Bill Related To Accelerated Approvals Reportedly Tweaked To Please Drugmakers
Stat reports on how some provisions in a bill designed to make it easier to withdraw drugs previously approved under accelerated timelines were pulled after drugmaker opposition. Also in the news: Truepill, Eli Lilly, Madrigal Pharmaceuticals, sickle cell drugs, and more.
Stat:
Pharma Wins Changes In Bill Regarding FDA’s Accelerated Approval
A bill that makes it easier for the Food and Drug Administration to withdraw drugs that were approved under an accelerated timeline also dropped provisions the industry had opposed, according to three drug lobbyists. (Wilkerson, 12/19)
In other pharmaceutical news —
Modern Healthcare:
DEA: Truepill Wrongfully Filled Prescriptions For Stimulants
The Drug Enforcement Administration has alleged that online pharmacy Truepill wrongfully filled thousands of prescriptions for stimulants used to treat Attention Deficit/Hyperactivity Disorder. (Turner, 12/16)
Stat:
Eli Lilly Diabetes Drug Mounjaro In Short Supply, FDA Says
The U.S. Food and Drug Administration has declared a shortage of Eli Lilly’s Mounjaro, the latest diabetes drug to be in short supply. Due to increased demand for the medication, pharmacies may experience delays in receiving full shipments of the 5-milligram dose through January, according to a posting this week on the regulator’s website. (Chen, 12/17)
Stat:
Madrigal Experimental NASH Drug Delivers Strong Results
Madrigal Pharmaceuticals said Monday that its experimental treatment for fatty liver disease improved liver biopsies by two different measures in a study of 950 patients, reducing the fat deposits and fibrotic scarring that are hallmarks of the disorder, known medically as NASH. (Herper, 12/19)
The New York Times:
They Created A Drug For Susannah. What About Millions Of Other Patients?
Susannah was the first person to receive a drug designed to treat KIF1A-associated neurological disorder, or KAND, a progressive disease caused by genetic mutations that affect just 400 people in the world. In doing so, the young girl and her parents have found themselves on the edge of personalized medicine. ... But other rare-disease experts doubt that a funding model based on donations will ever be large or sustainable enough to help millions of patients. They are searching for other ways to accelerate the technology’s development, which includes seeking help from for-profit businesses. (Hayden, 12/19)
On sickle cell disease —
Stat:
Drugmakers Race To Find New Sickle Cell Drugs
Next year, two curative gene therapies could be approved for sickle cell disease. Yet drugmakers are pouring billions into developing new and more conventional drugs for the disease, even if they are likely to be far less transformative for individual patients. (Mast, 12/19)
Stat:
Why Are So Few Patients Taking The New Sickle Cell Drugs?
At first, Lena Harvey didn’t realize anything had changed. She just started doing strange, unexpected things: Cleaning her living room, for example, or playing with her puppy, a black schnauzer named Apollo. It hit her as she walked in from the patio one day. She looked at her husband, Anthony, and asked, “Honey, I haven’t been complaining about pain, have I?” (Mast, 12/19)
