⚡First Single-Treatment EB Gene Therapy Approved

Good morning — it’s Saturday, May 3, and we’re covering the first FDA-approved single-application gene therapy for EB, new projections that measles could become endemic in the US, and more.

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Need To Know

FDA Approves First Single-Treatment Cell-Based Gene Therapy for EB

The FDA has approved Abeona Therapeutics’ ZEVASKYN (prademagene zamikeracel), the first autologous cell-based gene therapy requiring only a single application to treat recessive dystrophic epidermolysis bullosa (RDEB).

Approval was based on the Phase 3 VIITAL trial, which enrolled 11 patients with 86 large, chronic wounds. At six months, 81% of wounds treated with ZEVASKYN achieved ≥50% healing, compared to 16% of matched control wounds receiving with standard care (P < 0.0001). Safety data were favorable, with procedural pain and itching reported in fewer than 5% of patients.

In a separate Phase 1/2a study of seven patients with 38 chronic wounds, treated sites demonstrated durable responses, maintaining long-term improvement over a median follow-up of seven years (range: 4–8 years).

ZEVASKYN is produced by harvesting a patient’s keratinocytes, inserting a functional COL7A1 gene ex vivo using a retroviral vector, and culturing the cells into epithelial sheets. Up to 12 sheets—each the size of a credit card—can be grafted in a single procedure to cover large wounds or multiple distinct sites.

Priced at $3.1 million per treatment, ZEVASKYN will be one of the world’s most expensive medicines. It offers a one-time alternative to Krystal Biotech’s Vyjuvek, a topical gene therapy applied weekly and priced at roughly $631,000 per year. Abeona plans to offer partial refunds if retreatment is needed within three years.

Measles Predicted to Become Endemic in the US Within 20 Years

Although the US declared measles eliminated in 2000, declining childhood vaccination rates mean the disease could soon regain endemic status—defined as continuous local transmission lasting more than 12 months.

A modeling study in JAMA projects that at current immunization rates, measles could reestablish endemicity over the next 25 years, resulting in approximately 850,000 cases, 170,000 hospitalizations, and 2,500 deaths over that period. This outcome occurred in 83% of model simulations, with a mean time to endemicity of 21 years.

Even small changes in vaccination coverage could significantly alter this trajectory. A 10% drop could lead to over 11 million infections, while a 5% increase would reduce cases to fewer than 6,000—preventing endemic transmission entirely.

In contrast, the study found that rubella, diphtheria, and poliomyelitis are unlikely to reestablish endemicity unless vaccination rates decline significantly. 

The authors note key limitations, including the model’s simplified structure and the lack of modeling for reactive public health responses such as outbreak-driven vaccination.

They stress the urgency of improving coverage, warning that even small clusters of under-immunized people can trigger large-scale outbreaks.

Quick Hits

> Ivarmacitinib Shows Early Itch Relief and Strong Efficacy in Atopic Dermatitis: Ivarmacitinib, a highly selective oral JAK1 inhibitor, showed strong and rapid efficacy in patients with moderate to severe atopic dermatitis, according to a Phase 3 trial published in JAMA Dermatology. The study included a 16-week randomized trial comparing 4 mg and 8 mg ivarmacitinib with placebo, followed by a 36-week extension phase in which all participants received active treatment. Among 336 patients, 42% on the 8 mg dose achieved clear or almost clear skin (IGA 0/1) at week 16, compared to 9% on placebo (P < 0.001). Additionally, 66% reached at least a 75% reduction in disease severity (EASI 75) versus 22% with placebo (P < 0.001). Clinically meaningful itch reduction (≥4-point WI-NRS improvement) was seen as early as day 3. The safety profile was favorable. Two serious adverse events in the 4 mg group—sepsis and varicella—were possibly treatment-related, though both patients recovered. Ivarmacitinib’s 9-fold selectivity for JAK1 over JAK2 may help limit off-target hematologic effects.

