FDA Panel Meeting Today To Review Proposal For Sickle Cell Gene Therapy
A FDA advisory panel will hear proposals Tuesday from Vertex Pharmaceuticals and CRISPR Therapeutics in an all-day meeting. USA Today examines the potential and high cost of using gene therapy to treat diseases.
Gene Therapy Might Cure Sickle Cell, But At A Steep Cost For Patients, Society
Several companies are pursuing gene therapies to treat sickle cell. A federal advisory panel will review the first of those proposals in an all-day meeting on Tuesday. Dr. Jeffrey Glassberg, who has had several patients go through gene therapy trials, said the results were impressive. "These people simply don't have sickle cell anymore," said Glassberg, a hematologist at the Icahn Sinai School of Medicine at Mount Sinai in New York. (Weintraub, 10/31)
The FDA Weighs The Risks Of Candy-Like Nonprescription Drugs
M&Ms, Reese’s, gummy bears, and even cotton candy made an appearance at the Food and Drug Administration on the day before Halloween. Commissioner Robert Califf was not throwing a costume party. Regulators were debating the potential benefits and pitfalls of “candy-like” nonprescription drug products such as gummies, particularly for children who often cannot, or do not want to, swallow actual pills. (Florko, 10/30)
Cassava Reveals Details — And Problems — With Alzheimer's Study
Cassava Sciences has long claimed its experimental drug, called simufilam, slows the cognitive decline of people with Alzheimer’s. On Friday, we learned how: The company recruited a large number of people into its clinical trial who don’t have Alzheimer’s. People who almost certainly had Alzheimer’s were also included in the study, but in this group, a placebo outperformed Cassava’s drug. (Feuerstein, 10/30)
Fact Check: Do 95% Of U.S. Over-The-Counter Drugs Come From China?
The claim: Republican presidential candidate Vivek Ramaswamy ... urged the U.S. to declare “pharmaceutical independence,” saying that “95% of over-the-counter pharmaceuticals are coming from our enemy in China.” PolitiFact ruling: False. The 95% figure is greatly exaggerated. Available data puts the share at no bigger than 20%, and possibly even lower. (Jacobson and Putterman, 10/30)
KFF Health News:
An Arm And A Leg: John Green Vs. Johnson & Johnson (Part 2)
The final episode of this two-part series about YouTube star John Green and his fight to make tuberculosis drugs more affordable takes listeners halfway around the world to India. For nearly two decades, activists there have been organizing for patent reform. Host Dan Weissmann and producer Emily Pisacreta speak with one of them, drug patent expert Tahir Amin, about how legal victories in India (and some extra pressure from John Green’s online community of fans) have set the stage for generic manufacturing and lower-priced TB drugs. (10/31)