Krystal Biotech
NEWS
Novo Nordisk’s leadership gets another shake-up as President Trump promises to significantly slash prices for its GLP-1 drugs; Summit/Akeso, Exelixis and more present new data at ESMO 2025; Replimune pops as FDA accepts resubmitted BLA; FDA names first winners of Commissioner’s National Priority Voucher program; and more.
The company’s technology, a modified herpes simplex virus used to deliver gene therapies, was given the FDA’s new designation based on its approved topical skin cream. What this will mean for Krystal’s still-in-development eye drop is unclear.
After the FDA revoked Sarepta Therapeutics’ platform designation in July, Krystal Biotech is “poised to be the first major beneficiary” of this program, according to analysts at William Blair.
Under the FDA’s compassionate use program, an eyedrop formulation of Krystal Biotech’s Vyjuvek restored the vision of a teenager with the rare genetic disease dystrophic epidermolysis bullosa.
The topical treatment Vyjuvek got the FDA’s greenlight, making it the first redosable gene therapy and the first therapeutic for the rare skin disease dystrophic epidermolysis bullosa.
The FDA has four events lined up this week, including a target action date for Krystal Biotech’s gene therapy for a rare skin disease and an adcomm meeting for Pfizer’s RSV vaccine candidate.
The FDA has had a busy week, accepting drug applications, approving clinical trials and granting various special designations for Gamida Cell, Cellectis, Scynexis & more.
Krystal Biotech announced that it submitted a Biologics License Application to the FDA for its candidate B-VEC, intended for the treatment of dystrophic epidermolysis bullosa.
It was a particularly busy week for clinical trial announcements. Let’s take a look.
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