Review
Pharmacology & Pharmacy
Hannah A. Blair
Summary: Cipaglucosidase alfa (Pombiliti) is a two-component therapy for Pompe disease, developed by Amicus Therapeutics. It consists of a recombinant human acid alpha-glucosidase (GAA) product and the enzyme stabilizer miglustat. The therapy was approved in the EU as a long-term enzyme replacement therapy in combination with miglustat for the treatment of adult patients with late-onset Pompe disease. This article summarizes the milestones leading to the approval of cipaglucosidase alfa.
Article
Pharmacology & Pharmacy
Susan J. Keam
Summary: Efanesoctocog alfa, a recombinant Factor VIII concentrate, independent of von Willebrand factor, has been approved for the treatment of hemophilia A in the USA. It can be used for routine prophylaxis, on-demand treatment, and perioperative bleeding management.
Article
Pharmacology & Pharmacy
Susan J. Keam
Summary: This article summarizes the milestones in the development of olipudase alfa leading to its first approval for the treatment of patients with ASMD, including the drug's mechanism of action, approval status, and ongoing regulatory review.
Article
Pharmacology & Pharmacy
Sohita Dhillon
Summary: Avalglucosidase alfa is a novel drug developed for the treatment of Pompe disease, which has received approvals in the USA and EU, with regulatory reviews ongoing in the UK and Japan while clinical studies are being conducted worldwide.
Article
Pharmacology & Pharmacy
Hannah A. A. Blair
Summary: Efbemalenograstim alfa (Ryzneuta(& REG;)) is a subcutaneously administered recombinant fusion protein that has been approved in China for reducing infection in adult patients with non-myeloid malignant tumors who receive myelosuppressive anticancer drugs. It is currently under regulatory review for the management of chemotherapy-induced neutropenia in the EU and the USA.
Article
Clinical Neurology
Bartosz Karaszewski, Sebastian Szczyrba, Bartosz Jablonski, Dariusz Gasecki, Piotr Kraszewski, Adam Wyszomirski, Robert Kowalski, Wioletta Kaliszan, Malgorzata Dabrowska
Summary: This article presents a case of using andexanet alfa-rtPA therapy in a patient with non-large vessel occlusion acute ischemic stroke on rivaroxaban. The treatment was effective and provides valuable insights for managing selected clinical problems.
FRONTIERS IN NEUROLOGY
(2023)
Article
Cardiac & Cardiovascular Systems
Jr Tuman J. Milling, Saskia Middeldorp, Lizhen Xu, Bruce Koch, Andrew Demchuk, John W. Eikelboom, Peter Verhamme, Alexander T. Cohen, Jan Beyer-Westendorf, C. Michael Gibson, Jose Lopez-Sendon, Mark Crowther, Ashkan Shoamanesh, Michiel Coppens, Jeannot Schmidt, Pierre Albaladejo, Stuart J. Connolly
Summary: This study evaluated the safety and efficacy of andexanet alfa in patients with major bleeding caused by factor Xa inhibitors. The results showed that the drug reduced anti-FXa activity and achieved good or excellent hemostatic efficacy in 80% of the patients.
Article
Hematology
Farahnaz Rayatdoost, Till Braunschweig, Herbert Schoechl, Rolf Rossaint, Oliver Grottke
Summary: This study assessed the dose-related effectiveness of andexanet in reducing blood loss and improving survival in a porcine model with apixaban-induced bleeding. The results showed that andexanet dose-dependently decreased blood loss and increased survival rates. Lower andexanet doses were associated with a significant reduction in blood loss and improved survival compared to controls.
THROMBOSIS AND HAEMOSTASIS
(2023)
Article
Clinical Neurology
Carlin C. Chuck, Daniel Kim, Roshini Kalagara, Nathaniel Rex, Tracy E. Madsen, Leana Mahmoud, Bradford B. Thompson, Richard N. Jones, Karen L. Furie, Michael E. Reznik
Summary: Andexanet alfa was recently approved as a reversal agent for FXa inhibitors, but its impact on long-term outcomes in FXai-associated ICH is unknown. This simulation study explored the potential clinical implications of Andexanet alfa in FXai-associated ICH. The results suggest that even optimistic simulated hemostatic effects indicate the costs and potential benefits of Andexanet alfa should be carefully considered, and placebo-controlled randomized trials are needed before its definitive recommendation.
Article
Hematology
Alexander P. Benz, Lizhen Xu, John W. Eikelboom, Saskia Middeldorp, Truman J. Milling, Mark Crowther, Patrick Yue, Pamela Conley, Genmin Lu, Stuart J. Connolly
Summary: The study found that andexanet significantly decreased antifactor Xa activity and achieved good hemostasis in patients with acute major bleeding on edoxaban. The efficacy was similar to that observed in patients with bleeding on other anticoagulant drugs. The rate of thrombotic events in these patients was as expected.
THROMBOSIS AND HAEMOSTASIS
(2022)
Article
Medicine, General & Internal
Sebastian Rauch, Hans-Peter Mueller, Jens Dreyhaupt, Albert C. Ludolph, Jan Kassubek, Katharina Althaus
Summary: This study investigated the effectiveness of Andexanet alfa (AA) in treating acute FXa-inhibitor-associated non-traumatic ICH. The results showed that there was no hematoma expansion in the AA group, but the clinical outcomes remained worse. Therefore, larger randomized trials are needed to study the clinical outcomes of AA in these patients.
JOURNAL OF CLINICAL MEDICINE
(2022)
Article
Clinical Neurology
Andrew M. Demchuk, Patrick Yue, Elena Zotova, Juliet Nakamya, Lizhen Xu, Truman J. Milling, Tomoyuki Ohara, Joshua N. Goldstein, Saskia Middeldorp, Peter Verhamme, Jose Luis Lopez-Sendon, Pamela B. Conley, John T. Curnutte, John W. Eikelboom, Mark Crowther, Stuart J. Connolly
Summary: Andexanet alfa effectively reduced anti-FXa activity and demonstrated high hemostatic efficacy in patients with ICrH, suggesting significant benefits for this patient population.
Article
Pharmacology & Pharmacy
Sheridan M. Hoy
Summary: Motixafortide is a CXCR4 inhibitor used for mobilizing HSCs and treating cancer. It has been approved in the USA for use in combination with filgrastim to mobilize HSCs for collection and autologous transplantation. Motixafortide also has orphan drug designation for pancreatic cancer treatment in the EU and USA, as well as for acute myeloid leukemia treatment in the USA.
Article
Pharmacology & Pharmacy
Sohita Dhillon
Summary: Elranatamab is a bispecific antibody drug developed by Pfizer for the treatment of multiple myeloma. It activates T cells to kill myeloma cells by targeting CD3 on T cells and BCMA on myeloma cells. Elranatamab has received approval in the USA and is under regulatory review in other countries.
Article
Pharmacology & Pharmacy
Matt Shirley
Summary: Glofitamab is a bispecific monoclonal antibody developed by Roche for the treatment of B-cell non-Hodgkin lymphomas, including DLBCL. It received its first approval in Canada for relapsed or refractory DLBCL. The approval is also being sought in the EU and USA, with a positive opinion received in the EU. Global clinical development of glofitamab is ongoing for the treatment of non-Hodgkin lymphomas.
