fda-approval
7 stories tagged "fda-approval"
The FDA's 'Historic' Growing Heart Valve Has Been Stretched in Two Children
Children who get a new heart valve before age 2 can face up to five open-heart surgeries. Edwards' polymer valve is built to end that, and the trial behind its approval widened it in two of 62 kids.
Drugs & FDAOne Hormone Starves the Brain and Floods the Heart: The FDA's First MCT8 Drug Treats Only the Flood
Emcitate is being called the first treatment for MCT8 deficiency, but its FDA label covers the racing heart and the wasting body, not the brain disability that defines the disease.
Drugs & FDAThe FDA Approved a $3.95 Million Gene Therapy It Rejected Fourteen Months Ago
In July 2025 the FDA refused to approve this Sanfilippo gene therapy and faulted the factory, not the science. Fourteen months later it is the first treatment for a fatal childhood brain disease, cleared on 17 treated children against a comparison group the maker built, at $3.95 million a dose.
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 & 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 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 & 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.