Factor IX

Orphan designation: Recombinant adeno-associated viral vector serotype S3 containing codon-optimised expression cassette encoding human coagulation factor IX variant Treatment of haemophilia B, 26/10/2018 Withdrawn

Retrieved on: 
Thursday, April 18, 2024
Eurostat, Diagnosis, Civil service commission, Brain, Disease, Coagulation, Patient, Committee, Knowledge, Internal, Regulation, Haemophilia, EMA, IRIS, Joint, Human, Wound healing, Clinical trial, European, Therapy, COMP, Bleeding, Spinal cord, Sponsor, European Commission, Immune system, Antibody, Safety, Factor IX, Blood, Marketing, Limited, Haemophilia B, Medicine, Pharmaceutical industry

Orphan designation: Recombinant adeno-associated viral vector serotype S3 containing codon-optimised expression cassette encoding human coagulation factor IX variant Treatment of haemophilia B, 26/10/2018 Withdrawn

Key Points: 


Orphan designation: Recombinant adeno-associated viral vector serotype S3 containing codon-optimised expression cassette encoding human coagulation factor IX variant Treatment of haemophilia B, 26/10/2018 Withdrawn

Orphan designation: fidanacogene elaparvovec Treatment of haemophilia B, 19/11/2018 Withdrawn

Retrieved on: 
Tuesday, April 9, 2024
Eurostat, Diagnosis, Civil service commission, Brain, Disease, Coagulation, Patient, Committee, Knowledge, Internal, Regulation, Haemophilia, EMA, IRIS, Joint, Human, Wound healing, European, COMP, Bleeding, Spinal cord, Antibody, Sponsor, European Commission, Immune system, Safety, Factor IX, Blood, Marketing, Haemophilia B, Medicine, Pharmaceutical industry

Orphan designation: fidanacogene elaparvovec Treatment of haemophilia B, 19/11/2018 Withdrawn

Key Points: 


Orphan designation: fidanacogene elaparvovec Treatment of haemophilia B, 19/11/2018 Withdrawn

Hemophilia B Pipeline Insight Report 2024: Comprehensive Analysis of Emerging Therapeutics - ResearchAndMarkets.com

Retrieved on: 
Tuesday, March 5, 2024
Pharmaceutical, Health, Cardiology, Knowledge, Bleeding, Factor IX, Haemophilia B, Catalog, Genetic disorder, Research, Patient, Haemophilia, Pharmaceutical industry

The "Hemophilia B - Pipeline Insight, 2024" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Hemophilia B - Pipeline Insight, 2024" report has been added to ResearchAndMarkets.com's offering.
  • Significant advances in the treatment and management of Hemophilia B are forthcoming, according to a new comprehensive research publication now available on our website.
  • The global coverage within this report transcends geographical boundaries, providing a universal perspective on the research and development efforts aimed at combatting Hemophilia B.
  • The report elucidates the complexity of Hemophilia B diagnosis and underscores the importance of comprehensive blood coagulation tests.

Octapharma Announces Availability of Balfaxar® For Warfarin Reversal in Urgent Surgery & Invasive Procedures

Retrieved on: 
Wednesday, January 3, 2024
Phase, Coagulation, Food, Emergency medicine, Risk, Vitamin K, PCC, Hemostasis, FDA, Warfarin, Bleeding, INR, Massachusetts General Hospital, Hospital, Thrombophilia, MD, Doctor of Philosophy, Factor X, Factor IX, Clinical trial, Prothrombin time, Trial of the century, Coagulation factor VII, Octapharma, VKA, Patient, Safety, Harvard Medical School, Thrombin, Pharmaceutical industry

PARAMUS, N.J., Jan. 3, 2024 /PRNewswire/ -- Octapharma USA has announced Balfaxar® (prothrombin complex concentrate, human-lans; marketed in Europe and Canada as octaplex®) is now available. The new non-activated four factor prothrombin complex concentrate (4F-PCC), approved by the U.S. Food and Drug Administration (FDA) this summer, is indicated for the urgent reversal of acquired coagulation factor deficiency induced by vitamin K antagonist (VKA, e.g., warfarin) therapy in adult patients with need for urgent surgery or invasive procedures.

