Elaine Chen , 2025-04-29 13:58:00
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Good morning, we’ve got a bunch of news this morning about GLP-1 drugs, compounding, and biotechs’ interactions with the FDA. Let’s get into it.
The need-to-know this morning
- Abeona Therapeutics said the FDA approved its cell-based gene therapy for patients with recessive dystrophic epidermolysis bulosa, a rare skin condition that causes blistering wounds. The new treatment will be sold under the brand name Zevaskyn.
- We’re deep into earnings season. This morning delivered first-quarter financial reports from Pfizer, Regeneron, AstraZeneca, Novartis, Incyte, and Verona Pharma.
This is the end of GLP-1 compounding as we know it
Branded GLP-1 drugs are no longer in shortage. Compounding pharmacies and the telehealth companies they work with are no longer allowed to offer cheaper copies. That means patients on compounded drugs will just switch to the branded treatments, right?
The reality is not that simple, and many patients are actually left now with no good options.
Some telehealth companies that offered compounded GLP-1s have disappeared altogether. Others are pursuing strategies that present risks, like changing prescriptions, mixing in additives, or encouraging stockpiling of existing compounded products.
That leaves many patients back at square one, struggling to afford the brand-name medications, which carry list prices of about $1,000 a month. Novo Nordisk and Eli Lilly have started offering their treatments at lower prices of around $350 to $700 a month, but that’s still unaffordable for many patients.
“This is kind of like a lose-lose situation,” one doctor said.
Meanwhile, Novo is expanding its efforts to sell its lower-priced Wegovy directly to patients. This morning, it announced new agreements with telehealth companies including Hims & Hers and Ro. Read more.
Two biotechs, two very different experiences with FDA
Stealth BioTherapeutics, which is developing a treatment for a rare disease called Barth syndrome, received a letter from the FDA late last week saying there was a delay in its review of the drug. The agency also did not indicate when it may now complete its review.
Stealth has already struggled for years to win approval for its drug, but recent events suggest this new delay can be traced to the latest workforce cuts and upheaval at the FDA, according to a former official familiar with the matter.
FDA staff had gotten as far as discussing potential labeling for the medication, a step that typically indicates the agency is nearing approval for a drug, according to two sources. Read more from STAT’s Ed Silverman.
On the other hand, another biotech, Capricor Therapeutics, said that its interactions with the FDA have been the same as before.
The agency is nearly halfway through its review of Caprcior’s cell therapy for Duchenne muscular dystrophy, and “the information requests have been flowing on a regular basis and follow-up has been rapid and efficient,” CEO Linda Marbán said. Read more from STAT’s Adam Feuerstein.
‘Phenomenal’ data could expand reach of HPV vaccine
New data showed that a single dose of the vaccine used to prevent infection with HPV is just as effective as two, results that could transform efforts to reach the three-quarters of children globally who should receive the vaccines but don’t.
In developed countries, two doses of the vaccines are currently recommended. But in a clinical trial of 20,000 girls who were followed for 4 1/2 years, whether one dose or two doses were used, the results were equivalent. In all cases, the estimated efficacy of the vaccine was upwards of 97%.
Experts were fast to praise the results, with one doctor calling them “phenomenal.”
Read more from STAT’s Matt Herper.
Makary starts to echo RFK Jr.’s vaccine skepticism
Before Marty Makary was confirmed to lead the FDA, experts hoped he would shield the agency from the vaccine criticism of his boss, HHS Secretary RFK Jr. Colleagues had described him as a committed scientist, and the biopharma industry was broadly supportive of him.
But Makary’s recent actions suggest he’s just as willing to use his power and position to harshly scrutinize vaccines and vaccination policy, my colleague Lizzy Lawrence writes.
As one example — Makary over the weekend signaled that the FDA may depart from longstanding precedent by requiring Covid vaccine makers to submit new effectiveness data before adjusting their products for new strains.
The pharma industry’s reputation continues to slip
The pharmaceutical industry’s reputation among patient advocacy groups dipped in 2024, the second consecutive year it’s slipped, according to a new survey.
The majority of groups were satisfied with drug companies, with 56% reporting that the industry had an “excellent” or “good” reputation. But the consecutive declines come after several years in which the industry was seen more positively.
Notably, the industry fared most poorly when it came to drug pricing. Just 14% of the patient groups reported that companies had “excellent” or “good” reputations for fair pricing, and only 17% said the same concerning pricing transparency.
Read more from STAT’s Ed Silverman.
