* . *
  • About
  • Advertise
  • Privacy & Policy
  • Contact
Thursday, June 5, 2025
Earth-News
  • Home
  • Business
  • Entertainment
    Shows to Watch – The Advocate

    Must-See Shows You Can’t Miss!

    Fox News Entertainment Newsletter: Celebrities mentioned during Diddy’s high-profile sex trafficking trial – Fox News

    Fox News Entertainment Newsletter: Celebrities mentioned during Diddy’s high-profile sex trafficking trial – Fox News

    ‘Sinners,’ starring Michael B. Jordan, is now streaming on Prime Video – About Amazon

    Experience the Thrills of ‘Sinners’ Starring Michael B. Jordan – Now Streaming on Prime Video!

    California Mid-State Fair announces entertainment lineups for Frontier and Mission stages – KSBY News

    Exciting Entertainment Lineup Unveiled for California Mid-State Fair’s Frontier and Mission Stages!

    Spotify & OpenAI: Will Gen AI ‘Hollow Out’ Entertainment? – Technology Magazine

    Will Generative AI Transform the Future of Entertainment

    Author Richard Russo Will Speak At Sandwich Town Hall – CapeNews.net

    Join Us for an Inspiring Evening with Author Richard Russo at Sandwich Town Hall!

  • General
  • Health
  • News

    Cracking the Code: Why China’s Economic Challenges Aren’t Shaking Markets, Unlike America’s” – Bloomberg

    Trump’s Narrow Window to Spread the Truth About Harris

    Trump’s Narrow Window to Spread the Truth About Harris

    Israel-Gaza war live updates: Hamas leader Ismail Haniyeh assassinated in Iran, group says

    Israel-Gaza war live updates: Hamas leader Ismail Haniyeh assassinated in Iran, group says

    PAP Boss to Niger Delta Youths, Stay Away from the Protest

    PAP Boss to Niger Delta Youths, Stay Away from the Protest

    Court Restricts Protests In Lagos To Freedom, Peace Park

    Court Restricts Protests In Lagos To Freedom, Peace Park

    Fans React to Jazz Jennings’ Inspiring Weight Loss Journey

    Fans React to Jazz Jennings’ Inspiring Weight Loss Journey

    Trending Tags

    • Trump Inauguration
    • United Stated
    • White House
    • Market Stories
    • Election Results
  • Science
  • Sports
  • Technology
    Micron Technology (NASDAQ:MU) Stock Price Expected to Rise, UBS Group Analyst Says – MarketBeat

    UBS Analyst Predicts Surge in Micron Technology Stock Price!

    Domo to Participate in the D.A. Davidson Technology Summit – Business Wire

    Domo Set to Shine at the D.A. Davidson Technology Summit!

    When fiction becomes fact: 3 pieces of modern technology inspired by Star Trek – Redshirts Always Die

    From Screen to Reality: 3 Modern Technologies Inspired by Star Trek

    The Isfahan Center for Nuclear Technology – UCF, ZPP, and IRR10 – Alma Research and Education Center

    Exploring the Isfahan Center for Nuclear Technology: Innovations and Insights from UCF, ZPP, and IRR10

    Inside the tedious effort to tally AI’s energy appetite – MIT Technology Review

    Inside the tedious effort to tally AI’s energy appetite – MIT Technology Review

    Finland Set to Lead EU Quantum Technology Defense Project – IoT World Today

    Finland Set to Lead EU Quantum Technology Defense Project – IoT World Today

    Trending Tags

    • Nintendo Switch
    • CES 2017
    • Playstation 4 Pro
    • Mark Zuckerberg
No Result
View All Result
  • Home
  • Business
  • Entertainment
    Shows to Watch – The Advocate

    Must-See Shows You Can’t Miss!

    Fox News Entertainment Newsletter: Celebrities mentioned during Diddy’s high-profile sex trafficking trial – Fox News

    Fox News Entertainment Newsletter: Celebrities mentioned during Diddy’s high-profile sex trafficking trial – Fox News

    ‘Sinners,’ starring Michael B. Jordan, is now streaming on Prime Video – About Amazon

    Experience the Thrills of ‘Sinners’ Starring Michael B. Jordan – Now Streaming on Prime Video!

    California Mid-State Fair announces entertainment lineups for Frontier and Mission stages – KSBY News

    Exciting Entertainment Lineup Unveiled for California Mid-State Fair’s Frontier and Mission Stages!

    Spotify & OpenAI: Will Gen AI ‘Hollow Out’ Entertainment? – Technology Magazine

    Will Generative AI Transform the Future of Entertainment

    Author Richard Russo Will Speak At Sandwich Town Hall – CapeNews.net

    Join Us for an Inspiring Evening with Author Richard Russo at Sandwich Town Hall!

