Factor VIII

Octapharma AG: Indirect comparison data indicate that personalised prophylaxis with Nuwiq® may lead to fewer patients with haemophilia A having bleeds than with other recombinant factor VIII products

Retrieved on: 
Tuesday, October 31, 2023
Haemophilia, Factor VIII, European Journal, PROPEL, PROTECT, Exercise, Georgetown University Medical Center, VIII, Georgetown Lombardi Comprehensive Cancer Center, Efmoroctocog alfa, Patient, Pharmaceutical industry, Dentistry

The analysis used data from prospective clinical trials of personalised prophylaxis in adults with severe haemophilia A.

Key Points: 
  • The analysis used data from prospective clinical trials of personalised prophylaxis in adults with severe haemophilia A.
    Dr Kessler commented, "This MAIC exercise provides important comparative data on the efficacy and consumption of various recombinant FVIII products in adults with haemophilia A on personalised prophylaxis.
  • The results can be considered by clinicians and patients when treatment decisions are being made."
  • MAICs are a way of comparing the efficacy of different medical interventions or products in the absence of head-to-head randomised trials3.
  • In addition, a short video summarising the results is available in the online version of the article.

Baudax Bio Announces Corporate Update

Retrieved on: 
Wednesday, October 18, 2023
FDA, IND, Consultant, Neuromuscular blocking agents, MD, Autoantibody, Acquisition, Research, Drug development, Quality of life, Clinical trial, Haemophilia A, Factor VIII, Patient, Pharmaceutical industry, Haemophilia

MALVERN, Pa., Oct. 18, 2023 (GLOBE NEWSWIRE) -- Baudax Bio, Inc. (the “Company” or “Baudax Bio”) (NASDAQ: BXRX), a biotechnology company focused on developing T cell receptor (“TCR”) therapies utilizing human regulatory T cells (“Tregs”), as well as a portfolio of clinical stage Neuromuscular Blocking Agents (“NMBs”) and an associated reversal agent, today announced that following recent shareholder approval of corporate actions related to its June 29 2023 acquisition of TeraImmune, the Company is now poised to initiate clinical development of TI-168, the Company’s next-generation, FVIII specific Treg therapy designed to reliably and effectively address Hemophilia A patients with FVIII inhibitors. The Company anticipates initiating its Phase 1/2a clinical study of TI-168 in Hemophilia A patients with FVIII inhibitors with a modest initial budget in Q1 of 2024.

Key Points: 
  • The Company anticipates initiating its Phase 1/2a clinical study of TI-168 in Hemophilia A patients with FVIII inhibitors with a modest initial budget in Q1 of 2024.
  • “We are very pleased with the broad support received in last week’s special shareholder meeting, during which shareholders approved all remaining issues related to our acquisition of TeraImmune,” said Gerri Henwood, President and Chief Executive Officer of Baudax Bio.
  • “We believe this combination, which brings together TeraImmune’s research expertise with Baudax’s drug development experience for the initiation of clinical development of TI-168, represents the best path forward to create shareholder value.
  • This product candidate previously had an IND filed seeking to treat human subjects that received regulatory clearance, and has also recently been granted Orphan Drug Designation by the FDA.

Press Release: Once-weekly ALTUVIIIO® approved in Japan as a new class of factor VIII therapy for hemophilia A

Retrieved on: 
Monday, September 25, 2023
US Foods, Bleeding, MHLW, Factor VIII, European Medicines Agency, ABR, Quality of life, Haemophilia, Fast Track, Coordinated Specialty Care, Arthralgia, European Commission, Marketing, Breakthrough therapy, Orphan drug, Patient, Physician, Risk, VIII, Sanofi, Joint, MAA, FDA, Haemophilia A, Chronic pain, Headache, Food, Birth control, Pharmaceutical industry, Welfare, Health

The Japanese Ministry of Health, Labor, and Welfare (MHLW) has granted marketing authorization for ALTUVIIIO®[Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein], a first-in-class, high-sustained factor VIII replacement therapy.

Key Points: 
  • The Japanese Ministry of Health, Labor, and Welfare (MHLW) has granted marketing authorization for ALTUVIIIO®[Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein], a first-in-class, high-sustained factor VIII replacement therapy.
  • ALTUVIIIO is indicated for control of bleeding tendency in patients with hemophilia A (factor VIII deficiency).
  • ALTUVIIIO was also recently approved by the Taiwan Food and Drug Administration for treatment of adults and children with hemophilia A on August 31, 2023.
  • The high-sustained factor activity levels will enable patients and physicians to reimagine living with hemophilia.

Precision BioLogic's Factor VIII Deficient Plasma with VWF Now FDA-Cleared for Sale in U.S.

