Good morning, everyone, and how are you today? We are doing just fine, thank you, despite cloudy skies looming over the Pharmalot campus. After all, the birds are still chirping and crisp autumn breezes are wafting by. Moreover, this is an exciting day on this side of the pond, where millions of voters are casting their ballots in hopes of changing their world. We can only imagine what lies ahead. So to cope, we are doing what we do best — firing up the coffee kettle for another cup of stimulation. Our choice today is Jack Daniels, a necessary — and genuine — choice. Please feel free to join us. Meanwhile, here are a few items of interest that may provide not only useful information, but a welcome distraction. Hope your day goes well. …
A patient with sickle cell disease died while participating in a clinical trial of a CRISPR-based treatment from Beam Therapeutics, threatening to overshadow early signals of effective gene editing, STAT reports. Beam said the patient succumbed to respiratory failure, deemed to be “likely caused” by a regimen of chemotherapy required to prepare the patient for BEAM-101, a treatment that uses the company’s new, more precise form of CRISPR gene editing called base editing. The patient who died was one of six participants with sickle cell disease treated to date in Beam’s first clinical trial. The death occurred four months after the patient received BEAM-101 and the U.S. Food and Drug Administration, which was briefed on the fatality, has allowed Beam to continue the study without any changes. The fatality underscores the risks of one-time, genetic treatments for blood disorders like sickle cell and beta thalassemia. The gene-editing is 21st century, but it remains shackled to a highly toxic chemotherapy, called busulfan, approved in the 1950s.
Obesity drug coverage in Medicaid remains limited, with 13 state Medicaid programs covering GLP-1s for obesity treatment as of August 2024, according to a survey by KFF. Overall, a dozen states reported covering GLP-1s for weight loss under their Medicaid fee-for-service programs as of July 1, and North Carolina added coverage in August. All 12 states that reported coverage as of July 1 also reported that utilization controls were applied, mostly prior authorization or body mass index requirements. Eleven of the 12 states reported covering all three GLP-1s currently approved for treating obesity — Saxenda, Wegovy, and Zepbound. Meanwhile, the number of Medicaid prescriptions and gross spending on GLP-1s increased rapidly in recent years, with both nearly doubling from 2022 to 2023. Overall, from 2019 to 2023, the number of GLP-1 prescriptions increased by more than 400%, while gross spending increased by over 500%. Spending per prescription before rebates reached more than $900 per prescription in 2023.
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