Key Points: 
  • The new non-activated four factor prothrombin complex concentrate (4F-PCC), approved by the U.S. Food and Drug Administration (FDA) this summer, is indicated for the urgent reversal of acquired coagulation factor deficiency induced by vitamin K antagonist (VKA, e.g., warfarin) therapy in adult patients with need for urgent surgery or invasive procedures.
  • Octapharma has the Balfaxar® production flexibility to ensure consistent supply for hospitals and medical providers.
  • "Octapharma has the production flexibility to ensure consistent supply for hospitals and medical providers now and in the future.
  • Balfaxar® met the primary endpoint of hemostatic efficacy in patients on a vitamin K antagonist undergoing urgent surgery with significant bleeding risk.

FDA and EMA Accept Marstacimab Regulatory Submissions for the Treatment of Hemophilia A and B

Retrieved on: 
Monday, December 11, 2023
Biotechnology, FDA, Pharmaceutical, Health, Medicine, Safety, NYSE, Senior, PFE, Food, TFPI, Haemophilia, BLA, European Medicines Agency, European Commission, ASH, MAA, EMA, Civil service commission, Patient, Research and development, Research, Internal medicine, FIX, Rusyns, Exposition, Factor IX, Hemostasis, Development, Factor VIII, PDUFA, Society, Inc., Infection, Pharmaceutical industry, Pfizer

The European marketing authorization application (MAA) for marstacimab also passed validation and is currently under review by the European Medicines Agency (EMA).

Key Points: 
  • The European marketing authorization application (MAA) for marstacimab also passed validation and is currently under review by the European Medicines Agency (EMA).
  • If approved in the U.S. and EU, marstacimab is expected to become the first once-weekly subcutaneous treatment for people living with hemophilia B and the first treatment administered as a flat dose for people living with hemophilia A or B.
  • For appropriate patients living with hemophilia A and B, the goal of this treatment is to prevent potentially life-threatening bleeds with a once-weekly, subcutaneous flat-dose administration.
  • The submissions for marstacimab are based on efficacy and safety data from the Phase 3 BASIS trial ( NCT03938792 ).

Be Bio Announces New Preclinical Data Demonstrating its Novel Engineered B Cell Medicine (BCM), BE-101, Produces Active and Sustained Levels of Factor IX for the Treatment of Hemophilia B​

Retrieved on: 
Sunday, December 10, 2023
Health, Genetics, Research, Pharmaceutical, Science, Biotechnology, Partial thromboplastin time, Safety, Haemophilia, Food, HDR, Engineering, Plasma, Hematology, Bio-MEMS, Mortality, CCR5, Mouse, Multimedia, BCMS, PCR, Defence Scientific Advisory Council, World government, WFH, FIX, Committee, Availability, Investigational New Drug, World Federation of Hemophilia, Factor IX, Human services, Cas9, Bleeding, Person, Pain, Drug discovery, American Society of Hematology, DdPCR, Coronavirus Scientific Advisory Board, Society, Cell, BCM, Real-time, AAV, ELISA, Immunoglobulin G, Therapure BioPharma Inc, Pharmaceutical industry, Vaccine

“We are excited by these findings as we advance this novel BCM into clinical development to address enduring unmet medical needs for people living with hemophilia B.

Key Points: 
  • “We are excited by these findings as we advance this novel BCM into clinical development to address enduring unmet medical needs for people living with hemophilia B.
  • Many people living with hemophilia B still experience bleeding episodes, irreversible joint damage and pain with current extended-release factor IX biologic therapy.
  • “Our goal is to create a new, potentially transformative treatment option that will provide people with hemophilia B the freedom to live a normal life, free of limitations from hemophilia B.
  • The data confirmed the expected biodistribution of FIX-expressing BCMs in bone marrow tissue, where they engraft stably over time.

Marstacimab Phase 3 Data Presented at ASH 2023 Demonstrate Significant Bleed Reduction in Hemophilia A and B

Retrieved on: 
Sunday, December 10, 2023
Biotechnology, Health, Pharmaceutical, Clinical Trials, Cardiology, Safety, NYSE, Senior, PFE, Haemophilia, ASH, Risk, Research and development, Research, Internal medicine, FIX, ABR, Rusyns, Factor IX, Bleeding, Development, ATP, Factor VIII, Society, Inc., Infection, Pharmaceutical industry, Pfizer

The results from the BASIS trial demonstrated a statistically significant and clinically meaningful effect on annualized bleeding rate (ABR).