Retrieved on: 
Monday, September 18, 2023
Factor VIII, Haemophilia, Research, Biologic, ISTH, Von Willebrand factor, Plasma, VWF, Traction, Hemostasis, VIII, FDA, Vaccine, EU, NS

The latest in Precision BioLogic's family of factor deficient plasmas, CRYOcheck Factor VIII Deficient Plasma with VWF is intended for use in clinical laboratories to identify factor VIII (FVIII) deficiency in human plasma and aid in the management of hemophilia A.

Key Points: 
  • The latest in Precision BioLogic's family of factor deficient plasmas, CRYOcheck Factor VIII Deficient Plasma with VWF is intended for use in clinical laboratories to identify factor VIII (FVIII) deficiency in human plasma and aid in the management of hemophilia A.
  • CRYOcheck Factor VIII Deficient Plasma with VWF comes in a convenient frozen format, which eliminates reconstitution errors and reduces preparation time.
  • CRYOcheck Factor VIII Deficient Plasma with VWF launched in Canada, the EU, UK, Australia and New Zealand in 2021.
  • "CRYOcheck Factor VIII Deficient Plasma with VWF offers labs a readily available and reliable alternative to congenital FVIII deficient plasmas.

Press Release: ALTUVIIIO late-breaking data at ISTH demonstrates highly effective bleed protection in children with severe hemophilia A with once-weekly dosing

Retrieved on: 
Sunday, June 25, 2023
Injection, SHL, Maintenance, US Foods, Pediatrics, Bleeding, Freedom, Allergy, Factor VIII, European Medicines Agency, ABR, Fever, Quality of life, Haemophilia, Pharmacokinetics, Fast Track, Associate, European Commission, Marketing, Embolism, Orphan drug, Rare, Medical College of Wisconsin, Patient, Standard of care, VIII, Risk, Blood, Anaphylaxis, Annual general meeting, Sri Siddhartha Medical College, Joint, EHL, Thrombosis, MAA, Upper respiratory tract infection, FDA, Haemophilia A, Chronic pain, International Society, Coagulopathy, Safety, Dietary supplement, Vaccine, Birth control, Pharmaceutical industry, ISTH

XTEND-Kids data confirm the efficacy and safety profile of ALTUVIIIO with simple, weekly 50 IU/kg dosing for both adults and children

Key Points: 
  • XTEND-Kids data confirm the efficacy and safety profile of ALTUVIIIO with simple, weekly 50 IU/kg dosing for both adults and children
    Paris, June 25, 2023.
  • These data confirm that a once-weekly 50 IU/kg dose of ALTUVIIIO provides highly effective bleed protection in both children and adults and can be used across clinical scenarios.
  • Achieving high-sustained factor activity with once weekly dosing means a freedom from the tradeoffs between treatment burden and efficacy we often see in treating severe hemophilia A.
  • The most common treatment-emergent adverse events (>10%) were SARS-CoV-2 test positive, upper respiratory tract infection, and fever (pyrexia).

BioMarin to Present ROCTAVIAN™ (valoctocogene roxaparvovec) Data from Longest and Largest Hemophilia Gene Therapy Clinical Trial Program at the International Society on Thrombosis and Haemostasis (ISTH) 2023 Congress

Retrieved on: 
Thursday, June 22, 2023
Transfer, Additive effect, Impact, Life, DSM-IV codes, BioMarin Pharmaceutical, Factor VIII, Requirement, Quality of life, Haemophilia, Patient, European Commission, Patient-reported outcome, Congress, SAN, International Society, PDUFA, Thrombosis, Health, FDA, Haemophilia A, EC, Exercise, BLA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Food, Safety, Pharmaceutical industry, Medical device, Vaccine, ISTH, New Data Seal

SAN RAFAEL, Calif., June 22, 2023 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, today announced it will present new data from the ROCTAVIAN™ (valoctocogene roxaparvovec) gene therapy clinical trial program. These data will be shared this week in four oral presentations and two posters at the 31st Congress of the International Society on Thrombosis and Haemostasis (ISTH) in Montreal, Canada.

Key Points: 
  • These data will be shared this week in four oral presentations and two posters at the 31st Congress of the International Society on Thrombosis and Haemostasis (ISTH) in Montreal, Canada.
  • The U.S. Food and Drug Administration (FDA) has set a PDUFA Target Action Date of June 30, 2023, for the Company's Biologics License Application (BLA) for valoctocogene roxaparvovec gene therapy.
  • The European Commission (EC) granted conditional marketing authorization under the brand name ROCTAVIAN on August 24, 2022.
  • "These data presented at ISTH will highlight the clinical impact of ROCTAVIAN for people living with severe hemophilia A more than three years post-infusion."