> Rilzabrutinib Effective In Chronic Spontaneous Urticaria: In a Phase 2 trial published in JAMA Dermatology, Sanofi’s rilzabrutinib—a Bruton tyrosine kinase (BTK) inhibitor—met all primary and most secondary endpoints. Investigators randomized 143 antihistamine-refractory, omalizumab-naïve patients with moderate to severe chronic spontaneous urticaria (CSU) to receive rilzabrutinib or placebo while continuing background antihistamine therapy. At week 12, 34% of patients receiving rilzabrutinib 1200 mg/day achieved well-controlled disease (UAS7 ≤6), compared to 11% on placebo (P = 0.04). Clinical effects were apparent as early as week 1 and supported by reductions in biomarkers including anti-FcεRI antibodies and interleukin-31. The drug was well tolerated, though diarrhea, nausea, and headache were more common in the treatment group. Rilzabrutinib selectively and reversibly inhibits BTK, which plays a key role in IgE- and IgG-mediated mast cell and basophil activation, as well as autoantibody production.

> Best Responders to Novel Calcipotriol and Betamethasone Cream in Psoriasis: A post hoc analysis of two Phase 3 trials identified “best responders” to calcipotriol and betamethasone dipropionate (CAL/BDP) cream formulated with polyaphron dispersion (PAD) technology in 1271 adults with mild-to-moderate psoriasis. Best response was defined as clear or almost clear skin (PGA 0/1), minimal disease severity (modified PASI <2), and no impact on quality of life (DLQI 0/1). The cream outperformed both CAL/BDP gel and vehicle: at week 8, 27% of users met the best responder criteria, compared to 16% on gel and 4% on vehicle (P < 0.05 for both). Among users with baseline DLQI <7 and modified PASI <4, 64% achieved this outcome, suggesting greater efficacy in select patients. PAD technology improves skin penetration while offering a more cosmetically acceptable alternative to greasy or sticky formulations. Limitations include the exploratory design and lack of real-world validation for response predictors.

> First Randomized Trial Finds No Link Between Creatine and Hair Loss: A randomized controlled trial in the Journal of the International Society of Sports Nutrition found no evidence that creatine—a widely used supplement to enhance muscular strength and training performance—causes hair loss. Thirty-eight resistance-trained males received 5 g/day of creatine monohydrate or placebo for 12 weeks. No significant differences emerged between groups in dihydrotestosterone levels or hair parameters, including density, follicular unit count, and cumulative shaft thickness. Minor changes in testosterone levels occurred over time but were seen in both groups. Despite prior speculation and limited evidence linking creatine to elevated dihydrotestosterone, this trial found no corresponding impact on hair health. Limitations include the short duration, small sample size, and exclusion of female participants.

Derm Picks

Quizzes, cases, and perspectives to sharpen your clinical eye.

🖼️ Image Challenge

• A painful rash on the thigh after walking on sea rocks (View)

• Scarring alopecia in a 66-year-old woman (View)

• An inguinal annular plaque with central maceration (View)

• A 67-year-old man with split fingernails, underdeveloped thumbnails, and triangular lunulae (View)

🔎 Under the Dermatoscope

• Dermoscopy of traction alopecia in black patients: a literature review (with images) (View)

✂️ Cut & Close

• How to make a wick: a controlled and accurate cryotherapy technique for benign lesions (paywall restricted) (Read)

📚 Guides & Reviews

• Canadian consensus guidelines for the management of atopic dermatitis with topical therapies (Read)

• Colloidal oat as an emollient ingredient for the prevention and treatment of atopic dermatitis in infants (Read)

• The relative efficacy and safety of monotherapies for alopecia areata: a network meta-analysis study (Read)

• Is there still a role for clindamycin and rifampicin in the treatment of hidradenitis suppurativa: a review of the literature (paywall restricted) (Read)

• A guide to the management of hidradenitis suppurativa in pregnancy and lactation (Read)

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In a 6-month pilot study, a wearable electrostimulation device increased hair density by 19% and thickness by 9% in men with androgenetic alopecia, with no adverse events (More)