Sarepta’s $3.2M Gene Therapy Mess: FDA Fights, Biotech Tricks, and MAGA Duped Big Time

Duchenne muscular dystrophy is a soul-crushing, rare, terminal disease that destroys young kids’ muscles, often killing them as young adults.

A ray of light emerged a few years ago, when a company called Sarepta offered hope in the form of a gene therapy called Elevidys to deliver a micro-dystrophin gene using the AAVrh74 vector to produce a shortened version of the dystrophin protein, which is deficient in DMD patients. Unfortunately, the proof of concept phase 3 EMBARK trial, completed in 2023, that aimed to measure improvement in motor function using the North Star Ambulatory Assessment (NSAA) score as its primary endpoint didn’t hit the mark. The trial did not meet its primary endpoint, showing no significant improvement in NSAA scores compared to placebo. However, secondary endpoints showed promise: Elevidys increased micro-dystrophin expression in muscle tissue and improved individual mobility measures, like time to rise from the floor and 10-meter walk/run times. There were also some indications of improved walking ability in kids aged four to seven. Based on these secondary outcomes, Peter Marks, then director of the FDA’s Center for Biologics Evaluation and Research (CBER), granted accelerated approval in June 2023 for boys aged four to five who could still walk, and later in June 2024, expanded it to include patients four and older, ambulatory or not. This decision was controversial because multiple FDA review teams recommended rejection due to insufficient evidence of clinical benefit, and Marks overruled them, relying on micro-dystrophin levels as a surrogate marker. This raised eyebrows among long time regulators and parents of muscular dystrophy patients being asked to participate in these trials.

The frustration was perhaps best encapsulated by Catherine Collins, a mother of a child with Duchenne’s, and a vocal advocate in the DMD community, who made a direct and emotional appeal during a July 2024 Parent Project Muscular Dystrophy Conference Q&A session, confronting a Sarepta representative about their slow walk when it came to releasing data and doing the confirmatory follow up trials they were required to do.

“You have 3 drugs that are accelerated approval that are conditioned on future clinical trial that have not yet been completed. Now you're on your 5th drug sitting up here asking all of us to be part of your drug, but you don't do any of the follow up studies, you don't produce any of the data, you haven't delivered any of this evidence for this 3.2 million dollar drug. You don't think you can do a little more effort, and give us a little bit more, a little bit more information. Now I have got my spidey sense up… where you have 5 drugs in the work that have not been fully approved .. while they are accelerated approval, the confirmatory trials that were required by the FDA have not been done. And I'm talking years. So if you're looking for your son who isn't ambulatory to get the results of some study from you... Fat chance!!......When you expand a label from 4-5 years to everybody, you don't think we're going to get suspicious? … This is a lifetime decision… You're asking us to put everything on the line but you give us nothing in return… We are the people who give you millions of dollars and you don’t give us anything back which is facts, data and science… you’re just like… taking money”

Catherine Collins

The actual video is a must watch masterpiece:

The other major concern was safety. Snipping you DNA forever is scary enough, but it was well known early on that the AAVrh74 vector used to deliver the therapy activated the immune system and could wreck the liver.

With reports of a 3rd and 4th death after receiving a Sarepta therapy, critics fears of the safety profile may be well founded. Sneakily, the third child that died from liver failure after getting a different Sarepta gene therapy, (not Elevidys) was publicy reported right after Sarepta’s July 17 earnings call where they did not disclose the bad outcome.

The CBER, now led by a new Sheriff (Vinay Prasad) who took over the position formerly held by Peter Marks, responded to the death by immediately requested Sarepta voluntarily halt shipment of its drug. Sarepta gave the FDA the proverbial middle finger, and initially refused, stating in a public letter to the FDA that they saw “no new safety signal in the approved population” and believed the deaths were related to “unique patient conditions” rather than Elevidys itself. They argued continued access was critical for DMD patients, citing the lack of alternative treatments, and compliance with existing FDA approvals.

But here’s where things get juicier than a Game of Thrones episode. Right after Prasad’s FDA moved on Sarepta, Laura Loomer—yep, that Laura Loomer— the queen bee of MAGA influencers, who wields a large following despite social media bans, and has claimed multiple Trump administration scalps including Maurene Comey (yep, James’s kid), CBP’s Monte Hawkins and DOJ’s Carolyn Feinstein for their supposed anti-Trump views— dropped a spicy hit piece on her website and X, painting Prasad as a ‘progressive leftist’ sabotaging Trump’s ‘Make America Healthy Again’ agenda. She dug up his old publicly professed love for liberal senators Warren and Sanders, and accused him of dragging his feet on FDA drug approvals tied to the Right to Try legislation — a 2018 Trump 1.0 law allowing terminally ill patients early access to drugs in development.

This is a particularly wild accusation because Prasad spent most of the last 4 years being roasted as a ‘right-wing grifter’ during COVID for slamming Biden’s lockdowns and masks. Now he’s a far-left villain?

What’s particularly interesting is that Loomer, who isn’t exactly FDA’s gossip columnist, specifically highlighted recent deadlines on novel biological therapies the FDA had missed. The whole affair certainly reeks of a story planted by biotech interests interested in axing Prasad to keep company revenues intact and stock prices high.

Given the timing of the hit piece, it’s possible the Sarepta hype squad could have fed her this hit to kneecap Prasad for questioning their pricey, iffy drug. But here’s the irony: Loomer’s MAGA/MAHA crew, who normally lose their minds over COVID mRNA vaccines, screaming they’re ‘gene-modifying’ (a hotly debated claim), are now cheerleading for Elevidys—an actual gene therapy with three deaths linked to similar treatments and sketchy proof it works—just to dunk on Prasad.

Talk about flipping the script! It’s worth repeating: Loomer’s crew is upset Prasad’s FDA division isn’t approving therapies like Elevidys fast enough — a $3.2 million gene therapy that might not even work. !

While the FDA circus rages, with biotech investors swooning over Sarepta and MAGA foot soldiers being played like fiddles, let’s not lose sight of what’s at stake: desperate parents watching their kids waste away from Duchenne muscular dystrophy, clinging to any shred of hope. Here’s hoping the big shots stirring this mess haven’t forgotten the real tragedy.

Anish Koka is a full time cardiologist and a part time medical commentator. Follow him on @X (formerly Twitter) @anish_koka . You can also listen to him on The Doctors’ Lounge . podcast.