Best in Biotech 22 Jan 2024
Eight newly approved drugs that could become blockbusters in 2024
The much-awaited Drugs to Watch list by Clarivate is out. Let’s do a deep dive into the therapies that made the cut in 2024.
Hemophilia
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In Depth 16 Nov 2023
uniQure: is the gene therapy pioneer en route to becoming a biotech rebuff?
Once thought to be a frontrunner in gene therapy, uniQure’s fame may no longer be coveted, as the biotech looks to reorganize by slashing its workforce, in an attempt to stay afloat. But how did things go awry for the company that was once in its prime in the DNA game? The Danish company, which […]
News and Trends 30 Jun 2023
FDA approves BioMarin gene therapy for hemophilia A
BioMarin Pharmaceutical Inc. has announced that the United States Food and Drug Administration (FDA) has approved ROCTAVIAN (valoctocogene roxaparvovec-rvox) gene therapy for the treatment of adults with severe hemophilia A (congenital factor VIII (FVIII) deficiency with FVIII activity < 1 IU/dL) without antibodies to adeno-associated virus serotype 5 (AAV5) detected by an FDA-approved test. The […]
In Depth 18 Apr 2023
Gene therapy: a powerful tool for hemophilia B?
The approval of HEMGENIX, the first-ever gene therapy for hemophilia B, by the U.S. Food and Drug Administration (FDA) followed by the European Commission, has been momentous in rare disease therapeutic research, paving the way for various gene therapies that are currently being studied. As we observed World Hemophilia Day on April 17, let us […]
Interview 3 Mar 2023
Interview: how has the first gene therapy for hemophilia B transformed research?
Hemophilia B, a rare disease that affects more than 230,000 people worldwide is caused by the lack of or defective clotting protein factor IX often due to a spontaneous mutation in the F9 gene, leading to excessive bleeding. Until recently, routine prophylactic infusions of factor IX replacement therapy to maintain enough clotting factor to prevent […]
News and Trends 28 Feb 2023
European Commission approves first gene therapy for hemophilia B
The European Commission has approved the first gene therapy for the treatment of hemophilia B developed by biotech company CSL Behring. The one-time gene therapy etranacogene dezaparvovec sold under the brand name Hemgenix is manufactured by UniQure Inc. and distributed by CSL Behring. The gene therapy is used to treat severe and moderately severe hemophilia […]
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News and Trends 28 Feb 2023
GC Biopharma acquiring hemophilia rare disease pipeline
GC Biopharma Corp., a South Korean provider of biopharmaceutical products, has signed an asset purchase agreement with Catalyst Biosciences to acquire three programs related to the orphan hematology disorders. The agreement includes Marzeptacog alfa (MarzAA), an engineered factor VIIa, which is ready for phase 3 clinical stage development. In its previous clinical development trials, MarzAA […]
News and Trends 24 Feb 2023
Sanofi hemophilia drug gets FDA approval
The U.S. Food and Drug Administration (FDA) has approved Sanofi’s ALTUVIIIO [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl], previously referred to as efanesoctocog alfa, a first-in-class, high-sustained factor VIII replacement therapy. ALTUVIIIO is indicated for routine prophylaxis and on-demand treatment to control bleeding episodes, as well as perioperative management (surgery) for adults and children with hemophilia […]
News and Trends 16 Dec 2022
CSL closer to gene therapy treatments for hemophilia B patients in Europe
CSL says the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending conditional marketing authorization (CMA) of etranacogene dezaparvovec. Etranacogene dezaparvovec is a one-time gene therapy for the treatment of appropriate adults with hemophilia B that reduces the rate of annual bleeds with a single infusion. […]
News and Trends 24 Nov 2022
Gene therapy approval “game changer” in hemophilia B treatment
The approval of CSL’s gene therapy Hemgenix has ushered in a new era for hemophilia B treatment. With the addition of other approvals like Rocktavian and Upstaza, 2022 could be a turning point for the broader gene therapy field. Earlier this week, etranacogene dezaparvovec (Hemgenix) made history as the first gene therapy approved by the […]
News and Trends 23 Nov 2022
FDA approves CSL gene therapy for hemophilia B
CSL has revealed that the U.S. Food and Drug Administration (FDA) has approved HEMGENIX (etranacogene dezaparvovec-drlb), the first and only one-time gene therapy for appropriate adults with hemophilia B. HEMGENIX is approved for the treatment of adults with hemophilia B who currently use factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage or […]
News and Trends 2 Nov 2022
Enzyre secures €12M series A funding to help hemophilia patients test from home
Dutch company, Enzyre, which is developing ambulant diagnostic technology for blood coagulation testing, today (November 2) announced it has successfully raised €12 million ($11.9 million) in series A financing. The round was led by new investor, Oost NL, with participation from i&i Biotech Fund, Demcon Investment as technology partner and existing investors, Takeda Ventures and […]
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