FDA Approves First Oral PCSK9 Inhibitor to Lower LDL Cholesterol
FDA approves new kind of cholesterol pill

The FDA has approved Lipfendra, the first oral PCSK9 inhibitor, offering a once-daily tablet alternative to injectable therapies for adults with high cholesterol. This breakthrough provides a new option for patients needing further LDL-C reduction despite existing treatments, marking a significant step forward in managing cardiovascular disease risks.
Cardiovascular disease remains the leading cause of death in the United States, and elevated LDL cholesterol is one of its most important modifiable risk factors.
- marcosfelt
There's a nice writeup by Derek Lowe on the synthesis of this molecule: https://www.science.org/content/blog-post/futuristic-synthes...
It's quite a beast!
- hankbond
What is the current state of understanding on high cholesterol as a leading or trailing indicator of poor health? I vaguely remember reading one theory that cholesterol was used by the body to mitigate arterial damage from other causes?
- JumpCrisscross
“Proprotein convertases (PPCs) are a family of proteins that activate other proteins. Many proteins are inactive when they are first synthesized, because they contain chains of amino acids that block their activity. Proprotein convertases remove those chains and activate the protein” [1].
- sneak
It works via the same mechanism as an incredible drug called Repatha (generic name evolocumab), but Repatha is quite expensive, requires refrigeration, and must be injected subcutaneously every two weeks. The news here is that this is administered orally.
- derektank
Not really a new kind of pharmaceutical, pcsk9 inhibitors have been on the market for a while now. What’s new is that it’s an oral medication rather than an injectable