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Good morning, we’ve received a bunch of stories this morning about GLP-1 medicine, compounding, and biotechs’ interactions with the FDA. Let’s get into it.
The necessity-to-know this morning
- Abeona Therapeutics mentioned the FDA permitted its cell-based gene remedy for sufferers with recessive dystrophic epidermolysis bulosa, a uncommon pores and skin situation that causes blistering wounds. The brand new therapy will likely be bought below the model identify Zevaskyn.
- We’re deep into earnings season. This morning delivered first-quarter monetary reviews from Pfizer, Regeneron, AstraZeneca, Novartis, Incyte, and Verona Pharma.
That is the tip of GLP-1 compounding as we all know it
Branded GLP-1 medicine are not in scarcity. Compounding pharmacies and the telehealth corporations they work with are not allowed to supply cheaper copies. Meaning sufferers on compounded medicine will simply change to the branded remedies, proper?
The truth shouldn’t be that easy, and plenty of sufferers are literally left now with no good choices.
Some telehealth corporations that supplied compounded GLP-1s have disappeared altogether. Others are pursuing methods that current dangers, like altering prescriptions, mixing in components, or encouraging stockpiling of current compounded merchandise.
That leaves many sufferers again at sq. one, struggling to afford the brand-name medicines, which carry checklist costs of about $1,000 a month. Novo Nordisk and Eli Lilly have began providing their remedies at decrease costs of round $350 to $700 a month, however that’s nonetheless unaffordable for many sufferers.
“That is sort of like a lose-lose state of affairs,” one physician mentioned.
In the meantime, Novo is increasing its efforts to promote its lower-priced Wegovy on to sufferers. This morning, it introduced new agreements with telehealth corporations together with Hims & Hers and Ro. Learn extra.
Two biotechs, two very totally different experiences with FDA
Stealth BioTherapeutics, which is growing a therapy for a uncommon illness known as Barth syndrome, obtained a letter from the FDA late final week saying there was a delay in its overview of the drug. The company additionally didn’t point out when it could now full its overview.
Stealth has already struggled for years to win approval for its drug, however latest occasions counsel this new delay will be traced to the newest workforce cuts and upheaval on the FDA, in line with a former official conversant in the matter.
FDA employees had gotten so far as discussing potential labeling for the remedy, a step that sometimes signifies the company is nearing approval for a drug, in line with two sources. Learn extra from STAT’s Ed Silverman.
However, one other biotech, Capricor Therapeutics, mentioned that its interactions with the FDA have been the identical as earlier than.
The company is almost midway via its overview of Caprcior’s cell remedy for Duchenne muscular dystrophy, and “the data requests have been flowing frequently and follow-up has been fast and environment friendly,” CEO Linda Marbán mentioned. Learn extra from STAT’s Adam Feuerstein.
‘Phenomenal’ information may develop attain of HPV vaccine
New information confirmed {that a} single dose of the vaccine used to stop an infection with HPV is simply as efficient as two, outcomes that would rework efforts to succeed in the three-quarters of kids globally who ought to obtain the vaccines however don’t.
In developed nations, two doses of the vaccines are presently advisable. However in a medical trial of 20,000 women who have been adopted for 4 1/2 years, whether or not one dose or two doses have been used, the outcomes have been equal. In all circumstances, the estimated efficacy of the vaccine was upwards of 97%.
Specialists have been quick to reward the outcomes, with one physician calling them “phenomenal.”
Learn extra from STAT’s Matt Herper.
Makary begins to echo RFK Jr.’s vaccine skepticism
Earlier than Marty Makary was confirmed to steer the FDA, specialists hoped he would defend the company from the vaccine criticism of his boss, HHS Secretary RFK Jr. Colleagues had described him as a dedicated scientist, and the biopharma business was broadly supportive of him.
However Makary’s latest actions counsel he’s simply as keen to make use of his energy and place to harshly scrutinize vaccines and vaccination coverage, my colleague Lizzy Lawrence writes.
As one instance — Makary over the weekend signaled that the FDA could depart from longstanding precedent by requiring Covid vaccine makers to submit new effectiveness information earlier than adjusting their merchandise for brand new strains.
The pharma business’s status continues to slide
The pharmaceutical business’s status amongst affected person advocacy teams dipped in 2024, the second consecutive 12 months it’s slipped, in line with a brand new survey.
Nearly all of teams have been glad with drug corporations, with 56% reporting that the business had an “glorious” or “good” status. However the consecutive declines come after a number of years through which the business was seen extra positively.
Notably, the business fared most poorly when it got here to drug pricing. Simply 14% of the affected person teams reported that corporations had “glorious” or “good” reputations for honest pricing, and solely 17% mentioned the identical regarding pricing transparency.
Learn extra from STAT’s Ed Silverman.
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