And so, another working week will soon draw to a close. Not a moment too soon, yes? This is, you may recall, our treasured signal to daydream about weekend plans. Our agenda is, so far, rather ambitious. We hope to escort Mrs. Pharmalot to a theatrical undertaking, consort with people from years gone by and, yes, hold still another listening party, where the rotation will likely include this, this, this, this and this. And what about you? This may be an opportunity to catch up on your reading or visit an unusual eatery. You could reach out to someone special. Or perhaps tune out the growing tumult by binge-watching something on the telly. Well, whatever you do, have a grand time. But be safe. Enjoy, and see you soon. …
Vertex Pharmaceuticals won approval from the U.S. Food and Drug Administration for a non-opioid pain drug, clearing the way for the rollout of a product that has simultaneously sparked high hopes and mixed opinions among physicians and market analysts, STAT explains. The drug, Journavx, was approved for the twice-daily treatment of moderate to severe acute pain, such as the sort of short-lived but sharp aches that might follow a bike accident or an operation. The FDA decision was based on a pair of late-stage trials that found that patients undergoing tummy tuck or bunion surgery had significantly less pain after taking the drug than those who took a placebo. The trials also found the medicine was generally safe, with fewer adverse events among those on Journavx than in the placebo group. Journavx was priced at $31 a day. Market analysts have forecasted the drug could bring in $1.5 billion in annual revenue by 2030. That does not include sales for chronic pain, where there is a larger market opportunity and where Vertex is also running trials.
Meanwhile, people in England with sickle cell disease will now be able to access the world’s first CRISPR-based medicine under a pricing and reimbursement agreement reached between U.K. health officials and Vertex Pharmaceuticals, STAT notes. The deal is notable because the U.K.’s cost-effectiveness watchdog initially raised concerns about the durability of the treatment, called Casgevy, in a preliminary ruling last year, saying it was not yet recommending the therapy. But the National Institute for Health and Care Excellence now called the one-time treatment a potential cure, and said that Casgevy would be available through the National Health Service to patients 12 years and older under a managed access scheme. This will allow officials to track how long the benefits of the therapy last and whether patients who receive it have any ongoing sickle cell complications that require additional care. The U.K. list price of Casgevy is 1.65 million pounds, or nearly $2.1 million, but the deal makes it available to the NHS at an undisclosed discount. The U.S. list price is $2.2 million.
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