Factor VIII

Octapharma shares new data at ISTH 2022 in its mission to meet the challenges faced by people with bleeding disorders

Retrieved on: 
Monday, July 4, 2022
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The data will be featured in eleven poster presentations and two Supported Symposia during this major international meeting.

Key Points: 
  • The data will be featured in eleven poster presentations and two Supported Symposia during this major international meeting.
  • Octapharma is proud to be a Gold Supporter of the ISTH 2022 Congress.
  • Safety and pharmacokinetics of a subcutaneous recombinant FVIII (OCTA101) in adult patients with severe haemophilia A. Presenting Author: Sigurd Knaub
    PB0559.
  • "Despite advances in the field, managing emergency bleeding and bleeding disorders still comes with considerable challenges.

BioMarin Receives Positive CHMP Opinion in Europe for Valoctocogene Roxaparvovec Gene Therapy to Treat Adults with Severe Hemophilia A

Retrieved on: 
Friday, June 24, 2022
People, Institute, Delayed onset muscle soreness, Science, CMA, Haemophilia, Patient, ALT, Adult, BMRN, Malignancy, EC, CHMP, University Clinical Centre in Gdańsk, European Commission, BioMarin Pharmaceutical, Committee, Nausea, Committee for Medicinal Products for Human Use, Company, Factor VIII, VIII, Public health, Haemophilia A, Pain, Compliance, EMA, Risk, Bleeding, AST, AE, Royal commission, Experimental Hematology, Antibody, Standard of care, Fatigue, Advanced Therapy Medicinal Product, United Kingdom Haemophilia Centre Doctors' Organisation, NASDAQ, Safety, Injury, Hope, Mutation, Conference, Transfusion medicine, Vaccine, Pharmaceutical industry, EU

SAN RAFAEL, Calif., June 24, 2022 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending conditional marketing authorization (CMA) for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A. A final approval decision, typically consistent with the CHMP recommendation, is expected from the European Commission in Q3 2022.

Key Points: 
  • ET
    SAN RAFAEL, Calif., June 24, 2022 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending conditional marketing authorization (CMA) for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A.
  • Roctavian is the first gene therapy to be recommended for approval in Europe for hemophilia A.
  • BioMarin has multiple clinical studies underway in its comprehensive gene therapy program for the treatment of hemophilia A.
  • Individuals with the most severe form of hemophilia A make up approximately 50 percent of the hemophilia A population.

Press Release: FDA grants efanesoctocog alfa Breakthrough Therapy designation for hemophilia A

Retrieved on: 
Wednesday, June 1, 2022
People, Science, SNY, Breakthrough therapy, European Commission, Headache, Bleeding, Intellectual property, Research, EMA, SEK, Von Willebrand factor, Social responsibility, LinkedIn, Annual report, Forward-looking statement, Nasdaq Stockholm, VIII, Form 20-F, FC, YouTube, Half-life, Pain, Arthralgia, Back pain, FDA, Male, Private Securities Litigation Reform Act, Fast Track, AMF, Review, COVID-19, Alfa, Quality of life, NASDAQ, SAN, SEC, File, Factor VIII, Medicine, Degenerative disease, Haemophilia, Safety, Pharmaceutical industry, Medical device, Sanofi, EU, Swedish Orphan Biovitrum

Breakthrough Therapy designation is designed to expedite the development and review of drugs in the US that target serious or life-threatening conditions.

Key Points: 
  • Breakthrough Therapy designation is designed to expedite the development and review of drugs in the US that target serious or life-threatening conditions.
  • The Breakthrough Therapy designation highlights efanesoctocog alfas potential to transform treatment for people with hemophilia A by providing higher protection for longer duration.
  • This designation supports the innovation of efanesoctocog alfa and acknowledges its potential to fulfill an unmet medical need for people living with hemophilia A.
  • The FDA granted efanesoctocog alfa Orphan Drug designation in August 2017 and Fast Track designation in February 2021.

Global Blood Plasma Derivatives Market Report 2022: A $40.5 Billion Market in 2021 - Industry Trends, Share, Size, Growth, Opportunity and Forecasts to 2027 - ResearchAndMarkets.com

Retrieved on: 
Wednesday, May 25, 2022
Health, Cardiology, Blood, Osmotic pressure, Immunodeficiency, Hypogammaglobulinemia, Thermoregulation, Haemophilia, Factor IX, Infection, Patient, Government, Hepatitis B, Hepatitis C, IX, Vitamin, COVID-19, Protease, Salt, Industry, Plasma, Awareness, Factor VIII, Immunity, Hospital, Mineral, Respiration, Hormone, Adoption, Coagulopathy, Protein, HIV, CAGR, Pharmaceutical industry, Blood product, Vaccine, Potash, Growth

The "Blood Plasma Derivatives Market: Global Industry Trends, Share, Size, Growth, Opportunity and Forecast 2022-2027" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Blood Plasma Derivatives Market: Global Industry Trends, Share, Size, Growth, Opportunity and Forecast 2022-2027" report has been added to ResearchAndMarkets.com's offering.
  • The global blood plasma derivatives market reached a value of US$ 40.5 billion in 2021.
  • How has the global blood plasma derivatives market performed so far and how will it perform in the coming years?
  • What is the structure of the global blood plasma derivatives market and who are the key players?