Key Points: 
  • The results from the BASIS trial demonstrated a statistically significant and clinically meaningful effect on annualized bleeding rate (ABR).
  • The findings were presented today at the 65th American Society of Hematology (ASH) Annual Meeting and Exposition in San Diego.
  • In addition to the BASIS study, fidanacogene elaparvovec and giroctocogene fitelparvovec are investigational gene therapy treatments being studied for the treatment of adults living with hemophilia B and hemophilia A, respectively.
  • Updated data from both gene therapy programs, including an oral presentation of four-year results from Pfizer’s Phase 1/2 study of giroctocogene fitelparvovec in adults living with severe hemophilia A, will be presented at the ASH meeting.

CSL Behring's HEMGENIX® (etranacogene dezaparvovec-drlb) Demonstrates at Three Years Post-Treatment Long-Term Durability, Safety and Greater Bleed Protection Versus Prophylactic Treatment in People Living with Hemophilia B

Retrieved on: 
Tuesday, December 12, 2023
CSL, European Economic Area, Health Canada, Safety, Food, Pediatrics, ALT, European Commission, University, ASH, Pathology, MHRA, Factor IX, History, Civil service commission, Patient, Abstract, European, Male, Alanine transaminase, FDA, ASX, Medication, ABR, Exposition, Bleeding, HIV, Phase, Health care, Society, Pharmaceutical industry, Medical device, Vaccine, Haemophilia

The Phase III, open label, single-dose, single arm HOPE-B trial included 54 male participants with severe or moderately severe hemophilia B with or without pre-existing AAV5 neutralizing antibodies.

Key Points: 
  • The Phase III, open label, single-dose, single arm HOPE-B trial included 54 male participants with severe or moderately severe hemophilia B with or without pre-existing AAV5 neutralizing antibodies.
  • HEMGENIX produced mean factor IX levels of 41.5 IU/dL at year one, 36.7 IU/dL at year two and 38.6 IU/dL at year three post-treatment.
  • Additionally, an analysis on the indirect treatment comparison of HEMGENIX and factor IX prophylaxis was recently published in Haemophilia.
  • Earlier this year, CSL announced the first person living with hemophilia B had received HEMGENIX.

Be Biopharma to Present New Preclinical Study Demonstrating Its Precision-Engineered B Cell Medicine Produces Active and Sustained Levels of Factor IX

Retrieved on: 
Thursday, November 2, 2023
Biotechnology, Pharmaceutical, Health, Other Health, FIX, BCM, Factor IX, Therapure BioPharma Inc, Engineering, BCMS, Research, Blood, Society, Hematology, Pharmaceutical industry, ASH

The research will describe the development of an ex vivo precision-engineered B Cell Medicine (BCM) that produces active and sustained levels of factor IX (FIX).

Key Points: 
  • The research will describe the development of an ex vivo precision-engineered B Cell Medicine (BCM) that produces active and sustained levels of factor IX (FIX).
  • The abstract is now available online in a special issue of the ASH official journal, Blood .
  • “We look forward to presenting our research at the ASH annual meeting, the pre-eminent global congress for scientific exchange in hematology,” said Dr. Rick Morgan, Chief Scientific Officer, Be Bio.
  • “Our team of dedicated researchers is inspired by the potential of this bold new class of medicines.”
    Details of the oral presentation are as follows:

CSL and uniQure Win 2023 Prix Galien USA Award

Retrieved on: 
Friday, October 27, 2023
ASX, FDA, CSL, European Economic Area, United Kingdom, Food, Health care, European Commission, Haemophilia, Medication, Hope, Health Canada, EC, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Development, IX, Civil service commission, MHRA, Research and development, Factor IX, Rod Humble, European, Patient, Pharmaceutical industry

KING OF PRUSSIA, Pa. and LEXINGTON, Mass., Oct. 27, 2023 /PRNewswire/ -- Global biotechnology leader CSL (ASX: CSL) and uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the companies have received the 2023 Prix Galien USA Award in the category of Best Product for Rare/Orphan Diseases for HEMGENIX® (etranacogene dezaparvovec-drlb).

Key Points: 
  • "CSL is humbled and honored to receive a Prix Galien award for HEMGENIX," said Dr. Bill Mezzanotte, Head of R&D, CSL.
  • CSL received approval from the U.S. Food and Drug Administration (FDA) for HEMGENIX in November 2022.
  • "CSL is humbled and honored to receive a Prix Galien award for HEMGENIX," said Dr. Bill Mezzanotte, Head of Research and Development, CSL.
  • The Prix Galien USA is America's preeminent prize acknowledging the leading-edge of scientific advances in life sciences.