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The FDA Just Greenlit a Cancer Drug Class That Didn't Exist 24 Hours Ago
Bristol Myers Squibb's iberdomide (brand name Zenbexus) just became the first-ever CELMoD therapy to win FDA approval, creating an entirely new drug class for multiple myeloma. CELMoDs are essentially the next-generation upgrade to IMiDs like lenalidomide: they grip the same molecular target (cereblon) but bind tighter and destroy cancer-sustaining proteins faster. The accelerated approval covers patients who've tried at least one prior line of therapy, and BMS still needs to deliver confirmatory data from its EXCALIBER-RRMM trial to convert to full approval. With two more CELMoDs in its pipeline, BMS is betting this platform can fill the revenue gap as its older blockbusters lose exclusivity.
Why it matters: This approval validates targeted protein degradation as a real therapeutic strategy, not just a conference-poster concept, and gives BMS a tangible answer to the patent-cliff question Wall Street keeps asking.
Read more →Gene Therapy: Highs and Lows
Sarepta's Flagship Gene Therapy Gets the FDA's Scarlet Letter
Sarepta just added a black box warning (the most severe in medicine) to Elevidys, its Duchenne muscular dystrophy gene therapy, after fatal liver failures in patients. The company simultaneously cut 500 jobs and paused most limb-girdle muscular dystrophy programs. Elevidys revenue cratered from $375 million in Q1 2025 to $110 million by Q4, and the non-ambulatory patient indication was stripped entirely from the label.
Read more →PTC Scoops a Near-Approval Gene Therapy Out of Bankruptcy Court
PTC Therapeutics won a bidding war for Sangamo's Fabry disease gene therapy, paying $111 million cash (plus $100 million in approval milestones) at a bankruptcy auction. The therapy already has a rolling FDA submission underway and could launch by 2027. The Fabry treatment market is worth approximately $2.5 billion, and no competitor is as close to the finish line.
Read more →Clinical and Regulatory
The Lung Cancer Drug That Keeps Cracking an Impossible Mutation
Zipalertinib scored its second Phase 3 win, this time beating chemotherapy alone as a first-line treatment for EGFR exon 20 insertion lung cancer, a mutation most drugs can't touch. An FDA decision on its post-chemo indication is set for February 2027, and the companies plan to pursue the first-line approval too. If both come through, it would be the first oral option spanning multiple treatment lines for this mutation.
Read more →The Lyme Vaccine That Fear Killed 24 Years Ago Just Cleared a Major Hurdle
Europe's medicines agency validated the application for Pfizer and Valneva's Lyme disease vaccine, making it the first candidate to reach this stage since LYMErix was pulled in 2002 (killed by lawsuits, not bad science). Phase 3 data showed roughly 74% efficacy with a clean safety profile. A European decision could come by late 2027; a U.S. filing timeline remains unclear.
Read more →Alzheimer's Has a Seeing Problem. This Brain Scan Could Fix It.
Lantheus' MK-6240 tau PET imaging agent hit its FDA decision date after both pivotal trials met their primary goals. With new Alzheimer's drugs requiring precise patient selection, a better tau scan could determine who actually benefits from treatment. The tracer is already used in nearly 100 clinical trials, giving it a research footprint that could accelerate commercial adoption.
Read more →Business and Policy
Lilly's $449 Gambit to Kill the Zepbound Black Market
Eli Lilly is offering Zepbound's highest doses in single-dose vials at $449 per month through LillyDirect, a discount of about 59% off the standard pen price. The move targets millions of cash-paying patients tempted by a booming (and dangerous) compounded GLP-1 market that's racked up over 1,700 adverse event reports with the FDA. Meanwhile, Lilly filed six new lawsuits against sellers of its unapproved pipeline drug retatrutide.
Read more →The UK Bets £250 Million It Can Compete in Life Sciences Again
The UK government committed £250 million toward a new National Biosecurity Centre in Harlow, Essex, part of a broader £2 billion life sciences push. The catch: the facility won't be fully operational until 2038. With life sciences foreign investment down 58% since 2021 and a projected talent shortfall of 133,000 workers by 2030, the real question is whether the money moves fast enough.
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