$89.76 Billion Blood Plasma Derivatives Markets - Global Opportunity Analysis and Industry Forecasts, 2021-2030 - ResearchAndMarkets.com

Retrieved on: 
Wednesday, May 4, 2022
General Health, Health, Other Health, Antibody, COVID-19, VIII, Coagulopathy, EUA, Plasma, Safety, Haemophilia, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Food, Immunodeficiency, Prevalence, IX, Criticism of the Food and Drug Administration, Hypogammaglobulinemia, Emergency Use Authorization, Patient, Factor IX, FDA, Blood, Factor VIII, Desmopressin, Adoption, Severe acute respiratory syndrome coronavirus 2, Conditional sentence, Income, CAGR, Infection, Growth, Awareness, Risk, Blood product, Pharmaceutical industry, Vaccine

The "Blood Plasma Derivatives Market by Product Type, Application, and End User: Global Opportunity Analysis and Industry Forecast, 2021-2030" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Blood Plasma Derivatives Market by Product Type, Application, and End User: Global Opportunity Analysis and Industry Forecast, 2021-2030" report has been added to ResearchAndMarkets.com's offering.
  • The factors that drive the growth of the blood plasma derivatives market include increase in prevalence of life-threatening diseases such as immunodeficiency diseases & hemophilia, rise in awareness toward blood & plasma donation, and growth in adoption of blood plasma derivatives products.
  • Moreover, other factors that are responsible for the growth of the plasma derived derivatives are increase in awareness of blood & plasma donation, rise in geriatric population, & higher adoption of the blood plasma derivative products.
  • Moreover, surge in prevalence of geriatric population, who are more vulnerable to chronic disease escalate the blood plasma derivatives market growth.

Global Coagulation Testing Lab and POC Market (2022 to 2026) - Analysis of Current and Emerging Technologies and Their Potential Market Applications - ResearchAndMarkets.com

Retrieved on: 
Tuesday, April 26, 2022
Health, Medical Devices, Cardiology, ACT, Computer, Factor VII, Factor IX, APTT, Thrombin time, Factor XIII, Coagulation, Heparin-induced thrombocytopenia, Fibrinogen, Protein C, The Queen's Award for Enterprise: Innovation (Technology) (2011), Bleeding time, Factor, Activated protein C resistance, Factor X, Prothrombin time, Factor VIII, Prekallikrein, Factor V, Antithrombin, POC, Protein S, Factor XII, Factor XI, Fine chemical, Plasmin

The "2022-2026 Global Coagulation Testing Lab and POC Market: US, Europe, Japan--Supplier Sales and Shares, Volume and Sales Segment Forecasts, Competitive Strategies, Innovative Technologies, Instrumentation Review" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "2022-2026 Global Coagulation Testing Lab and POC Market: US, Europe, Japan--Supplier Sales and Shares, Volume and Sales Segment Forecasts, Competitive Strategies, Innovative Technologies, Instrumentation Review" report has been added to ResearchAndMarkets.com's offering.
  • This comprehensive seven-country report is designed to help current suppliers and potential market entrants identify and evaluate business opportunities emerging in the global coagulation testing market during the next five years.
  • New specific and sensitive markers of coagulation will be increasingly used on automated instrumentation.
  • Coagulation testing will also become more standardized, offering opportunities for quality control products and services.

DGAP-News: Biotest AG: Biotest exceeds sales guidance in fiscal year 2021

Retrieved on: 
Friday, April 1, 2022
Plasma, Population, Therapy, VIII, EBT, Federal Ministry of Finance (Germany), III, Factor VIII, Marketing, Growth, Patient, Research, Next Level, Paul Ehrlich Institute, Board of directors, Czech Republic, EBIT, Federal Executive Boards, Tax, COVID-19, EAT, Annual report, Education, Acquisition, Security (finance), Fine chemical, Pharmaceutical industry, Health, Grifols

In fiscal year 2021, the Biotest Group generated revenues of 515.6 million, compared to 484.2 million in the previous year.

Key Points: 
  • In fiscal year 2021, the Biotest Group generated revenues of 515.6 million, compared to 484.2 million in the previous year.
  • This result is a consequence of growth in key sales markets due to the positive sales development of the main product Intratect(R).
  • EBIT amounted to - 47.1 million in fiscal 2021, compared to - 1.3 million in the previous year.
  • The Biotest Group's loss (EAT) for the financial year 2021 was - 63.4 million, compared to - 31.4 million in 2020.

