First Gene-Silencing Drug Approved By FDA — And It Comes With An Eye-Popping Price Tag
Slicing genes with drugs is the latest in a wave of hot new treatments geared toward fighting diseases in unique ways. But the price on innovation is steep. In other news, the FDA plans to streamline drug safety evaluations and a super PAC goes after Rep. Anna Eshoo (D-Calif.) over the money she's taken from the pharmaceutical industry.
The Wall Street Journal:
New Kind Of Drug, Silencing Genes, Gets FDA Approval
The U.S. Food and Drug Administration on Friday approved the first drug that combats disease by silencing the genes driving it, the newest technology transforming the arsenal of medicines. Onpattro, from Alnylam Pharmaceuticals Inc., is the first treatment approved to treat nerve damage caused by a genetic disorder that also causes heart and digestive disease and can be fatal. It is based on Nobel Prize-winning research that drugmakers have had a tough time translating into effective medicines. (Loftus, 8/10)
Stat:
Amicus' Rare Disease Drug, Once Indefinitely Delayed, Wins FDA Approval
After 12 years, millions of dollars, and a major reversal of fortune, Amicus Therapeutics won Food and Drug Administration approval on Friday for its first therapy, a treatment for a rare and sometimes deadly disease. The drug, called Galafold, targets Fabry disease, an inherited disorder in which the lack of a key enzyme leads to buildups up fats that can cause fatal organ damage. As it stands, Fabry patients rely on costly, bimonthly injections of synthetic enzymes to keep symptoms at bay. Amicus’s drug is an every-other-day pill that works not by replacing the missing enzyme but by boosting the effects of what patients already produce. (Garde, 8/10)
Stat:
Gottlieb: FDA Will Streamline Drug Safety Evaluations
The Food and Drug Administration will soon standardize the way it handles data on the safety and effectiveness of drugs in an effort to reduce inconsistencies in the drug review process, agency Commissioner Scott Gottlieb said Friday. “Rather than just looking at drug safety parameters in terms of the tables that are submitted to us, we’re going to actually take the raw data and evaluate it into custom tables, that the agency’s going to develop, that are going to be standardized across all our review divisions,” Gottlieb said. (Swetlitz, 8/10)
Stat:
New Ads Attack Rep. Anna Eshoo For Being 'In The Pocket Of Big Pharma'
A group that’s buying ads to try to elevate the issue of high drug prices in the midterm elections has identified its latest target: a Democratic congresswoman from Silicon Valley who wants to lead on health care issues. Patients For Affordable Drugs Action will spend $500,000 on ads attacking Rep. Anna Eshoo for her legislative record and for accepting campaign contributions from the drug industry, the group announced Friday. Eshoo is expected to be re-elected easily in November. (Robbins, 8/10)
The Hill:
Drug Pricing Watchdog Group Targets California Dem In $500K Ad Buy
“Anna Eshoo’s record on drug prices is terrible, and the reason why is obvious,” said David Mitchell, founder of Patients For Affordable Drugs Action. “She’s taken enormous sums of money from drug corporations, and she does their bidding in Washington." (Hellmann, 8/10)
