Big Pharma just taught its critics a powerful lesson: Don’t mess with companies selling million-dollar drugs.
Even if they don’t work. Especially if they don’t work.
In May, Dr. Vinay Prasad joined the Food & Drug Administration as the top regulator for vaccines, as well as stunningly expensive “biological” medicines.1 I cheered his hire, writing Prasad knows “the games Big Pharma plays to win approvals for expensive new drugs that all-too-often have little benefit — and hidden risks.”
But Prasad apparently knew the games too well. Last week, Laura Loomer attacked him for years-old social media posts criticizing Donald Trump. The Wall Street Journal joined her. They won – with stunning speed. On Tuesday, Prasad resigned.
Prasad’s posts made him vulnerable, no doubt. But they’re not why he was targeted. He was targeted because he posed a direct threat to Big Pharma profits. As he proved to a company called Sarepta Therapeutics just days before Loomer came for him.
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(Truth-tellers aren’t always loved. But they’re needed. Please, stand with me.)
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On July 18, Prasad and the FDA told Sarepta, a Massachusetts biotechnology company, it needed to halt shipments of its gene therapy for Duchenne muscular dystrophy, or DMD.
DMD is devastating. It robs kids of their ability to walk and eventually kills them. Sarepta’s treatments work by helping them produce more of a protein they need.
In theory.
In reality, Sarepta has never shown its drugs actually benefit patients.
It has shown they raise levels of a protein that kids with DMD lack.2 But those protein levels are a marker on a blood test. They don’t necessarily translate into giving kids with DMD better, much less longer, lives.
Sarepta won approval for its first drug based on protein production in 2016. At the time, the company itself said “a clinical benefit [of the drug] has not been established” and promised “confirmatory trials.”
Nine years later, Sarepta still hasn’t completed those trials. But that hasn’t stopped it from selling the drug — for up to $1.5 million per year. Yes, per year.
Sarepta then asked the FDA to approve a gene therapy it called Elevidys — again based on protein levels. In 2023, the FDA reviewed Sarepta’s application and reported:
A notes that the clinical studies conducted to date do not provide unambiguous evidence that [the drug] is likely beneficial… Additionally, FDA has safety concerns related to the possibility of administering an ineffective gene therapy.
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(Vinay did need some hair gel. That’s not a crime. I hope.)
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But in the end, the FDA buckled to the pressure. It is very hard to say no to parents who are looking for any possible help for their sick children, and Sarepta has proved masterful at using them to apply pressure.
Unfortunately, the agency’s safety concerns proved real.
At least two people have died of acute liver failure from Elevidys. A third died from a similar Sarepta gene therapy in clinical trials.
Those deaths and the lack of proof Elevidys works — not concerns about its $3.2 million per-patient cost — were what led Prasad and the FDA to demand that Sarepta pull the drug. As Prasad said in the July 18 announcing the FDA’s move:
Protecting patient safety is our highest priority, and the FDA will not allow products whose harms are greater than benefits. The FDA will halt any clinical trial of an investigational product if clinical trial participants would be exposed to an unreasonable and significant risk of illness or injury.
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The news immediately caused Sarepta’s stock, which had already been tumbling, to crash another 35 percent. Days later, Loomer began her campaign against Prasad.
The question isn’t whether desperate patients have the right to try unproven treatments. It’s whether pharmaceutical companies should be allowed to sell and profit from drugs that haven’t been shown to benefit anyone and may even be dangerous.
In desperate cases, where people are dying of cancer or kids face terrible genetic illnesses, the FDA should move drugs forward as quickly as possible. But basic clinical trial work is still crucial. And if we’re going to allow these “accelerated approvals” based on non-clinical data or single-arm trials3 at all, the companies that run them need to agree to sell their drugs at the cost of production until they receive full approval.
As the nine-years-and-counting Sarepta delay shows, keeping pharmaceutical companies from profiting off drugs approved on weak data is the only way to make them keep their promises to run and finish confirmatory trials – and show real patient benefit.
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(Vinay went down. I’m still standing. With your help.)
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If parts of this disaster remind you of the mRNA Covid jab fiasco, you’re getting the picture. The shots were also moved forward based on rushed trials — at great profit to Pfizer and Moderna. We still have no idea how safe they are. And at this point we probably never will.
The irony is that stepping on science doesn’t benefit the industry in the long run either.
Drugmakers charge extraordinary prices for their medicines. They need real evidence their products work to justify those prices — and to convince doctors to prescribe them and patients to take them at all.
If the FDA doesn’t hold them to that standard, doctors and patients will. Even desperate parents of sick kids aren’t going to subject their children to gene therapies without hard proof they help.
Pfizer stock is lower than it was before Covid started, despite its relentless promotion of the mRNAs. And Sarepta is down almost 90 percent this year and made only small gains after Prasad was forced out.
Yes, Big Pharma scalped Prasad for his honesty. Congratulations.
If only it were half, or a tenth, as good at making drugs that work.
Most drugs used to be fairly simple chemical compounds like aspirin that were synthesized in factories or in some cases derived from plants. Generally, they could be taken in pill form and. But today, many new medicines are far more complex “biologics” – much larger proteins, grown in genetically engineered cells and frequently used to treat autoimmune conditions and cancer.
It’s even more complicated than that, because the version of the protein that Sarepta’s medicines help kids produce is different and less effective than the ones healthy people naturally make.
A single-arm trial is one where a drug has not been tested against placebo but given to all patients, in the hope of showing they do better than patients with their disease have fared on average in the past. The problem is that single-arm trials by definition cannot show a drug works better than placebo — perhaps the patients who enrolled were highly motivated and would have done better than the average patients anyway.
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Author: Alex Berenson
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