FDA Approves First Gene Therapy For Duchenne Muscular Dystrophy
In what NPR calls an "eagerly anticipated decision," the FDA approved Thursday a treatment called Elevidys from Sarepta Therapeutics to treat Duchenne muscular dystrophy by targeting the genetic defect behind it. The treatment costs $3.2 million. Also in the news: the high cost of drugs.
NPR:
In A First, FDA Approves Sarepta Gene Therapy For Duchenne Muscular Dystrophy
In an eagerly anticipated decision, the Food and Drug Administration Thursday approved the first gene therapy for muscular dystrophy. "Today's approval addresses an urgent unmet medical need and is an important advancement in the treatment of Duchenne muscular dystrophy, a devastating condition with limited treatment options, that leads to a progressive deterioration of an individual's health over time," said Dr. Peter Marks, director of the FDA's Center for Biologics Evaluation and Research, in a statement. (Stein, 6/22)
The Wall Street Journal:
The Price Of First Gene Therapy For Muscular Dystrophy: $3.2 Million
Young children with Duchenne muscular dystrophy can now get the first gene therapy for the crippling muscle condition. The one-time treatment will cost $3.2 million. The drug, known as Elevidys and made by Sarepta Therapeutics, becomes the first treatment that seeks to treat the disease by repairing the genetic defect at its root. The Food and Drug Administration approved the therapy on Thursday, following a fast-track review. (Whyte, 6/22)
On the high cost of drugs —
Stat:
Medicare Drops New Coverage Details For Alzheimer’s Drug Leqembi
The health care system is on the precipice of broad access to a treatment for Alzheimer’s for the first time — and it’s scrambling to figure out how to handle it. Right now, most people with mild cognitive impairment who would qualify for Eisai and Biogen’s drug Leqembi are in the Medicare program, which has restricted which patients can receive the medication to those who are enrolled in clinical trials. But the floodgates could open if the Food and Drug Administration grants the drug full, traditional approval in the coming weeks. (Cohrs, 6/22)
Stat:
The Strategy Behind Pharma's Lawsuits Over Drug Pricing Reform
The pharmaceutical industry has been filing a lawsuit here, there, and pretty much everywhere. Drugmakers lost a two-decade long lobbying fight in Congress last summer when Democrats gave Medicare more power to choose what it pays for prescription drugs. Now, they’re taking their battle to the courts. (Cohrs, 6/22)
Philadelphia Inquirer:
Mark Cuban Cost Plus Drug Co. Lowers Price Of Lifesaving Drugs For Wilson Disease
For decades, the drug that keeps Drew Katz alive sold for less than $1 a pill. Then its manufacturer started to raise the list price — slowly at first, then sharply, increasing it in 2015 to more than $200 a dose. Even when generic versions came along to treat the condition, called Wilson disease, the price stayed high. (Avril, 6/23)
In other pharmaceutical news —
Stat:
FDA Rejects Intercept’s NASH Drug, Prompting A Pivot
The Food and Drug Administration rejected Intercept Pharmaceuticals’ investigational treatment for NASH on Thursday, derailing what would have been the first approved medicine for a prevalent liver disease. (Garde, 6/22)
Stat:
U.S. Seeks A New Trial Over Royalties On Gilead HIV Prevention Pills
The U.S. government asked a federal judge to overturn a recent case in which a jury decided that Gilead Sciences did not infringe on patents held by the Centers for Disease Control and Prevention for a pair of groundbreaking HIV pills. (Silverman, 6/22)
