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Antibody-Based Therapies
Published in David E. Thurston, Ilona Pysz, Chemistry and Pharmacology of Anticancer Drugs, 2021
At the time of writing, three bsAbs have been approved for the treatment of different diseases. Catumaxomab (Removab™) was the first to be approved for the treatment of malignant ascites but was later withdrawn for economic reasons. Blinatumomab (Blyncito™), developed by Amgen, was the next to be approved in 2014 through the FDA’s Accelerated Approval Program for the treatment of Philadelphia chromosome (Ph) negative relapsed or refractory B-cell precursor ALL in adults. Later, in 2015, it was granted approval for the treatment of ALL in children, and then full approval for use in adults in 2017. In 2018, the FDA extended the approval for use in minimal residual disease-positive B-cell precursor ALL, with blinatumomab becoming the first agent to be approved for this condition. Currently, blinatumomab is the lead bsAb with many clinical trials still ongoing in various hematological malignancies. In 2018, a third bsAb, emicizumab-kxwh (Hemlimbra™), was approved for the treatment of hemophilia A without factor VIII inhibitor, and is not used as a cancer therapy.
New directions to develop therapies for people with hemophilia
Published in Expert Review of Hematology, 2023
Pooja Gogia, Michael Tarantino, Wolfgang Schramm, Louis Aledort
The first two key trials were carried out in patients with FVIII inhibitors were the HAVEN 1 and HAVEN 2. HAVEN 1 included 109 individuals ages 12 years or older and HAVEN 2 trial included 85 children aged 2 to 11 [86,87]. The ABR ranged between 0.2 and 2.9 and the rate of zero bleeding events ranged between 63% and 90% in both the trials. Following the promising results obtained in HAVEN 1 and 2, emicizumab has also been evaluated in patients without inhibitors. In the HAVEN 3 trial, 152 individuals ages 12 years or older with severe HA without inhibitor were enrolled. Study participants had much lower ABR than those not on prophylaxis [1.5 and 1.3 vs. 38.2] and much higher rates of zero bleeds (50% and 40% vs. 0%). In the subgroup of 48 patients previously treated with routine FVIII prophylaxis, transition to emicizumab reduced the ABR by 68% [88] .
Early real-world experience with emicizumab and concomitant factor VIII replacement products in adult males with Hemophilia A without inhibitors
Published in Journal of Medical Economics, 2022
Lorraine Cafuir, Adina Estrin, Er Chen, David Hinds, Patricia Prince, Jennifer Thorburn, Henry Mead, Christine L. Kempton
Clotting factor concentrates (CFCs) have historically been the treatment of choice for people with hemophilia A (PwHA) and hemophilia B for treating and preventing bleeding events. Treatment guidelines recommend CFC prophylaxis as the standard of care for individuals with severe HA3. The development of non-factor replacement therapies such as emicizumab provides an alternative prophylactic treatment approach. Emicizumab was initially approved for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients with HA with FVIII inhibitors in November 20174 and subsequently approved for prophylaxis in patients without inhibitors in October 20185. Emicizumab is a subcutaneous injection administered weekly, every 2 weeks, or every 4 weeks.
Bispecific antibodies for the treatment of hemophilia A
Published in Expert Opinion on Biological Therapy, 2022
In conclusion, emicizumab is a new non-clotting factor therapeutic for patients with hemophilia A, irrespective of the presence of FVIII inhibitor. Emicizumab mimicks the cofactor function of FVIIIa, and the mode of action is different from that of native FVIII. Long-term, real-world, clinical, and post-marketing studies supported the application of emicizumab in both pediatric and adult patients with or without inhibitors. Several issues remain, however, and further discussions are required regarding emicizumab prophylaxis, especially including longer-term real-world experiences, applications in early childhood, ITI supplemented with emicizumab, and monitoring methods. Nevertheless, new concepts and protocols for prophylactic treatment in hemophilia A are being considered in the modern emicizumab era.