* . *
  • About
  • Advertise
  • Privacy & Policy
  • Contact
Saturday, September 27, 2025
Earth-News
  • Home
  • Business
  • Entertainment
    Cardi B Adds More Dates to Little Miss Drama Tour: ‘Y’all Making Me Work’ – Yahoo

    Cardi B Extends Little Miss Drama Tour: “Y’all Making Me Work

    ‘Today’: Sheinelle Jones Thanks Katie Couric for Support After Husband’s Death – CBS 19 News

    Sheinelle Jones Expresses Heartfelt Thanks to Katie Couric for Support After Husband’s Passing

    Sate your hunger at DBA’s Taste of Downtown – Bakersfield.com

    Indulge Your Cravings at DBA’s Taste of Downtown!

    Caesars Entertainment (CZR): Assessing Valuation After Times Square Casino Setback and Mounting Investor Concerns – simplywall.st

    Caesars Entertainment Faces Times Square Casino Hurdles as Investor Concerns Mount

    Why Hilaria Baldwin Has Found the ‘DWTS’ Process ‘Embarrassing’ At Times – WFXG

    Hilaria Baldwin Opens Up About the Embarrassing Moments on Her ‘DWTS’ Journey

    Harvest Fest 2025 – yadkinripple.com

    Celebrate the Bounty: Harvest Fest 2025 is Coming!

  • 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
    How Sustainable Technology is Shaping a Greener Future – Technology Magazine

    How Sustainable Technology is Driving the Revolution Toward a Greener Future

    Aurora police hope to add facial recognition technology to crime-fighting tools – CBS News

    Aurora Police Aim to Boost Crime-Fighting with New Facial Recognition Technology

    Autonomous Solutions shows off cutting-edge technology for the public – Cache Valley Daily

    Autonomous Solutions Unveils Cutting-Edge Technology for the Public

    Amazon to Pay $2.5 Billion in Prime Membership Settlement – The New York Times

    Amazon to Pay $2.5 Billion in Prime Membership Settlement – The New York Times

    What are we really gaining from technology? – Fast Company

    What Are We Really Gaining from Technology?

    TOMI Environmental Solutions, Inc. Expands SteraMist iHP Technology Services in Healthcare Sector with New Provider Partnership – Quiver Quantitative

    TOMI Environmental Solutions Accelerates SteraMist iHP Technology Expansion in Healthcare with New Provider Partnership

    Trending Tags

    • Nintendo Switch
    • CES 2017
    • Playstation 4 Pro
    • Mark Zuckerberg
No Result
View All Result
  • Home
  • Business
  • Entertainment
    Cardi B Adds More Dates to Little Miss Drama Tour: ‘Y’all Making Me Work’ – Yahoo

    Cardi B Extends Little Miss Drama Tour: “Y’all Making Me Work

    ‘Today’: Sheinelle Jones Thanks Katie Couric for Support After Husband’s Death – CBS 19 News

    Sheinelle Jones Expresses Heartfelt Thanks to Katie Couric for Support After Husband’s Passing

    Sate your hunger at DBA’s Taste of Downtown – Bakersfield.com

    Indulge Your Cravings at DBA’s Taste of Downtown!

    Caesars Entertainment (CZR): Assessing Valuation After Times Square Casino Setback and Mounting Investor Concerns – simplywall.st

    Caesars Entertainment Faces Times Square Casino Hurdles as Investor Concerns Mount

    Why Hilaria Baldwin Has Found the ‘DWTS’ Process ‘Embarrassing’ At Times – WFXG

    Hilaria Baldwin Opens Up About the Embarrassing Moments on Her ‘DWTS’ Journey

    Harvest Fest 2025 – yadkinripple.com

    Celebrate the Bounty: Harvest Fest 2025 is Coming!

