to establish efficacy. Novel therapy includes Bispecific antibodies, RNA silencing, and Anti-TFPI.
The key players influencing this research are:
Bayer AG, Biogen Idec, Biotest AG, CSL, Genentech, Inc., Kedrion S.p.A, Novo Nordisk A/S, Pfizer Inc., Sanofi, Octapharma AG.
Source: marketrealist & The Insight Partners