Читать книгу Biologics, Biosimilars, and Biobetters - Группа авторов - Страница 112

Like the scientific developments that improved the production of insulin, blood coagulation factor products for the treatment of hemophilia have also undergone dramatic and important advances. Hemophilia A and B are bleeding disorders caused by dysfunction or deficiencies in coagulation factors VIII and IX that result in impaired blood clot formation and potential hemorrhage. In the 1950s, the only treatment option available was the infusion of human whole blood or plasma. Then in the mid‐1960s, scientists discovered how to collect factor products that allowed for collection in a more concentrated form. Donors were now able to donate plasma for production of Factor VIII for replacement. Industries developed around sourcing the material, and patients with hemophilia benefited significantly, being able to treat themselves in their home for the first time.²³

Unfortunately, the donated pool of plasma used in this process became a host for transmission of blood‐borne disease such as hepatitis and HIV. The development of recombinant factor was a way to avoid reliance on human‐produced product and to limit viral contamination. In 1984, factor VIII DNA was cloned and in 1992, the first recombinant FVIII product, Recombinate^®, was licensed for marketing.²³ Today, there are over a dozen recombinant products developed for hemophilia treatment, with subsequent iterations focusing on using less animal and human‐derived elements, limiting the development of inhibiting antibodies and transmission of blood‐borne pathogens. Recent drug development efforts have been aimed at extending the half‐life of factor products to allow for less frequent dosing, potentially resulting in greater compliance with prophylactic dosing and less reliance on acute bleeding treatment. Human cell lines have been used to produce the latest generation of factor products and genetic therapy is being studied, which could potentially require a single one‐time treatment.²³