  • 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
    How Sustainable Technology is Shaping a Greener Future – Technology Magazine

    How Sustainable Technology is Driving the Revolution Toward a Greener Future

    Aurora police hope to add facial recognition technology to crime-fighting tools – CBS News

    Aurora Police Aim to Boost Crime-Fighting with New Facial Recognition Technology

    Autonomous Solutions shows off cutting-edge technology for the public – Cache Valley Daily

    Autonomous Solutions Unveils Cutting-Edge Technology for the Public

    Amazon to Pay $2.5 Billion in Prime Membership Settlement – The New York Times

    Amazon to Pay $2.5 Billion in Prime Membership Settlement – The New York Times

    What are we really gaining from technology? – Fast Company

    What Are We Really Gaining from Technology?

    TOMI Environmental Solutions, Inc. Expands SteraMist iHP Technology Services in Healthcare Sector with New Provider Partnership – Quiver Quantitative

    TOMI Environmental Solutions Accelerates SteraMist iHP Technology Expansion in Healthcare with New Provider Partnership

    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

FDA Panel Reluctantly Backs Patisiran for ATTR Cardiomyopathy

September 14, 2023
in Health
FDA Panel Reluctantly Backs Patisiran for ATTR Cardiomyopathy
Share on FacebookShare on Twitter

FDA advisors conceded on Wednesday that the benefit-risk balance of patisiran (Onpattro) technically checks out in wild-type or hereditary transthyretin amyloid cardiomyopathy (ATTR-CM, also known as cardiac ATTR amyloidosis) — though they were unconvinced that the benefits are actually meaningful.

At the end of the day, the Cardiovascular and Renal Drugs Advisory Committee ultimately voted 9-3 that patisiran’s benefits outweigh its risks in ATTR-CM, with members expressing minimal concern about the safety of the RNA interference agent from Alnylam Pharmaceuticals. The panel’s majority-yes votes were commonly attributed to the technicality of the question.

There is a “light wind for benefit and no wind for risk, so if you’re asking do benefits outweigh the risks, the answer is yes,” said Edward Kasper, MD, of Johns Hopkins School of Medicine in Baltimore.

Kasper suggested that patisiran would likely be a “niche” product at best, however, as its evidence is limited to monotherapy and not as a rescue for patients not responding to tafamidis (Vyndaqel or Vyndamax), the existing standard-of-care medication indicated to reduce cardiovascular mortality and cardiovascular-related hospitalization in ATTR-CM.

The crux of the issue is whether patisiran has enough benefit in cardiomyopathy to merit an expansion of its existing indication for polyneuropathy in ATTR amyloidosis.

Committee chairperson Javed Butler, MD, MPH, MBA, of the University of Mississippi Medical Center in Jackson, said he voted no because he was not sure if patisiran’s benefits were clinically meaningful, a recurring remark among fellow panelists throughout the day.

Patisiran’s application for an expanded indication rests on the phase III APOLLO-B trial showing small gains in 6-minute walk test (6MWT) and health status and quality of life (as measured by the Kansas City Cardiomyopathy Questionnaire) — none meeting subjective thresholds for clinical significance — when comparing the gene silencer with placebo.

“What level of evidence would I give this? This wouldn’t be a I or IIa. It would be IIb. The level of evidence is not high here,” Kasper said.

Notably, APOLLO-B showed that these small benefits did not apply to people on background tafamidis. Like tafamidis, patisiran is intended to stop or slow the progression of cardiac amyloidosis.

The argument that nonresponders to tafamidis could be helped by having an alternate option in patisiran was shot down by Butler, who pointed out the negative interaction in the trial suggesting that patients in New York Heart Association class III — likely those progressing nonresponders — were a subgroup that did not see benefit in 6MWT.

“That makes the interpretation a little bit difficult, to say that the nonresponders are definitely going to respond to patisiran therapy,” he said.

Moreover, some said it would be an indirect safety concern if patients mistook patisiran to be a viable alternative to tafamidis. “I would hate to see any unintended consequences if patients don’t get put on tafamidis because of this drug, so I do think there could be harm,” said David Moliterno, MD, of the University of Kentucky Medical Center in Lexington, adding that he did ultimately vote yes.

And while there may not be direct safety risks with the drug, there is nevertheless potential harm and the cost, time, and effort needed for an IV infusion every 3 weeks for patisiran therapy, suggested C. Noel Bairey Merz, MD, of Cedars-Sinai Medical Center in Los Angeles, who voted no.

