News: FDA weighs novel pain drug amid opioid crisis

Tuesday, January 7, 2025

FDA weighs novel pain drug amid opioid crisis

Jan 07, 2025

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Driving The Day

A hand reaching for a pill bottle of OxyContin.

If FDA approved, a novel pain drug that its manufacturer says is nonaddictive could be an alternative to opioids. | Jessica Hill/AP

A PAIN-RELIEF INNOVATION? The FDA will decide later this month whether to approve a novel drug for acute pain that could offer a nonaddictive alternative to opioids — if patients can afford them, Lauren reports with POLITICO’s Carmen Paun.

Suzetrigine, the candidate drug by Vertex Pharmaceuticals, would be the first selective sodium channel blocker approved for pain. The drug works differently than existing sodium channel blockers like lidocaine — a local anesthetic commonly used by dentists — by targeting specific pain signals in the body, enabling patients to maintain abilities like motor function. Because it doesn’t affect the central nervous system directly, the company said it expects the drug won’t pose an addiction threat to patients.

But it’s unclear how much the medication would cost consumers at the pharmacy counter and whether drug-pricing middlemen will favor it on their formularies over well-known and cheap opioids.

Why it matters: The market entry of a nonopioid pain reliever would be a landmark development for the category, which hasn’t seen a new product offering in decades. Meanwhile, the opioid crisis killed more than 81,000 Americans in 2023, according to the CDC.

And the first iterations of novel drug classes don’t always perform well. Dr. Stephen Waxman, a Yale School of Medicine neurology professor who studies selective sodium channel blockers, likened suzetrigine and its potential to early statins that “were not terribly good, but they propelled important research.”

Remaining questions: Vertex is still studying suzetrigine’s efficacy for chronic pain, an area with a higher need. Mid-stage trial results released last month showed it didn’t perform better than a placebo in treating sciatica, a lower-back nerve pain that can linger for months or longer.

Investors at the investment banking firm William Blair called Vertex’s vow to advance clinical trials for that condition “aggressive and risky” given Phase II results.

“While we understand the hypothesis around site issues leading to placebo responses, management of placebo responses is always challenging in pain studies, and we are skeptical that management will be able to fully mitigate the responses with site selection/training or with an innovative study design,” they said in a Dec. 19 investor note.

What’s next: The FDA is slated to make a final call on Vertex’s application by Jan. 30.

IT’S TUESDAY. WELCOME BACK TO PRESCRIPTION PULSE. We hope you enjoyed the break — and D.C.’s first significant snow in a while!

Send your thoughts and tips to David Lim (dlim@politico.com or @davidalim) and Lauren Gardner (lgardner@politico.com or @Gardner_LM).

A message from AMAC Action:

The Inflation Reduction Act took billions from the Medicare Part D prescription drug program to fund electric vehicle tax credits and green energy investments, and now seniors’ prescription drug premiums are skyrocketing. Seniors are calling on Congress to pause the Inflation Reduction Act and return those funds back to Medicare where they belong. Visit PauseTheIRA.com.

Eye on the FDA

Surgeons work.

Among the slew of recommendations released by the FDA is one that aims to help prevent donor tissue from transmitting disease. | Getty Images

POUR ONE OUT FOR THE TREES — Welcome to 2025. The FDA released a torrent of 26 guidance documents Monday covering a range of policies from artificial intelligence to accelerated approval to tissue donation, Lauren and POLITICO’s Ruth Reader report.

Artificial intelligence: One draft guidance focuses on aspects of drug manufacturing that occur after a company discovers a drug and puts guardrails around AI models used to help create or support data submitted to prove a drug’s safety, effectiveness and quality.

Accelerated drug approval: The agency released draft guidance describing how the FDA will consider a confirmatory clinical trial “underway” for accelerated approvals.

Off-label uses: The FDA published final guidance on how pharmaceutical companies can communicate scientific information to health care providers about unapproved uses for a drug or device that was approved or cleared for different uses, commonly known as off-label use.

Tissue donation: The agency released recommendations to reduce the risk of disease transmission via donor tissue products.

NICOTINE PROPOSED RULE CLEARS OMB — The FDA’s plan to limit the amount of nicotine in combustible cigarette products cleared the White House Office of Management and Budget’s review “consistent with change” — meaning the proposed rule could be released any day.

