Drugs & FDA
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A Pill Doubled Survival in Advanced Pancreatic Cancer: Six Months to Thirteen, at $39,800 a Month
The best result advanced pancreatic cancer has seen in a generation, and the FDA cleared it in about five weeks. For patients who have run out of chemo, it turns a median of six months into thirteen, at a list price of $39,800 a month.
Drugs & FDAJ&J Won the First-Ever Drug Approval for Warm Autoimmune Hemolytic Anemia. It Helped About One in Four Patients.
It works by starving rogue antibodies of the recycling route that keeps them alive. Whether it beats the drugs doctors already reach for is the question the first-ever headlines skip.
Drugs & FDAThe FDA Is Building an On-Ramp for Medical AI That Can Hallucinate
The FDA has cleared more than 1,000 AI medical devices and not one large language model. Its new discussion paper starts building the pathway for the ones that can make things up, with faster delivery to market named as an agency priority.
Drugs & FDAA $2.7 Million Gene Therapy, Approved Because Patients Needed Less Cornstarch
Ultragenyx's Genglycos won accelerated approval on a surrogate: a 31 percent cut in daily cornstarch. But the disease is low blood sugar, and by the company's own numbers the treated group spent slightly more time below 70 mg/dL, while liver enzymes spiked in 71 percent and every patient needed ster
Drugs & FDATakeda Fixed the Narcolepsy Drug That Was Poisoning Livers. Now You Can't Buy It.
Oveporexton is the first drug to replace the orexin signal narcolepsy destroys, instead of stimulating around a tired brain. At the higher dose it leaves 60 percent of patients newly insomniac, and it still has to clear DEA scheduling before it reaches a pharmacy shelf.
Drugs & FDAThe Screwworm America Eradicated With Sterile Flies Is Back, and This Time Washington Also Reached for an Emergency-Authorized Chemical
Dicyclanil, an Australian sheep-blowfly chemical with mouse liver tumors in its toxicology file, is now cleared for cattle, swine, goats and wildlife under section 564, the same emergency-use power heavily used for pandemic countermeasures.
Drugs & FDAThe FDA's Reviewers Called This Melanoma Data 'Not Interpretable.' Nine Days Later, the Agency Approved the Drug.
Twice the FDA rejected Replimune's engineered-herpes melanoma therapy, its reviewers calling the single-arm trial not interpretable. The design never changed. A 10-3 advisory vote came anyway, and nine days after that memo, the accelerated approval.
Drugs & FDANIH Said Its Cervical-Cancer AI Beat Human Doctors. Six Years Later, Its Own Author Called It Overfitting.
In 2019, NIH announced an algorithm that read cervix photos better than doctors, an AUC of 0.91. Its senior author has since written that the number came from overfitting and that the follow-on projects failed. Nature Medicine is now calling the same idea the 'missing piece.'
Drugs & FDAThe FDA Approved Moderna's mRNA Flu Shot for Seniors Without Proving It Works in Them
In February the FDA's own vaccine chief refused to even review it. One week later the agency reversed course, and the trial data never changed.
Drugs & FDAThe FDA Said No to Moderna's mRNA Flu Shot in February. By August, the Two Men Who Said No Were Gone.
The FDA refused to even review Moderna's mRNA flu shot in February because it was never tested against the enhanced vaccines seniors are steered toward. The two officials who objected are gone, and the agency approved it anyway, for the oldest patients, on a promise to prove it works later.
Drugs & FDAThe FDA Approved Narcolepsy's First Orexin Agonist. Takeda's Last One Was Pulled for Liver Injury.
Takeda's oveporexton switches on the orexin receptor that narcolepsy type 1 patients can no longer signal. The company's last orexin drug was halted in 2021 after patients crossed serious liver-injury thresholds.
Drugs & FDAFor Years the Muscle Aches Were Called a Nocebo Effect. A McMaster Lab Found Statins Injuring the Muscle Cell Itself.
