March 1, 2024
Barry Bunin, PhD
Founder & CEO
Collaborative Drug Discovery
FDA Approved 55 New Molecular Entities in 2023. The FDA approved a remarkable 55 new molecular entities in 2023, up from 37 in 2022, according to a recent article in C&EN. The article notes that the 31 small-molecule therapies approved last year accounted for 56% of the new drugs, up from 46% in 2022. For a detailed look at the newly approved vs historical drugs see The Pharmaceutical Knowledgebase (www.PharmaKB.com) covering FDA and EMA approved drugs, competitive target and disease landscapes, clinical pipelines, post-approval adverse event signals with financial and scientific publication keyword trends for data-driven investment hypotheses.
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Abortion Pills Delivered via Telehealth and the Mail Confirmed to Be Safe—Ahead of Supreme Court March Hearing. A newly released study published in Nature Medicine found abortion pills prescribed via telehealth to be safe and effective, bolstering an earlier FDA finding of the same. The authors noting the need for telehealth delivery wrote: “In 2021, the US Food and Drug Administration (FDA) removed the in-person dispensing requirement on mifepristone, the first drug used in a medication abortion. This ruling allowed clinicians to begin offering a ‘no-test’ telehealth model of medication abortion care. Clinicians could now offer entirely remote consultations, using the patient’s self-reported medical history instead of ultrasonography or other tests to screen for medical eligibility. Moving abortion out of the clinic reduced travel cost and stigma-related barriers and increased convenience for patients.” The study's lead author, Ushma Upadhyay of the University of California – San Francisco, told NPR “These results shouldn't be surprising. That's consistent with the over 100 studies on mifepristone that have affirmed the safety and effectiveness of this medication." The Supreme Court hears arguments in the case on March 26. Upadhyay said: "I don't know if they can enter new evidence into the case at this point. But I do hope it impacts the perception of how safe this medication is."
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“Drug Shortages Trigger FTC Probe.” That’s the headline from a recent Wall Street Journal article on whether hospital purchasing groups and drug distributors have played a role in low supplies of chemotherapies, other drugs. The article says the FTC is exploring whether the companies that broker drug purchases for hospitals, along with the middlemen that ship the medicines, have misused their market power to push down prices of generic drugs so much that some manufacturers can’t profit and have stopped production, causing shortages. “For years Americans have faced acute shortages of critical drugs, from chemotherapy to antibiotics, endangering patients,” said FTC Chair Lina Khan. “Our inquiry requests information on the factors driving these shortages and scrutinizes the practices of opaque drug middlemen.”
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“Ancient Skeletons Give Clues to Modern Medical Mysteries.” The New York Times carries this headline about how DNA fragments from thousands of years ago are providing insights into multiple sclerosis, diabetes, schizophrenia and other illnesses. The article says a new study found clues in the skeletal remains of a lost tribe of nomads who herded cattle across the steppes of western Asia 5,000 years ago. It turns out that the nomads carried genetic mutations that most likely protected them from pathogens carried by their animals, but that also made their immune systems more sensitive. These genes, the study suggests, made the nomads’ descendants prone to a runaway immune response. The finding is part of a larger, unprecedented effort to understand how the evolutionary past has shaped the health of living people. Researchers are analyzing thousands of genomes of people who lived between Portugal and Siberia and between Norway and Iran roughly 3,000 to 11,000 years ago. By pulling DNA from ancient human bones, they hope to trace the genetic roots of not only multiple sclerosis, but also diabetes, schizophrenia and many other modern illnesses. Eske Willerslev, a geneticist at the University of Copenhagen who led the effort, said: “We are taking ancient human genomics to a whole new level.”
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Obelisks and RNA: “Heading into the Unknown.” Derek Lowe, in his recent blog in Science, describes a new preprint about “small RNA species, hitherto unknown, that seem to be colonizing bacteria in the human oral mucosa and gut.” He writes that these things are a bit like viroids, which are circular RNA species that are pathogens in plant species, but they have no sequence relationships with any known viroid. These new things, deemed “obelisks” for their predicted rod-like shape, are not just variants of the plant viroids. “The discovery of the obelisks makes it even more clear that there’s a lot of RNA action going on out there that we haven’t been noticing. They seem to be all over the place—searching through public sequencing databases, the authors have already identified what look like about thirty thousand different ones, all of which seem to be replicating in various bacterial species.” He says the discovery may point toward the RNA World hypothesis that the biology of life developed first around RNA, with DNA being a later development that took over. Lowe concludes: “Alternatively, these various transmissible RNA species might have arisen later on their own, or of course the whole RNA world idea could be wrong from the start. But whatever direction you take on these questions, you’re heading into the unknown - that’s for sure.” And the whole point.
Barry A. Bunin, PhD, is the Founder & CEO of Collaborative Drug Discovery, which provides a modern approach to drug discovery research informatics trusted globally by thousands of leading researchers. The CDD Vault is a hosted biological and chemical database that securely manages your private and external data.