Key facts
- The FDA approved Merck's Lipfendra, a new pill for reducing high cholesterol.
- Lipfendra is the first non-injectable drug targeting the PCSK9 liver protein.
- The medication is for high-risk patients whose cholesterol remains high despite statin use.
- Studies showed Lipfendra reduced LDL cholesterol by over 55% in six months.
- Side effects were similar to placebo, including dizziness and diarrhea.
The Food and Drug Administration has approved Merck's Lipfendra, a novel pill designed to significantly lower cholesterol levels in high-risk patients. This marks the first non-injectable medication that targets the PCSK9 liver protein, a mechanism previously only available through more expensive injectable drugs from companies like Amgen.
Lipfendra is indicated for patients whose artery-clogging cholesterol persists despite treatment with statins, the standard medication for reducing heart attack risk. Even at high doses, statins are insufficient for many individuals to reach recommended LDL cholesterol levels.
Merck's approval was based on two studies involving over 3,000 high-risk patients who added Lipfendra to their existing treatment regimens. These studies demonstrated that Lipfendra reduced LDL cholesterol by more than 55% after six months, with a similar reduction observed in a second study. The benefits were sustained for over a year, and side effects, such as dizziness and diarrhea, were comparable to those experienced by patients taking a placebo. A key administration requirement is taking the pill on an empty stomach.
The FDA's review utilized a program designed for expedited approval of promising medications serving the public interest, a pathway established by former FDA chief Dr. Marty Makary.