Drugs & FDA
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The Data on the First Sanfilippo Gene Therapy Were Ready Last Summer. The FDA Spent the Next Year on the Factory.
The FDA said Ultragenyx's clinical data were robust in July 2025, then rejected its Sanfilippo gene therapy over manufacturing questions. Approval came fourteen months later, for children who lose skills and die on a clock.
Drugs & FDATwenty Years, Four Botanical Drugs: The FDA Wants to Know What's Blocking the Rest
Four botanical drugs in two decades, under a framework built for single molecules. Now, inside RFK Jr.'s HHS, the agency that wrote those rules is asking what blocked the rest, and whether it means it.
Drugs & FDAThe FDA's New Pilot Lets a Drug Sponsor Choose the Institution Helping Build Its First-in-Human Application, and Reviews the Pieces as They Arrive
The agency will select 8 to 10 sponsor-QRI pairs to co-develop first-in-human trial applications it reviews on a rolling basis, and its stated reason is that China and Australia get there faster.
Drugs & FDAA Fatal Brain Disease in Children Finally Has a Drug, and It Lists at $1.14 Million a Year
Ionis's zilganersen is the first therapy ever approved for Alexander disease, and it lists near $1.14 million a year. The same FDA approval handed Ionis a rare pediatric disease voucher, the transferable coupon that has resold for as much as $350 million.
Drugs & FDAThe FDA Approved a Breast Cancer Drug Its Own Advisers Had Just Rejected, 6 to 3
An FDA advisory panel voted 6 to 3 that AstraZeneca had not shown its breast cancer drug delivered a meaningful benefit. The agency approved it four months later anyway.
Drugs & FDASickle Cell Finally Has a Cure. It Costs $2.2 Million and Has Reached 64 People.
Casgevy has kept Victoria Gray crisis-free for seven years. Getting it still means wiping out a patient's bone marrow with a chemo tied to infertility and malignancy warnings and paying about $3 million all in, and two years after approval only 64 people have been infused.
Drugs & FDAThe 'Bone-Building Switch' Is Back in the Headlines. It Was Tested in Mice, and Only Mice
A publicly funded Leipzig compound rebuilt bone in ovariectomized mice, 8 per treated group. More than a year later the same experiment is still being sold to readers as a way to keep their bones strong for life.
Drugs & FDATwo Factories Make the Starter IV Nutrition America's Preemies Live On. Both Are Closing, and the Backup Plan Has Killed Babies Before.
The predominant source of starter IV nutrition for premature newborns is two outsourcing facilities, and both are permanently closing. The FDA's fix: let hospital pharmacies mix it themselves for 180 days, sending the most unforgiving product in medicine back into the loosely regulated compounding t
Drugs & FDAThe FDA Has Approved 4 Plant Drugs Since 2004. Now It's Asking the Public Why So Few Made It.
A request for public input is not reform. After two decades of a pathway that produced four approvals, the question is whether the agency wants plant medicine or just wants to talk about it.
Drugs & FDAIonis Priced the First Alexander Disease Drug at $1.14 Million a Year, and Got a Federal Voucher It Can Resell for More Than $100 Million
Zilganersen is the first drug for Alexander disease, an antisense therapy designed to lower the toxic protein that drives a fatal brain disease. It also carries a $1.14 million annual price and a pediatric voucher Ionis can resell for more than $100 million.
Drugs & FDAThe FDA Approved a Breast Cancer Drug Four Months After Its Advisers Voted No
On April 30 the FDA's oncology advisers voted 6 to 3 that AstraZeneca's camizestrant hadn't shown a clinically meaningful benefit. On September 4 the agency approved it anyway, conceding in its own approval that the benefit is "not yet confirmed."
Drugs & FDAChina Runs More Drug Trials Than America. The FDA Says It Can No Longer Trust Data From Sites It Can't Inspect.
China went from under 8 percent of the world's drug trials in 2010 to passing the United States by 2020. After three deaths in Chinese studies surfaced late, the FDA says it has sometimes been denied access to the very sites whose data underpins American approvals.
Drugs & FDAThe Pancreatic-Cancer Pill That Doubles Survival: $39,800 a Month, and a Fast Lane Congress Never Approved
Rasonque roughly doubles median survival in metastatic pancreatic cancer. Revolution Medicines priced it at $477,600 a year, more than double Keytruda, and the FDA cleared it early through a voucher program lawmakers say Congress never authorized.
Drugs & FDAA 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.