The Food and Drug Administration has approved a new oral medicine to help lower cholesterol and, in the process, shown that a controversial program to speed up drug reviews may still be alive.
The agency on Thursday approved Lipfendra, a pill Merck & Co. developed to drop levels of low-density lipoprotein, or “bad” cholesterol in the blood. Lipfendra has been approved for use alongside diet and exercise in people with high cholesterol, including those with an inherited condition called “HeFH.”
Merck’s medicine is the first of its kind, a pill that’s able to block a cholesterol-regulating protein called PCSK9. There are already multiple injectable treatments available that inhibit PCSK9, but they’ve faced a long and winding road to convince payers and physicians of their worth. Sales totals disappointed until recently. The top medication, Amgen’s Repatha, generated $3 billion last year.
Merck has long contended that it will have a better chance of success with Lipfendra. Formerly known as enlicitide, the drug is a “macrocyclic” peptide taken via a daily pill. In a pair of large studies, Merck accumulated study data indicating the therapy might plummet cholesterol levels as powerfully as its injectable counterparts. Analysts at Leerink Partners found the results so compelling that, in a recent research note, they claimed that doctors “will view the options as interchangeable.”
Merck doesn’t yet have data definitively proving Lipfendra can lower the risk of death, an issue that caused insurers to push back against earlier PCSK9 drugs. Testing is ongoing. Still, the company has argued that it should be able to avoid reimbursement hassles, as it can produce Lipfendra at a low cost and, as a result, price it more competitively.
A 30-day supply of Lipfrenda will have a list price of $315, a spokesperson said in an email to BioPharma Dive.
“We believe this will enable broad access for patients, regardless of insurance, including the majority of Medicare patients with coinsurance requirements,” the spokesperson said.
That price represents a “meaningful discount” to the $500-$600 monthly cost of injectable PCSK9 drugs, as well as the $400 to $600 charged for a branded statin, wrote RBC Capital Markets analyst Trung Huynh. But it’s still unclear how Lipfendra’s ultimate “net” price will compare to the others, making Merck’s rebating strategy particularly important, Huynh added.
Lipfendra is one of the medications the big drugmaker is counting on to grow sales even after its top-selling cancer immunotherapy, Keytruda, loses patent protection. Questions about its post-Keytruda future have weighed on Merck, but it’s pointed to Lipfendra, a cancer drug it’s developing with Kelun-Biotech, and multiple other products it’s gained through company acquisitions to reassure investors about its future.
The drug’s review was accelerated by a controversial voucher program meant to drastically speed up the evaluations of medicines that align with “national interests.” That program was put in place by former FDA commissioner Martin Makary and slashes the process down to as little as one to two months. But the initiative has been widely criticized as being susceptible to political interference, and multiple vouchers — including Merck’s — were only awarded after developers cut drug pricing deals with the White House.
Makary’s resignation in May left the future of the voucher program unclear. But the agency held a public hearing in June, during which staffers spoke with a variety of organizations on how to improve the program. And now Merck, which received its voucher in December, has been issued a faster-than-usual approval.
The FDA didn’t mention the voucher specifically in its own statement, only noting that Lipfendra received a “priority review.”
“We’re proud of our work with regulators on this rigorous and efficient review process, bringing the first and only FDA-approved oral PCSK9 inhibitor to adults with high LDL-C,” the Merck spokesperson said in an email to BioPharma Dive.
RBC’s Huynh expects the drug to generate more than $5 billion in annual sales at its peak.
