Sarepta and Capricor learned of key regulatory decisions from the media and investors, and Duchenne muscular dystrophy families have turned to the news for answers. Meanwhile, the FDA insists it remains committed to notifying companies of any regulatory action before sharing information with the media or public.
As Sarepta Therapeutics’ roller-coaster ride with its Duchenne muscular dystrophy gene therapy Elevidys continues to race off the tracks, patient families and clinicians are “looking at social media, they’re looking at the news constantly, trying to get an update,” according to Chet Villa, a professor in the Department of Pediatrics at Cincinnati Children’s Hospital Medical Center.
Families have been left reeling, Villa told BioSpace: “It leaves people in a very challenging position where there’s a lot of new data kind of flying around.”
Some of them were literally in the queue to receive their dose of Elevidys. Craig McDonald, director of the MDA Neuromuscular Disease Clinics at UC Davis, had a patient at a hotel, waiting to be treated, when the news began to break. “There were press releases going out from the FDA and from Sarepta” to provide clarification on previous information reported by an investment reporter regarding an FDA-requested hold on dosing with Elevidys for all patients. “Meanwhile, my poor patient and their family are sitting in a hotel room ready to get infused the next day,” McDonald told BioSpace.
Ultimately, Sarepta agreed to pause shipment of Elevidys at close of business on July 22, so McDonald’s patient was treated just prior to the voluntary hold. “But I think that whole method of communication, with what seemed to be leaks by the FDA to the media, put our family in a very difficult and stressful position, which I don’t think is appropriate,” he said.
The FDA’s public spat with Sarepta is a symptom of a larger problem, companies and patient advocates say. Capricor Therapeutics—which also works in the Duchenne muscular dystrophy (DMD) space—also found itself caught unawares when it was informed through non-official channels of the cancellation of an advisory committee meeting for its asset and then-Center for Biologics Evaluation and Research (CBER) director Vinay Prasad’s alleged plans to reject the drug.
“When you run a public company, you have to be able to respond when the investors call you, and so if you don’t know first, you really are sort of put off your game,” Capricor CEO Linda Marbán told BioSpace in an interview.
Sarepta Blindsided
Sarepta’s problems with Elevidys began this spring, with the deaths of two teenage patients due to acute liver failure, a known complication of the AAV vectors used to deliver gene therapies. The situation escalated earlier this month when the biotech reported the death of a third patient—an adult participant in a Phase I study for SRP-9004 in limb-girdle muscular dystrophy (LGMD), which uses the same underlying technology as Elevidys. The FDA then stepped in, requesting that Sarepta pause all shipments of Elevidys. Chaos ensued.
In over three decades, I’ve never seen the FDA been this engaged with investment reporters with these types of leaks without really engaging companies through appropriate channels.
When asked how he and his patients first received updates about Elevidys during the past two weeks, Villa responded: “It’s the press and social media. There’s always been a little bit of that, but the sheer volume and the seriousness of the information that’s being conveyed makes this a different time than usual.”
Sarepta itself has acknowledged this reality. In a statement on July 18, in which the biotech acknowledged and refused an “informal request” from the FDA to voluntarily halt shipment of Elevidys for all DMD patients, Sarepta said it “first heard of this potential request earlier in the day at the same time the public and our patient communities did, through media reports.”
“In over three decades, I’ve never seen the FDA been this engaged with investment reporters with these types of leaks without really engaging companies through appropriate channels,” McDonald said.
The leaks have begun to erode the foundation of the FDA’s credibility, one analyst noted. In an investor note Tuesday addressing Prasad’s sudden departure the previous evening, BMO Capital Markets’ Kostas Biliouris wrote of the CBER’s recent “missteps” around Elevidys, “Unprecedentedly, there were multiple press leaks from the FDA, negatively impacting the confidentiality/credibility of the FDA.”
