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‘A defining disease of this century,’ a Duchenne gene therapy decision, & the legal strategy behind all those pharma lawsuits

June 23, 2023
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Health

Diabetes will be 'a defining disease of this century'

STAT

The figures are enormous: The number of people with diabetes worldwide is set to more than double to 1.3 billion by 2050. By that time, about 1 in 10 people around the world are predicted to have the disease, representing a 60% surge in prevalence, according to a Lancet study in its series on global inequities in diabetes. "Diabetes will be a defining disease of this century," editors of the Lancet write in an editorial, and not one limited to high-income countries.

Rising rates will be driven by type 2 diabetes, and while obesity is a primary risk factor for type 2, the new GLP-1 drugs aren't the answer. Ozempic, Wegovy, and Mounjaro are highly effective in treating diabetes and obesity, but addressing diabetes worldwide requires not only medical interventions, but also broad changes to the environments people live in, the Lancet editors write. STAT's Elaine Chen has more.


reproductive health

Biden seeks to boost contraception access on eve of Dobbs' anniversary

President Biden will sign an executive order today directing health officials and other federal agencies to boost contraceptive access, almost one year after the U.S. Supreme Court's Dobbs decision overturned national abortion access, triggering a patchwork of state reproductive health care laws.

Biden's order instructs the Department of Health and Human Services to ensure people on commercial insurance are receiving contraception at no cost — a requirement under the Affordable Care Act — and consider moves to boost access in Medicare and Medicaid, though the latter is limited by how states build their programs. There also is no timeline to the order, which stops short of suggesting federal agencies implement new coverage and access requirements. Instead, the order directs HHS leaders to issue guidance on providing contraception and "best practices" to build awareness. Read more from STAT's Sarah Owermohle.


rare disease

Gene therapy for Duchenne muscular dystrophy wins conditional FDA approval

The FDA granted limited approval to a gene therapy for Duchenne muscular dystrophy yesterday, marking two firsts: It's the first gene therapy approved for the fatal disease in which boys progressively lose muscle. It's also the first time the agency has given a gene therapy accelerated approval, through which a drug is provisionally authorized based on a surrogate marker. That marker is dystrophin, and the therapy made by Sarepta delivers a shortened version of the gene that makes dystrophin but is broken in Duchenne.

While not a cure, there is hope the gene therapy will help preserve muscle before it's lost in a disease that affects about 1 in every 3,500 newborn boys. FDA has also limited the age of patients who may receive it. STAT's Jason Mast and Adam Feuerstein explain.



Closer Look

Divide and conquer: The strategy behind pharma's flurry of lawsuits challenging drug pricing reform

Have you been following the lawsuits filed by pharma companies and trade groups seeking to overturn a new law that allows Medicare to negotiate certain drug prices? The legal actions have been coming fast and furious, first from Merck and Bristol Myers Squibb, followed by the U.S. Chamber of Commerce and PhRMA. The courts where they filed are across the country and the arguments sound familiar. As STAT's Rachel Cohrs puts it, they're dividing and conquering to advance different legal arguments.

To wit: The Merck and Bristol Myers Squibb lawsuits, which rely on the same legal reasoning, were filed by the same firm and even a few of the same attorneys. The PhRMA and Chamber of Commerce suits were also similar to each other, despite different arguments. The scattershot approach could mean the U.S. Supreme Court may have to resolve conflicting decisions, legal experts told Rachel. Read more.


drug supply

Looming penicillin shortage most acute for pregnant adults with syphilis and their newborns

Supply chain problems combined with greater demand for the safest penicillin to treat syphilis in pregnancy and early childhood add up to a critical shortage. Pfizer, which manufactures Bicillin, said it will run out of the version for children as early as the end of this month and for adults in September. That could make already rising syphilis rates climb higher, racial disparities grow wider, and access to the drug in lower-income countries become harder.

"For pregnant adults, this really is the drug of choice," said Erin Fox of University of Utah Health. "Even if you have a penicillin allergy, they will work to desensitize you." The sexually transmitted infection, passed during pregnancy to a newborn, can cause birth defects or death. Having syphilis also weakens how well the body fights HIV and limits how well it responds to HIV treatments. STAT's Bree Iskandar and Abdullahi Tsanni have more.


policy

Medicare puts out new details for covering the Alzheimer's drug Leqembi

We may be near a turning point for a treatment that would slow the inexorable decline after an Alzheimer's diagnosis. Right now most people with mild cognitive impairment who would qualify for Eisai and Biogen's drug Leqembi are on Medicare, which says only those who are enrolled in clinical trials can receive the medication. But full FDA approval could come in just weeks. Yesterday Medicare released the first details about how it plans to collect data from doctors about the drug, which costs $26,500 a year without insurance:

  • Patient registry: Doctors must submit information about the patients' diagnosis, blood thinner use, scans or tests of amyloid plaques, any brain swelling or bleeding, and cognitive test results.
  • Genotyping patients: It's not required, but may be on offer because patients with two copies of the e4 variant of the ApoE gene are at higher risk of brain swelling or bleeding.

STAT's Rachel Cohrs has more.


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What we're reading

  • At a defunct power station, the U.K. and Moderna celebrate a partnership meant to be an engine for science, STAT
  • In 'Cancer Alley,' a key Biden climate push draws fire from environmentalists, Washington Post
  • New research underscores benefits of gender-affirming hormones, rebutting anti-trans claims, STAT
  • 'You can't pour from an empty cup': U.S. violence prevention workers tell of burnout, The Guardian
  • How a six-in-one vaccine could soon help the world eradicate polio for good, STAT
  • Deadly fungal infections confound doctors — 'It's going to get worse,' Wall Street Journal

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