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This week on The Readout LOUD podcast, host Adam Feuerstein announced his first order of business if he ever becomes FDA commissioner: “Outlawing” FDA drug approval announcements on Fridays. Despite this position, he bravely covered the historic approval of the first CRISPR-based medicine last week. Read all about it below.
A historic FDA approval for CRISPR-based medicine
The FDA on Friday approved the world’s first medicine based on CRISPR gene-editing technology, a groundbreaking treatment for sickle cell disease that delivers a potential cure for people born with the chronic and life-shortening blood disorder. The new medicine, called Casgevy, is made by Vertex Pharmaceuticals and CRISPR Therapeutics. Its authorization is a scientific triumph for the technology that can efficiently and precisely repair DNA mutations — ushering in a new era of genetic medicines for inherited diseases.
In a clinical trial, Casgevy was shown to eliminate recurrent episodes of debilitating pain caused by sickle cell, which afflicts approximately 100,000 people in the U.S., a vast majority of whom are Black. The same day, the FDA also approved another sickle cell treatment, a gene therapy from Bluebird Bio called Lyfgenia. Patients will now have the option of two cutting-edge therapies that provide potentially curative benefits.
Read more from STAT’s Adam Feuerstein about the approvals. And our amazing biotech team also had two Q&As on the news. Read on…
Inside the historic approval with CRISPR CEO and Vertex founder
First, Adam spoke with the CEO of CRISPR Therapeutics, Samarth “Sam” Kulkarni:
When writing about the approval of Casgevy, we focus mostly on the clinical data showing how the treatment benefits patients, but can you describe some of the other, less-recognized elements that got you to this point?
One of the things that really helped us was our interaction with the regulators on manufacturing issues. They were willing to provide feedback on what would be required, but you have to take what they say seriously, and then just do it.
And we aren’t done yet! Check out the conversation that STAT’s Allison DeAngelis had with Vertex’s founder, Joshua Boger, during a visit to his Boston-Seaport-area penthouse:
If you had gone back 29 years and told yourself where we’ve ended up, what would you have thought?
I would have lost a house in a bar bet if I had bet that Vertex would be the first company to bring a CRISPR drug to the world.
The quest to develop a new class of non-opioid painkillers
For decades, the biotech industry has tried — and failed — to develop new and effective painkillers without the addictive potential of opioids. There’s reason to think that could change soon.
Vertex Pharmaceuticals is on the cusp of announcing results from late-stage trials of a drug that precisely blocks pain-sensing neurons from signaling the brain. The company will announce data early next year from Phase 3 studies testing the molecule’s effectiveness at treating post-surgical pain. And before the end of the month, Vertex will share results from a mid-stage trial in people with diabetes who have chronic nerve pain.
If the Phase 3 trials are successful, Vertex plans to seek approval to treat moderate to severe acute pain — such as the kind experienced after surgery, a bad fall, or other specific events. Market analysts believe the therapy could become a future blockbuster if approved. But opinions surrounding its current experimental drug, a small molecule dubbed VX-548, are mixed. Vertex has argued that the treatment’s benefits are clinically meaningful, while some outside researchers and observers say those benefits seem modest based on data the company has shared so far. Read more from STAT’s Jonathan Wosen.
U.S. medical students aren’t being trained to treat a population of more than 7.4 million
Though there are 7.4 million Americans with intellectual and developmental disabilities, physicians are often uncomfortable treating them. In a recent survey of 714 Massachusetts physicians across various specialties, only 40% reported being very confident treating physically or intellectually disabled patients. It is no wonder that families struggle to find disability-friendly practices, and that people with intellectual and developmental disorders are four times more likely than neurotypical people to have unmet health care needs.
Harvard Medical School student Romila Santra knows firsthand what these barriers can look like. Her twin brother is among the 1 in nearly 10 U.S. children diagnosed with an intellectual and/or developmental disability. “His profound autism and nonverbal status mean my family lives with anxiety about taking him to the hospital or even about finding physicians willing to treat him,” Santra writes in a new First Opinion. Read more from Santra about what medical schools can do to help train doctors who are better equipped to serve all patients.
How preterm birth rates vary across the U.S.
As many of my fellow STAT reporters have written, there are often wide geographic disparities when it comes to health care access and certain health conditions. (If you haven’t seen it yet, check out the three part video series on rural health disparities from STAT’s Hyacinth Empinado.) Now, a new study from JAMA Network Open explores the difference in rates of preterm birth (before 37 weeks of pregnancy) by U.S. county between 2007-2019.
The study authors found that the rate of preterm birth varied widely between counties, with some in the Southeast region consistently seeing the highest rates of preterm birth compared to those in California and New England, which often had the lowest rates. At the national level, there was no significant change in the preterm birth rate over the study time period, but 15% of counties saw increases. The study authors found that social vulnerability by county — particularly regarding socioeconomic measures like income, employment, and education — was associated with the difference in birth rates.
What we’re reading
- All the carcinogens we cannot see, The New Yorker
- Tough road ahead for Bluebird Bio despite FDA approval for sickle cell therapy, STAT
- Why haven’t more older adults gotten the R.S.V. vaccine? New York Times
- Historic approval of sickle cell gene therapies met with cautious optimism by experts at ASH, STAT
- Arizona is the next abortion battleground, Wall Street Journal