It appears the leaks irked Prasad in the days leading up to his departure, too. In a letter to CBER colleagues on Saturday evening, obtained by STAT News and posted on LinkedIn by Acadia Strategy Partners principal Courtney Rice, Prasad also alluded to leaks within his department. “Some things I do find unacceptable,” he wrote, “These include surreptitiously recording meetings, screenshotting emails, and posting confidential FDA conversations on public forums/ social media or via leaking to the media.” Two days later, Prasad was gone.
Capricor Caught Off Guard
These recent communication issues go beyond the Sarepta saga. In June, Prasad canceled a previously scheduled advisory committee meeting for Capricor Therapeutics’ investigational cell therapy deramiocel, being developed for cardiomyopathy associated with DMD. Prasad made the decision unilaterally, according to a person familiar with the matter, STAT News reported at the time. Deramiocel was rejected early last month, with the FDA citing insufficient evidence to establish its effectiveness in a complete response letter.
But on the matter of the adcomm, Marbán was caught off guard. The meeting, which had been scheduled to take place on July 30, was listed on the Federal Register—and then, ten minutes later, it wasn’t.
“Investors watch these boards all day, every day, so my phone started blowing up with ‘your adcomm has been canceled. It’s been taken down. Why?’ I had no answers to give,” Marbán said. “I couldn’t make an official statement.”
Capricor reached out to Prasad and other FDA offices but got “no response whatsoever,” Marbán said, until one week later, the company received a one-line email saying the adcomm had been cancelled “for now.”
In addition, Capricor was contacted by a STAT reporter, Marbán said, seeking comment regarding the ouster of Nicole Verdun, the director of the FDA’s Office of Therapeutic Products, who was placed on administrative leave in June along with her deputy, Rachael Anatol.
“The real issue for us was when somebody from allegedly inside the agency allegedly told this reporter that Nicole Verdun and Rachael Anatol were put on administrative leave because of an argument about our file, and that Prasad wanted to CRL us and Nicole was fighting it,” Marbán said.
McDonald, who serves as lead investigator on Capricor’s HOPE-2 and HOPE-3 trials, agreed that biopharma companies should not learn about regulatory decisions through the media. “In my 30-plus years of doing clinical trials and research, I’ve never seen something like that from the FDA,” he said.
In an emailed statement, an FDA spokesperson told BioSpace the agency “remains committed to our long-standing practice to notify companies of any regulatory action before sharing information with the media or the public.”
Too Much Information?
Pat Furlong, founding president and CEO of Parent Project Muscular Dystrophy, also expressed concern with the way in which Duchenne patient families have been receiving information regarding Elevidys.
“Parents should not receive information from the press or from social media,” she told BioSpace. “They should receive it in ways that can be explained and understood.”
Indeed, even in its official communications, the FDA has been unusually forthcoming with the media. In an email to BioSpace on July 18, following the revelation of the death of the LGMD trial participant, a Health and Human Services official told BioSpace the agency was “taking a hard look at pulling [Elevidys] from the market.”
This type of public rumination is not new for the current HHS leadership. In April, FDA Commissioner Marty Makary raised questions when he posted on X regarding the delayed decision for Novavax’s COVID-19 vaccine: “To be clear, this is a new product that Novavax is trying to introduce to the market with a study of a different product from 2021. New products require new clinical studies.” This appeared to contradict the company’s understanding of the situation.
Since taking office, Makary has proposed several policies, including a new conditional pathway for therapies for ultrarare diseases, lowering industry user fees, and the Commissioner’s Priority Voucher Program to speed drug reviews—all without many details.
“The way the administration speaks in generalities about these proposals is not helpful for manufacturers who want to understand what their rights are, and observers trying to understand what the admin is doing,” Rachel Sachs, a professor of law at Washington University in St. Louis who works on health law and food and drug regulation, recently told BioSpace.
Marbán agreed with this sentiment. “I was raised with the idea that actions speak louder than words, and there’s a lot of words,” she said. She pointed to a recent Squawk Box interview where Makary discussed regulatory flexibility and accelerating drug approvals, “and I think, ‘this is fantastic. This is amazing. Now let’s see it happen.”