Efanesoctocog alfa met primary and key secondary endpoints in pivotal study in hemophilia A, demonstrating superiority to prior factor prophylaxis treatment

Retrieved on: 
Wednesday, March 9, 2022
People, SEK, ABR, Haemophilia, Pharmacokinetics, SNY, Degenerative disease, Private Securities Litigation Reform Act, Nasdaq Stockholm, VIII, Half-life, Factor VIII, Social responsibility, Male, US, LinkedIn, Safety, Development, Observational study, Form 20-F, Orphan, Bleeding, FDA, Alfa, Arthralgia, Intellectual property, Patient, European Commission, SEC, FC, NASDAQ, Annual report, Research, EMA, Pain, Observation, Mayur Vihar Phase - 3, Back pain, Von Willebrand factor, Medicine, Forward-looking statement, AMF, Genetic disorder, Royal commission, Science, US Foods, COVID-19, Haemophilia A, YouTube, Food, Arm, SAN, Headache, Medical imaging, Birth control, Pharmaceutical industry, Hospital, Sanofi, Swedish Orphan Biovitrum, EU

The study met the primary endpoint, showing a clinically meaningful prevention of bleeds in people with severe hemophilia A receiving weekly prophylaxis with efanesoctocog alfa over a period of 52 weeks.

Key Points: 
  • The study met the primary endpoint, showing a clinically meaningful prevention of bleeds in people with severe hemophilia A receiving weekly prophylaxis with efanesoctocog alfa over a period of 52 weeks.
  • The key secondary endpoint was also met, demonstrating once-weekly efanesoctocog alfa was superior to prior prophylactic factor VIII replacement therapy, showing a statistically significant reduction in ABR based on intra-patient comparison.
  • Global Head of Development and Chief Medical Officer, Sanofi
    While advances have been made in the treatment of hemophilia, unmet medical needs still exist.
  • These positive topline data, showing a very low annualized bleeding rate, enhance efanesoctocog alfas potential to transform hemophilia A therapy.

Global Hemophilia Market (2022 to 2026) - Size, Trends & Forecast with Impact of COVID-19 - ResearchAndMarkets.com

Retrieved on: 
Wednesday, March 2, 2022
Health, Pharmaceutical, People, Risk, Patient, Prevalence, Population, Coagulopathy, Blood, Haemophilia, Injury, VIII, COVID-19, FIX, Male, Protein, Bleeding, Coronavirus, Incidence, Half-life, Factor VIII, Factor IX, Pharmaceutical industry

People with hemophilia have low levels of either factor VIII (8) or factor IX (9).

Key Points: 
  • People with hemophilia have low levels of either factor VIII (8) or factor IX (9).
  • The severity of hemophilia that a person has is determined by the number of factors in the blood.
  • The global hemophilia market has increased during the years 2017-2021.
  • Moreover, the market growth would succeed by various market trends like increasing innovation in gene therapy, development of novel hemophilia treatments, shifting focus to extended half-life th

ASC Therapeutics and Professor Steven W. Pipe Publish Expert Review on Novel Gene Therapies of Hemophilia A

Retrieved on: 
Tuesday, January 11, 2022
Health, Genetics, Other Health, Clinical Trials, Pharmaceutical, Biotechnology, Patient, Pathology, HC, Haemophilia, University, Haemophilia A, Liver, Clinical trial, FRG, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, US, Degenerative disease, Unfolded protein response, Allotransplantation, Safety, Pediatrics, Program, ASC, DOI, Immunotherapy, Learning, IND, DNA, Review, Advancing Human Rights, Lists of diseases, Factor VIII, Half-life, Therapy, Gene editing, Ministry of Education (Malaysia), Doctor of Philosophy, Multimedia, Coagulopathy, Protein folding, Food, Treatment, Maple syrup urine disease, FDA, VIII, Principal, Orphan, Biology, Medical device, Vaccine, Pharmaceutical industry, MD, Hemophilia A, ASC618, ASC Therapeutics, HEMOPHILIA A, ASC618, ASC THERAPEUTICS

This publication provides an update of scientific and clinical advances in gene replacement therapies for hemophilia A with focus on:

Key Points: 
  • This publication provides an update of scientific and clinical advances in gene replacement therapies for hemophilia A with focus on:
    This press release features multimedia.
  • Oscar G. Segurado, MD, PhD, Chief Medical Officer at ASC Therapeutics, added We are thrilled that the journal Expert Opinion in Biological Therapyhas published this seminal review to inform the medical and scientific community of these exciting developments with gene therapies in hemophilia A.
  • The major challenges of current treatment regimens, such the short half-life of hemophilia therapeutics with need for frequent intravenous injections, justify ongoing focus on gene replacement therapies.
  • ASC618 is an AAV8-based gene therapy for the treatment of hemophilia A, affecting approximately 1 of every 5000 live-born males.