An estimated 7 to 29 percent of statin users report muscle pain. The flagship trial called most of it a placebo response. In mouse and human muscle cells, a McMaster team has now traced a cholesterol-independent pathway by which statins damage muscle directly.
Drugs & FDAThe FDA Twice Called This Melanoma Trial Uninterpretable. Then It Approved the Drug, at $450,000 a Course.
Federal reviewers rejected Replimune's melanoma therapy twice, faulting a single-arm trial they called not adequate and well-controlled and its results uninterpretable. No new controlled data ever answered them. After a 10-3 advisory vote, the FDA approved it anyway, at $450,000 a course.
Drugs & FDAGilead Priced Its Near-Perfect HIV Shot Out of the Countries Where the Virus Is Spreading Fastest
Lenacapavir drove new HIV infections to nearly zero across two Gilead trials and a year-long extension. Gilead lists it at $28,218 a year in America; researchers estimate generics could be made for as little as $25, and the fastest-growing epidemics were left off the cheap-generic map.
Drugs & FDAU.S. Special Operators Carried French Freeze-Dried Plasma for 15 Years Before the FDA Licensed an American One
Allied militaries and U.S. commandos have relied on freeze-dried plasma for years. Washington licensed its own version this week, decades after an American doctor first invented the powder.
Drugs & FDAThe FDA Cleared a CRISPR Cure for Two-Year-Olds Whose Own Trial Enrolled No One That Young
Casgevy's pediatric trials enrolled 11 children aged five and up, yet the FDA extended the $2.2 million therapy down to age two, where the conditioning chemo can permanently sterilize a child too young to bank eggs or sperm.
Drugs & FDARice Chemists Taught an Enzyme to Recycle Drug Synthesis's Most Expensive Ingredient
The chemistry that builds drugs like Spinraza just got cheaper. The $125,000 price tag never had much to do with the chemistry.
Drugs & FDAThe FDA Approved the First Cholesterol Pill of Its Kind Before Its Trial Could Show It Prevents Heart Attacks or Deaths
Lipfendra lowers LDL cholesterol by up to 60 percent. The 14,550-patient trial that would show whether that prevents heart attacks or deaths does not finish until November 2029, and Merck is charging roughly $3,800 a year in the meantime.
Drugs & FDAThe Blue Light Over Your Fish Tank Is Now Building Drug Molecules
University at Buffalo chemists swapped harsh UV for a soft blue LED and got something the old chemistry rarely gives: two modifications to a molecule from a single reaction step.
Drugs & FDAFor Years, a Foreign Factory Could Put the Active Ingredient in Your Medicine and Never Tell the FDA
The FDA led with a paperwork convenience for manufacturers. Buried underneath was the admission that it can't always trace where a drug's active ingredient was made, a blind spot Congress had to order it to close.
Drugs & FDAA $440,000 Drug Proved This Obesity Was Never About Willpower
Brain-tumor surgery can wreck the hypothalamus and leave hunger permanently switched on. Setmelanotide restored the missing signal in a real placebo-controlled trial, then arrived priced near half a million dollars a year.
Drugs & FDAThe Last Time Washington Went After Hospital Drug Markups, the Supreme Court Blocked It. This Time CMS Brought Receipts.
In 2018 the Supreme Court blocked CMS from cutting the markup 340B hospitals charge Medicare for discounted drugs, because the agency skipped a required survey. CMS ran the survey this time, and found seniors paying co-pays bigger than what the hospital paid for the drug.
Drugs & FDAThe FDA Cleared a Sickle-Cell Cure for Two-Year-Olds in 53 Days. It Starts With Chemo That Can Sterilize Them.
Casgevy is a real advance against a brutal disease. It is also a $2.2 million therapy that requires sterilizing chemotherapy, saw a child die in trials, and reached toddlers on a 53-day review Makary's FDA hands out as a favor.
Drugs & FDAThe FDA Office That Vets Gene Therapy Has Run Through Its Entire Top Tier in a Year
The office that decides whether gene therapies reach patients has lost its director, her deputy, and the man brought in to replace them, all inside twelve months.