  • General
  • Health
  • News

    Cracking the Code: Why China’s Economic Challenges Aren’t Shaking Markets, Unlike America’s” – Bloomberg

    Trump’s Narrow Window to Spread the Truth About Harris

    Trump’s Narrow Window to Spread the Truth About Harris

    Israel-Gaza war live updates: Hamas leader Ismail Haniyeh assassinated in Iran, group says

    Israel-Gaza war live updates: Hamas leader Ismail Haniyeh assassinated in Iran, group says

    PAP Boss to Niger Delta Youths, Stay Away from the Protest

    PAP Boss to Niger Delta Youths, Stay Away from the Protest

    Court Restricts Protests In Lagos To Freedom, Peace Park

    Court Restricts Protests In Lagos To Freedom, Peace Park

    Fans React to Jazz Jennings’ Inspiring Weight Loss Journey

    Fans React to Jazz Jennings’ Inspiring Weight Loss Journey

    Trending Tags

    • Trump Inauguration
    • United Stated
    • White House
    • Market Stories
    • Election Results
  • Science
  • Sports
  • Technology
    Micron Technology (NASDAQ:MU) Stock Price Expected to Rise, UBS Group Analyst Says – MarketBeat

    UBS Analyst Predicts Surge in Micron Technology Stock Price!

    Domo to Participate in the D.A. Davidson Technology Summit – Business Wire

    Domo Set to Shine at the D.A. Davidson Technology Summit!

    When fiction becomes fact: 3 pieces of modern technology inspired by Star Trek – Redshirts Always Die

    From Screen to Reality: 3 Modern Technologies Inspired by Star Trek

    The Isfahan Center for Nuclear Technology – UCF, ZPP, and IRR10 – Alma Research and Education Center

    Exploring the Isfahan Center for Nuclear Technology: Innovations and Insights from UCF, ZPP, and IRR10

    Inside the tedious effort to tally AI’s energy appetite – MIT Technology Review

    Inside the tedious effort to tally AI’s energy appetite – MIT Technology Review

    Finland Set to Lead EU Quantum Technology Defense Project – IoT World Today

    Finland Set to Lead EU Quantum Technology Defense Project – IoT World Today

    Trending Tags

    • Nintendo Switch
    • CES 2017
    • Playstation 4 Pro
    • Mark Zuckerberg
No Result
View All Result
Earth-News
No Result
View All Result
Home Health

EMA Authorizes Hemophilia B Gene Therapy

June 1, 2024
in Health
EMA Authorizes Hemophilia B Gene Therapy
Share on FacebookShare on Twitter

In its May 2024 meeting, the European Medicines Agency (EMA) gave the go-ahead for Durveqtix to treat severe and moderately severe hemophilia B in adults who do not have factor IX inhibitors or detectable antibodies to variant adeno-associated virus serotype Rh74 (AAVRh74var). 

The agency has also recommended Adzynma for the treatment of ADAMTS13 enzyme deficiency in patients with congenital thrombotic thrombocytopenic purpura.

Durveqtix

The new gene therapy for hemophilia B, Durveqtix (fidanacogene elaparvovec; Pfizer), was given conditional marketing authorization on the basis that its immediate availability fulfils an unmet medical need and its benefit to public health outweighs the inherent risk of additional data still being required. The medicine was supported through the EMA’s Priority Medicines (PRIME) scheme. 

Fidanacogene elaparvovec is a blood coagulation factor gene therapy. It uses a recombinant adeno-associated viral serotype Rh74 (AAVRh74var) to deliver a functional copy of the Padua variant of human factor IX transgene. The expressed factor IX replaces the missing coagulation factor IX required for proper coagulation of the patient’s blood. 

Most of the currently authorized medicines for hemophilia B require frequent and lifelong intravenous infusions to prevent or treat bleeding. Patients need more new treatments that provide sustained bleed protection, reduce frequency of infusions, and improve their quality of life, explained the EMA’s Committee for Medicinal Products for Human Use (CHMP).

Durveqtix is delivered as a single infusion.

The CHMP recommendation was based on the results of an ongoing single-arm, open-label, phase 3 trial in 45 men with moderately severe or severe hemophilia B who tested negative for neutralizing antibody to AAVRh74var and received a single intravenous infusion of fidanacogene elaparvovec. The study compared the annualized bleeding rate, which included both treated and untreated bleeding events, in participants treated with gene therapy vs the period when they were treated with routine factor IX prophylaxis regimen, administered as part of standard care, in a lead-in study.

In the clinical trial, most patients treated with fidanacogene elaparvovec experienced fewer bleeding episodes than before treatment when they were taking standard factor IX prophylaxis. After administration of fidanacogene elaparvovec , most patients no longer needed factor IX replacement therapy, and this benefit was maintained for at least 2 years. 

