More drug launches miss the mark than those that succeed, and even highly anticipated FDA decisions should be taken with a grain of salt. But that doesn’t stop the industry (not to mention financial institutions and media) from frequently hyping drugs that ultimately don’t live up to expectations.
The latest cautionary tale comes from Aduhelm, Biogen and Eisai’s yanked Alzheimer’s drug that was once predicted to reach $10 billion sales in 2023. Aduhelm received accelerated FDA approval, but was riddled with clinical red flags from the start that ultimately led to dismay.
Here we take a look at four hyped drugs that ultimately flopped.
Aduhelm
The hype: Biogen’s Alzheimer’s drug boasted several superlatives when it won accelerated approval in 2021: It was the first new treatment approved for Alzheimer’s since 2003 and the first to target what is understood to be the underlying disease process.
The flop: Aduhelm generated controversy right out of the gate, from its questionable endpoints and FDA approval that bucked the advice of independent experts and prompted resignations, to a broad label that the FDA later revised. There was also Aduhelm’s high initial price tag of $56,000 that the Alzheimer’s Association called “simply unacceptable,” not to mention whispers of shady dealings between Biogen and the FDA that prompted a House investigation. But even slashing the price by half couldn’t save the drug, after cripplingly limited Medicare coverage and continuing questions about its effectiveness hampered prescribing. In the end, pitiful sales — it made just $1 million in Q4 of 2021 — and unshakable controversies prompted Biogen to finally scrap it and terminate further studies.
The takeaway: Huge controversies that cling to a drug can kill it — and kill it quickly.
Glybera
The hype: UniQure’s Glybera became the first approved gene therapy in the Western world when it won European approval in 2012. The drug treated recurring acute pancreatitis in patients with lipoprotein lipase deficiency and helped usher in the age of genetic technology.
The flop: Glybera treated a very rare disease, but for commercialization, it was a little too rare. Lipoprotein lipase deficiency’s prevalence is just one in 1 million in the general population. It also cost about $1 million per dose, making it the world’s most expensive drug. Not only were there not enough patients to shell out that kind of money, but there also weren’t enough patients, period.
“Glybera’s usage has been extremely limited and we do not envision patient demand increasing materially in the years ahead,” uniQure’s CEO, Matthew Kapusta, said in a statement about the company’s decision to pull the drug. Only 31 people received the drug as of 2018.
The takeaway: Showing up with a cure doesn’t mean a company will be able to successfully commercialize it.
Belviq
The hype: Weight loss drugs come with lots of hype — witness the current frenzy around Ozempic and Wegovy — and Arena Pharmaceuticals’ Belviq was no different. It was the first FDA-approved weight loss drug in 13 years when it got the nod in 2012.
The flop: Like Meridia before it and Fen-Phen before that, safety problems got the better of Belviq. In fact, Belviq was dogged by safety concerns from the start, having first been rejected by U.S. regulators in 2010 due to worries about heart valve problems in humans and tumors in rats. Despite warnings to the FDA from critics like Public Citizen, which called a potential approval “dangerous and unconscionable,” the agency was eventually swayed two years later after additional research seemed to allay cardiovascular concerns. However, those safety worries didn’t go away. It also didn’t help that Belviq only produced modest weight loss. Finally, in 2020, the FDA asked that Belviq be removed from the market because of an increased occurrence of cancer.
The takeaway: Safety problems combined with middling efficacy aren’t worth the risk for patients or regulators.
Palforzia
The hype: Roughly 1 million American children are allergic to peanuts, and 1 in 5 outgrow the allergy, the FDA noted when it approved Palforzia in January 2020 to treat peanut allergies in children aged 4 to 17. It was the first drug approved to treat peanut allergies in children.
The flop: Not every drug flop ends with it being yanked from the market. Sometimes it ends with a company sinking a lot of money only to divest it a couple years later. That’s what happened with Palforzia. Just a few months after approval, Nestlé acquired what shares it didn’t already own of Palforzia’s developer, Aimmune, for more than $2 billion to create what it called “a global leader in food allergy prevention and treatment.” But Palforzia requires multiple in-person treatments, and just months after its approval, the pandemic made in-person treatments impossible. Even after the world emerged from lockdown, Palforzia still never took off, and eventually, Nestle divested its Palforzia business to the biopharma Stallergenes Greer.
The takeaway: Rigorous treatment regimens can sink even the most promising drugs, especially during a global pandemic.
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