Amgen's Repatha Becomes Second PCSK9 Approved by FDA

Highly effective cholesterol-lowering drug to challenge Regeneron & Sanofi’s Praluent.

The second in a new class of highly effective cholesterol lowering drugs received the green light from the FDA today.

Amgen’s evolocumab (Repatha) was approved by the FDA as a new entry to the proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitor drug class, following alirocumab (Praluent), which was approved on July 24, 2015.

The FDA’s approval was based on a 52-week placebo-controlled trial and 8 12-week placebo-controlled trials with patients with primary hyperlipidemia. In 1 of the 12-week studies that involved 329 patients with HeFH, those who were randomly selected to receive Repatha for 12 weeks had an average reduction of LDL cholesterol of around 60% compared with those taking placebo, according to the FDA.

Adverse effects from Repatha may include nasopharyngitis, allergic reactions, upper respiratory tract infection, flu, back pain, and reactions such as redness, pain, or bruising where the injection is given. According to the FDA, Repatha is currently being studied to see if its addition to statin therapy can help reduce cardiovascular risk.

Praluent was announced to have a cost of $14,600 annually. With such a price point, if the drugs were to be broadly approved for the approximately 2.3 million people who could potentially use PCSK9 inhibitors, it could cost up to approximately $23.3 billion annually, according to pharmacy benefit manager Prime Therapeutics.

Unlike current statins that are taken orally and have less costly generic alternatives, PCSK9 inhibitors are self-injected monoclonal antibodies that block the PCSK9 protein from inhibiting removal of LDL from the liver.

In response to a potential price war looming between the pair of PCSK9 inhibitors pharmacy benefit manager CVS Health recently asked the American College of Cardiology (ACC) and the American Heart Association (AHA) to revise guidelines for cholesterol-treatment to include previously disregarded numerical targets for LDL cholesterol.

"As PCSK9 inhibitors become available, the current cholesterol management guidelines do not provide clarity as to how these expensive new medications could fit in the treatment paradigm, potentially resulting in some scenarios where a prescriber could consider a PCSK9 inhibitor for a low-risk patient," said William H. Shrank, MD, MSHS, chief scientific officer at CVS Health. "There is a need for consensus around management strategies for patients with high cholesterol given that the cost differential between proven older therapies and this new class of drugs is substantial. In fact, if used broadly, PCSK9 inhibitors would likely be the most costly class of medications we've seen thus far."

Prior ACC/AHA guidelines recommended treating patients for specific cholesterol targets based on cardiovascular risk. The guidelines were discarded, however, for more aggressive cholesterol management with highly effective statins in patients with an elevated cardiovascular disease risk.

Prime estimates PCSK9 inhibitors could add between $0.93 and $6.71 per member per month (PMPM) to commercial insurance coverage costs and up to $15.66 PMPM to Medicare coverage costs depending on the conditions PCSK9 inhibitors are approved to treat. If the drugs were prescribed to 40% of approximately 600,000 Americans with abnormally high cholesterol levels that statins don’t reduce, it could add an additional $2.1 billion annually in new costs.

“If not managed appropriately, the costs of PCSK9s could have a dramatic impact on overall drug spend starting later this summer,” said Pete Clagett, senior vice president of integrated care and specialty at Prime. “In a couple years, PCSK9s could cost America the equivalent of $73 per person per year.”