ATTR-CM is a rare, rapidly progressive disease caused by a gene mutation resulting in misfolded transthyretin protein and excess amyloid buildup in the heart and other organs.

Patisiran works by blocking the production of transthyretin protein and is already FDA approved for treating polyneuropathy associated with hereditary ATTR amyloidosis. It won this initial approval in 2018 based on its ability to slow the progression of peripheral neuropathy in patients with hereditary ATTR amyloidosis over 18 months in the APOLLO trial.

Some expect more from future therapies in this arena, as recently reported cases of spontaneous reversal suggest it is possible for amyloid to be cleared from the heart using antibody treatment, for a return to near-normal cardiac structure and function without scarring.

author['full_name']

Nicole Lou is a reporter for MedPage Today, where she covers cardiology news and other developments in medicine. Follow

>>> Read full article>>>
Copyright for syndicated content belongs to the linked Source : MedPageToday – https://www.medpagetoday.com/cardiology/chf/106325

Tags: healthPanelReluctantly
Previous Post

IMRT Best for Locally Advanced NSCLC, Study Suggests

Next Post

Acetazolamide May Help COPD Patients Reach New Heights

Little Spokane River Spill – Washington State Department of Ecology (.gov)

Little Spokane River Spill – Washington State Department of Ecology (.gov)

September 27, 2025
St. Pete City Council voices support to save shuttered Science Center – FOX 13 Tampa Bay

St. Pete City Council voices support to save shuttered Science Center – FOX 13 Tampa Bay

September 27, 2025
LDWF Announces Temporary Closure of the Nature & Science Center at Port Wonder – Louisiana Department of Wildlife and Fisheries (.gov)

Temporary Closure of the Nature & Science Center at Port Wonder Announced

September 27, 2025
The One Ingredient That Gives Chickpeas That Perfect Crunch – Yahoo

The Secret Ingredient That Makes Chickpeas Irresistibly Crunchy

September 27, 2025
How Sustainable Technology is Shaping a Greener Future – Technology Magazine

How Sustainable Technology is Driving the Revolution Toward a Greener Future

September 27, 2025
What happens when President Trump attends major sports events – and why he does it – The Athletic – The New York Times

What happens when President Trump attends major sports events – and why he does it – The Athletic – The New York Times

September 27, 2025
New Delhi 2025: Preview, stars and how to watch the World Championships – Paralympic.org

New Delhi 2025 World Championships: Top Athletes to Watch and How You Can Catch the Action

September 27, 2025
Stock market exodus to Wall Street hits 20-year high – EL PAÍS English

Stock market exodus to Wall Street hits 20-year high – EL PAÍS English

September 27, 2025
October Prime Day TV Deals: Elevate Your Entertainment Space With These Early Savings – CNET

October Prime Day TV Deals: Upgrade Your Entertainment Space with These Early Savings

September 27, 2025
Georgia’s Medicaid work requirement program spent twice as much on administrative costs as on health care, GAO says – North Carolina Health News

Georgia’s Medicaid Work Requirement Program Spent Twice as Much on Administration as on Patient Care, GAO Finds

September 27, 2025

Categories

Archives

September 2025
M T W T F S S
1234567
891011121314
15161718192021
22232425262728
2930  
« Aug    
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 (840)
  • Economy (860)
  • Entertainment (21,734)
  • General (17,276)
  • Health (9,903)
  • Lifestyle (873)
  • News (22,149)
  • People (862)
  • Politics (870)
  • Science (16,070)
  • Sports (21,360)
  • Technology (15,843)
  • World (842)

Recent News

Little Spokane River Spill – Washington State Department of Ecology (.gov)

Little Spokane River Spill – Washington State Department of Ecology (.gov)

September 27, 2025
St. Pete City Council voices support to save shuttered Science Center – FOX 13 Tampa Bay

St. Pete City Council voices support to save shuttered Science Center – FOX 13 Tampa Bay

September 27, 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