Adzynma

The CHMP also recommended granting a marketing authorization under exceptional circumstances for Adzynma (rADAMTS13; Takeda), an enzyme replacement therapy indicated for the treatment of children and adults with congenital thrombotic thrombocytopenic purpura (TTP). In exceptional circumstances, an authorization may be granted subject to certain specific obligations, to be reviewed annually. This happens when the applicant can show that they are unable to provide comprehensive data on the efficacy and safety of the medicinal product owing to the rarity of the condition it is intended for, limited scientific knowledge in the area concerned, or ethical considerations involved in the collection of such data. 

The lack or a severe reduction of the von Willebrand factor (VWF) cleaving metalloprotease ADAMTS13 is associated with TTP. Adzynma is a synthetic version of the ADAMTS13 protein that works by replacing the missing or defective ADAMSTS13 enzyme. The treatment will be available as powder and solvent for solution for injection and is expected to reduce or eliminate the spontaneous formation of VWF-platelet microthrombi, responsible for platelet consumption and thrombocytopenia in patients with congenital TTP. 

The CHMP highlighted that compared with plasma-based therapies, the benefit of Adzynma is the reduction of acute and subacute TTP events, such as thrombocytopenia and microangiopathic hemolytic anemia, and TTP manifestations, including neurologic symptoms, renal dysfunction, and abdominal pain.

>>> Read full article>>>
Copyright for syndicated content belongs to the linked Source : Medscape – https://www.medscape.com/viewarticle/ema-authorizes-hemophilia-b-gene-therapy-2024a1000a9j

Tags: AuthorizeshealthHemophilia
Previous Post

Europe Drops UK ‘Mad Cow’ Blood Ban

Next Post

You can order four Apple Pencil Tips for just $12 right now

Iowa GOP Chair responds to report ranking Iowa as state with worst economy – KCRG

Iowa GOP Chair responds to report ranking Iowa as state with worst economy – KCRG

June 5, 2025
Shows to Watch – The Advocate

Must-See Shows You Can’t Miss!

June 5, 2025
Cattle Health Market Report 2025 – Global Industry Size, Share, Trends, Opportunity, and Forecast, 2020-2030: Impacts of Technological Advancements, Disease Outbreaks, Traceability and Food Safety – Yahoo Finance

Unlocking the Future of Cattle Health: Insights on Market Trends, Opportunities, and Innovations for 2025

June 5, 2025
Michigan anti-discrimination law would explicitly ban antisemitism under new proposal – Detroit Free Press

New Proposal Aims to Strengthen Michigan’s Anti-Discrimination Law by Explicitly Banning Antisemitism

June 5, 2025
Micron Technology (NASDAQ:MU) Stock Price Expected to Rise, UBS Group Analyst Says – MarketBeat

UBS Analyst Predicts Surge in Micron Technology Stock Price!

June 5, 2025
Senate votes to open RI to new sports betting apps. Would that boost state revenues? – The Providence Journal

Could New Sports Betting Apps Transform Rhode Island’s Revenue Landscape

June 5, 2025
Art meets ecology in the outback – UNSW Sydney

Where Art and Ecology Converge: A Journey Through the Outback

June 5, 2025
Tall ship brings ESA ocean science training to Nice – European Space Agency

Set Sail for Science: Tall Ship Anchors Ocean Research Training in Nice!

June 5, 2025
Immigration Research Shows Stephen Miller Wrong About American Science – Forbes

Debunking Stephen Miller: The Truth About Immigration’s Impact on American Science

June 5, 2025
Can Lifestyle Changes Save Lives in Colon Cancer? – Medscape

Transform Your Health: How Lifestyle Changes Can Combat Colon Cancer

June 5, 2025

Categories

Archives

June 2025
MTWTFSS
 1
2345678
9101112131415
16171819202122
23242526272829
30 
« May    
Earth-News.info

The Earth News is an independent English-language daily published Website from all around the World News

Browse by Category

  • Business (20,132)
  • Ecology (668)
  • Economy (683)
  • Entertainment (21,589)
  • General (15,265)
  • Health (9,725)
  • Lifestyle (685)
  • News (22,149)
  • People (683)
  • Politics (691)
  • Science (15,901)
  • Sports (21,187)
  • Technology (15,668)
  • World (668)

Recent News

Iowa GOP Chair responds to report ranking Iowa as state with worst economy – KCRG

Iowa GOP Chair responds to report ranking Iowa as state with worst economy – KCRG

June 5, 2025
Shows to Watch – The Advocate

Must-See Shows You Can’t Miss!

June 5, 2025
  • About
  • Advertise
  • Privacy & Policy
  • Contact

© 2023 earth-news.info

No Result
View All Result

© 2023 earth-news.info

No Result
View All Result

© 2023 earth-news.info

Go to mobile version