Future Retirement Success
  • Politics
  • Business
  • Investing
  • Stocks
  • Politics
  • Business
  • Investing
  • Stocks

Future Retirement Success

Investing

Will the Precautionary FDA Slow‐​Walk Approval of a Promising New Gene Therapy?

by May 31, 2023
May 31, 2023

Jeffrey A. Singer

Gene therapy researchers recently reported an encouraging development that can bring new hope to people at risk for cardiovascular disease. But how long will Americans have to wait before they can use it?

CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) is a gene editing technology that holds great promise for curing disease. Put simply, a compound is injected into a patient to alter a gene in the DNA and halt a disease process. One version of CRISPR‐​based therapy is called “base editing,” which involves swapping out a single letter on the DNA molecule.

Verve Therapeutics is a Cambridge, MA‐​based start‐​up biotech company that has devised a way to use base editing to turn off the PCSK9 gene, which lowers LDL—“bad cholesterol”—a significant driver of cardiovascular disease. And it turns off the gene without changing the DNA. If this works as a “one‐​and‐​done” treatment to lower LDL, it can reduce the need for people to take long‐​term medication for the problem.

Most people currently take statin drugs to lower LDL. But statins don’t work for everyone, and some have intolerable side effects. Researchers have developed two FDA‐​approved PCSK9 gene inhibitor drugs that turn off the gene. But they must be injected, patients must take them regularly, and they are extremely expensive.

Verve Therapeutics injects nanoparticles into patients to turn off the PCSK9 gene. A preclinical trial on monkeys resulted in a 70 percent reduction in LDL cholesterol levels after just two weeks, with levels remaining low after a two‐​year follow‐​up. Because of this exciting development, Phase 1 trials on humans have been approved and are underway in New Zealand and the U.K. Alas, not so fast in the U.S. Last November, the Food and Drug Administration decided to put the clinical trial on hold, requesting that Verve provide the agency with much more data and modify the trial protocol.

If Verve’s breakthrough therapy eventually gets approved in New Zealand and the U.K., why shouldn’t Americans also be free to choose the treatment?

As Michael F. Cannon and I suggest here:

[A]t a minimum Congress should eliminate the FDA’s ability to restrict truthful speech by manufacturers about their products and allow American consumers to purchase drugs that have won approval from designated certification agencies, including foreign regulatory bodies. According to one study, recognizing drug approvals by regulatory bodies in Canada and Europe between 2000 and 2010 would have given U.S. consumers quicker access to 37 “novel” drugs for which “no other FDA‐​approved prescription medicine had the same mechanism of action,” including 10 drugs treating mostly orphan diseases “for which no alternative therapy was available in the USA.” Such recognition would have allowed U.S. consumers to access those drugs a median of 13.6 months earlier.

In a blog post last November, I pointed out how Switzerland, Australia, the European Union, and Israel don’t block their residents from using medical devices and drugs approved in other developed countries. Some lawmakers call this policy reform “reciprocity.”

With rising health care and pharmaceutical costs gaining increased attention from lawmakers across the political spectrum, it is time that they seriously consider this intermediate step toward respect for patient autonomy and freedom of choice in health care.

0
FacebookTwitterGoogle +Pinterest
previous post
CBDC vs. Crypto: What’s the Difference?
next post
Conflict Over a “Citi Bike” Illustrates Popularity of a New Mobility Alternative

You may also like

Who You Calling Far Right?

March 28, 2024

Policymakers Won’t Cool It on Protectionism

July 27, 2023

FERC’s Order No. 1920: A Costly Shell Game

May 16, 2024

Biden Didn’t Cause the Border Crisis, Part 3: Would...

January 16, 2025

The Supreme Court Strikes Down Biden’s Loan‐​Forgiveness Program

June 30, 2023

Smoothing the Path for Families Navigating the Changing...

June 25, 2024

Reducing Illegal Immigration

January 31, 2024

The “Digital Gold” Fallacy, or Why Bitcoin Can’t...

November 29, 2024

Devolve Federal Power to Reduce Division

February 7, 2025

In 1932-33 Leading Intellectuals Used ‘Dictatorial’ as a...

September 22, 2023

    Get free access to all of the retirement secrets and income strategies from our experts! or Join The Exclusive Subscription Today And Get the Premium Articles Acess for Free

    By opting in you agree to receive emails from us and our affiliates. Your information is secure and your privacy is protected.

    Recent Posts

    • TIMELINE: Inside the evolving relationship between Trump and Musk from first term to this week’s fallout

      June 7, 2025
    • Deadly drone wars are already here and the US is horribly unprepared

      June 7, 2025
    • Week Ahead: NIFTY’s Behavior Against This Level Crucial As The Index Looks At Potential Resumption Of An Upmove

      June 7, 2025
    • FLASHBACK: Musk accused Trump, GOP leaders of not wanting to cut spending — here’s where they said they would

      June 7, 2025
    • ‘Right down the line’: Medicaid reform in ‘big, beautiful bill’ divides lawmakers by party

      June 7, 2025
    • FAST distribution and IA

      June 7, 2025

    Categories

    • Business (8,152)
    • Investing (2,019)
    • Politics (15,562)
    • Stocks (3,135)
    • About us
    • Privacy Policy
    • Terms & Conditions

    Disclaimer: futureretirementsuccess.com, its managers, its employees, and assigns (collectively “The Company”) do not make any guarantee or warranty about what is advertised above. Information provided by this website is for research purposes only and should not be considered as personalized financial advice. The Company is not affiliated with, nor does it receive compensation from, any specific security. The Company is not registered or licensed by any governing body in any jurisdiction to give investing advice or provide investment recommendation. Any investments recommended here should be taken into consideration only after consulting with your investment advisor and after reviewing the prospectus or financial statements of the company.

    Copyright © 2025 futureretirementsuccess.com | All Rights Reserved