“As the FDA has previously said, a proposed product standard to establish a maximum nicotine level to reduce the addictiveness of cigarettes and certain other combusted tobacco products, when finalized, is estimated to be among the most impactful population-level actions in the history of U.S. tobacco product regulation,” the FDA said in a statement.

TALC IN COSMETICS — The agency released a proposed rule on Dec. 26 to mandate standardized testing methods to detect and identify asbestos in talc-containing cosmetics, fulfilling a requirement from the 2022 law that overhauled FDA oversight of the product class a year late.

The proposal had been sitting at the White House Office of Management and Budget since early January 2024.

Talc and asbestos are found in the same rock types, raising the risk of cross-contamination. Check out Lauren’s July 2022 story on lawmakers’ efforts to increase cosmetics regulation for a refresher on the history of talc in personal care products.

In Congress

CARTER: HEALTH PACKAGE A PRIORITY — The top Republican on the House Energy and Commerce Health Subcommittee said Monday he hopes to “rush through” the health care package that President-elect Donald Trump and Elon Musk helped kill last year, POLITICO’s Ben Leonard reports.

“We’re going to make sure it happens,” Rep. Buddy Carter (R-Ga.) said. “My hope is we’ll be able to put it together and rush it through as soon as possible. If they want to use it in reconciliation as a pay-for, then so be it.”

A message from AMAC Action:

In the Courts

COMPOUNDERS HEAD TO COURT — The Outsourcing Facilities Association representing large manufacturers of compounded medicines is moving forward with its lawsuit challenging the FDA’s October and December determinations that a two-year shortage of a popular diabetes and weight-loss drug has resolved.

The group and the agency confirmed in a status report filed Jan. 2 in U.S. District Court in Texas that they wish to reopen the case, proposing a briefing schedule contingent upon the FDA providing OFA with information redacted from its Dec. 19 memo explaining its decision. The parties aim to file a motion for a protective order allowing the compounders to see that data by Jan. 15, they said.

Meanwhile: Judge Mark Pittman on Monday granted Eli Lilly’s Jan. 1 motion to intervene in the case, arguing that the association and the FDA don’t represent the company’s interests, which are directly implicated in the suit.

“Lilly has a protected interest in protecting the confidential information that it submitted to FDA that will likely be part of the administrative record,” the company’s lawyers wrote in their brief.

Scott Brunner, CEO of the Alliance for Pharmacy Compounding, said in a statement that Lilly’s brief contains claims that “mischaracterize” the ability of smaller retail compounders to legally prepare copies of FDA-approved drugs. The company argued that a product’s appearance on the agency’s drug shortage list is not a permissible circumstance.

“No doubt Lilly’s attorneys are betting that in a world in which courts’ deference to agency interpretation of statute has been weakened by the U.S. Supreme Court, a conservative court may not look friendly upon an agency defining via guidance the circumstances under which pharmacies can make copies of FDA-approved drugs,” he said in a statement. “But on this particular issue, the statute is clear in its delegation of authority to the agency to determine those circumstances.”

What’s next: Pittman will hold a hearing on the case Friday. He’s seeking to have OFA convert its motion for a preliminary injunction into a motion for summary judgment.

Document Drawer

The FDA’s Office of Pediatric Therapeutics is holding a public meeting on May 15 to discuss the implementation of the Best Pharmaceuticals for Children and Pediatric Research Equity programs, which encourage development of pediatric drugs.

The CBO posted an analysis examining how much the increased use of gene therapies for sickle cell disease could cost the federal government.

WHAT WE'RE READING

Rep. Steve Cohen (D-Tenn.) took to USA Today to write about his experience contracting polio in 1954, arguing that Robert F. Kennedy Jr. should not be put in charge of Americans’ health.

The first U.S. avian flu fatality was reported Monday by Louisiana’s Department of Public Health.

A message from AMAC Action:

The IRA took money from Medicare Part D to fund EV tax credits under the guise of a fake “drug price negotiation.” As premiums are skyrocketing, seniors are demanding their money back.

70% of seniors are calling on Congress to pause the Inflation Reduction Act.

85% of them want the money that was taken from Medicare to fund EV tax credits to be returned to Medicare where it belongs.

Congress: It’s time to pause the IRA and fix what it broke, first by giving money back to Medicare.

Visit PauseTheIRA.com to learn more.