Pfizer Announces Positive Marstacimab Results from Pivotal Phase 3 Hemophilia A and B Trial

Retrieved on: 
Tuesday, May 30, 2023
Biotechnology, FDA, Other Health, Health, General Health, Pharmaceutical, Oncology, Other Science, Research, Science, Clinical Trials, Inc., Factor IX, Bleeding, Factor VIII, PFE, ABR, Haemophilia, Pfizer, FIX, Assistant, McMaster University, Patient, Data, TFPI, Purpura, NYSE, Disease, Pharmaceutical industry

Pfizer Inc. (NYSE: PFE) today announced that the pivotal Phase 3 BASIS clinical trial ( NCT03938792 ) evaluating marstacimab has met its primary endpoints, having demonstrated statically significant and clinically meaningful effects.

Key Points: 
  • Pfizer Inc. (NYSE: PFE) today announced that the pivotal Phase 3 BASIS clinical trial ( NCT03938792 ) evaluating marstacimab has met its primary endpoints, having demonstrated statically significant and clinically meaningful effects.
  • “These results support the potential for marstacimab to become the first once-weekly non-factor treatment for people with hemophilia B and a treatment option that helps address the diverse needs of patients with hemophilia A or B without inhibitors.
  • The safety profile for marstacimab was consistent with Phase 1/2 results and treatment was generally well-tolerated.
  • Pfizer currently has three Phase 3 programs investigating novel treatment options for people living with hemophilia.

2seventy bio and Novo Nordisk Collaboration Delivers Key Proof of Concept Data, Triggering $15 Million Preclinical Milestone in In Vivo Gene Editing Hemophilia A Program

Retrieved on: 
Monday, May 1, 2023
Science, Biotechnology, Research, Oncology, General Health, Health, Genetics, Other Health, Bluebird bio, Nordisk, Factor VIII, Gene editing, Disclosure, Haemophilia, Coagulopathy, Messenger, Research and development, Office of Rare Diseases Research, Development, Patient, Vivo, Novo Nordisk, LNP, Megat Burhainuddin, Genome, Pharmaceutical industry, Vaccine

The milestone is a result of delivering key pre-clinical proof of concept data that the Company plans to present during a virtual Research and Development deep dive on May 19, 2023.

Key Points: 
  • The milestone is a result of delivering key pre-clinical proof of concept data that the Company plans to present during a virtual Research and Development deep dive on May 19, 2023.
  • “We are excited about the continued progress in this program based on our internally developed in vivo gene editing platform.
  • 2seventy bio is responsible for megaTAL development and expression using their in vivo mRNA platform.
  • 2seventy bio will host a virtual R&D deep dive on Friday, May 19, from 10:00 a.m. – 11:30 a.m.

Positive topline results from pivotal XTEND-Kids phase 3 study of efanesoctocog alfa in children under 12 years of age with haemophilia A: Swedish Orphan Biovitrum AB

Retrieved on: 
Thursday, March 2, 2023
Research and development, Doctor of Philosophy, Safety, Haemophilia, Factor VIII, Patient, Genetic disorder, Bleeding, Pain, Standard of care, MD, ABR, Civil service commission, Von Willebrand factor, Medicine, Pharmacokinetics, Development, Male, Swedish Orphan Biovitrum, Medical Affairs Bureau, European Commission, VIII, Pharmaceutical industry, Medical device, Sanofi, EU

No factor VIII inhibitors were observed in the 74 children enrolled in the study, of which 65 experienced at least 50 exposure days.

Key Points: 
  • No factor VIII inhibitors were observed in the 74 children enrolled in the study, of which 65 experienced at least 50 exposure days.
  • Haemophilia A is a rare, genetic disorder in which the ability of a person's blood to clot is impaired due to a lack of factor VIII.
  • Combined with the XTEND-1 phase 3 trial, these results will provide the basis for regulatory submission in the EU.
  • Efanesoctocog alfa was granted orphan designation by the European Commission in June 2019 and the medicine was recently approved in the US .

Positive topline results from pivotal XTEND-Kids phase 3 study of efanesoctocog alfa in children under 12 years of age with haemophilia A: Swedish Orphan Biovitrum AB

Retrieved on: 
Thursday, March 2, 2023
Research and development, Doctor of Philosophy, Safety, Haemophilia, Factor VIII, Patient, Genetic disorder, Bleeding, Pain, Standard of care, MD, ABR, Civil service commission, Von Willebrand factor, Medicine, Pharmacokinetics, Development, Male, Swedish Orphan Biovitrum, Medical Affairs Bureau, European Commission, VIII, Pharmaceutical industry, Medical device, Sanofi, EU

No factor VIII inhibitors were observed in the 74 children enrolled in the study, of which 65 experienced at least 50 exposure days.

Key Points: 
  • No factor VIII inhibitors were observed in the 74 children enrolled in the study, of which 65 experienced at least 50 exposure days.
  • Haemophilia A is a rare, genetic disorder in which the ability of a person's blood to clot is impaired due to a lack of factor VIII.
  • Combined with the XTEND-1 phase 3 trial, these results will provide the basis for regulatory submission in the EU.
  • Efanesoctocog alfa was granted orphan designation by the European Commission in June 2019 and the medicine was recently approved in the US .