2025 FDA dermatology approvals: A new gene therapy and two targeted therapies

The year 2025 brought notable advances to dermatologic care, highlighted by the FDA's new drug authorization of a diverse set of therapies, including the first-ever gene therapy for a rare skin disorder. These approvals spanned an autologous cell-based gene therapy, a topical Janus kinase (JAK) inhibitor and an oral Bruton's tyrosine kinase (BTK) inhibitor, offering more molecularly precise interventions and expanded options for patients across a spectrum of dermatoses.

First Gene Therapy Approved for Recessive Dystrophic Epidermolysis Bullosa

A notable approval for a rare disease arrived in April with the approval of Zevaskyn (prademagene zamikeracel), which is the first autologous, cell-based gene therapy for treating recessive dystrophic epidermolysis bullosa (RDEB), a rare, inherited disorder caused by mutations in the COL7A1 gene. This mutation prevents the production of a protein critical for anchoring the skin's dermal and epidermal layers, resulting in severe skin fragility, chronic blistering and non-healing wounds.

“The FDA approval of Zevaskyn marks a monumental step forward for individuals living with RDEB and their families, offering a much-needed, long-lasting treatment option for this devastating condition and providing hope for improved quality of life for these patients,” Anna L. Bruckner, M.D., co-director of the Epidermolysis Bullosa Clinic at Children’s Hospital of Colorado and professor of dermatology at the University of Colorado School of Medicine, said in a statement.

Zevaskyn harvests a patient's own skin cells, then genetically modifies them to express the correct COL7A1 gene and finally cultures these corrected cells into sheets. These cell sheets are surgically applied as a one-time treatment to chronic wound sites. Clinical data demonstrated that this approach promoted sustained wound healing and improved skin adhesion, addressing the fundamental genetic pathology of the disease.

The approval was based on the results of the phase 3 VIITAL study. This multicenter, randomized, intrapatient-controlled trial showed statistically significant healing of 43 large and chronic wounds treated with a single surgical application of Zevaskyn. At six months, 81% of wounds treated with Zevaskyn showed 50% or more healing from baseline compared with 16% of matched control wounds that were treated with standard of care. The study results were published in July in The Lancet.

The cost for this one-time treatment has been reported at $3.1 million.

“Based on the strength of our data across clinical trials, we are confident in Zevaskyn’s ability to deliver long-term results after a single treatment application,” Madhav Vasanthavada, Ph.D., MBA, chief commercial officer of Abeona, said in a statement. “We are committed to working closely with both commercial and government payers on outcome-based agreements that stand behind the promise of Zevaskyn for patients, and expedite access.”

Targeting Chronic Hand Eczema With Topical JAK Inhibition

In July, Anzupgo (delgocitinib) cream was approved, becoming the first and only therapeutic agent specifically indicated for the treatment of moderate-to-severe chronic hand eczema in adults in the U.S. Chronic hand eczema is a long-lasting form of eczema that has historically lacked targeted, non-corticosteroid topical options and impacts nearly one in 10 U.S. adults.

“Chronic hand eczema can be a very difficult disease for adults to manage, especially given the lack of treatment options in the U.S. until now,” Robert Spurr, executive vice president and president of North America at LEO Pharma, said in a statement.

Anzupgo is a topical pan-JAK inhibitor and works by blocking the activity of the JAK-STAT signaling pathway, a central component in the inflammatory cascade that drives the symptoms of chronic hand eczema. By providing highly concentrated, localized inhibition of this pathway, Anzupgo offers an effective, steroid-free option for patients who have not responded adequately to traditional topical treatments or who cannot use them.

The approval was based on the results of 2 pivotal phase 3 trials, DELTA 1 and DELTA 2. These randomized, double-blinded, vehicle-controlled studies were conducted at more than 100 trial centers in Europe and Canada. Roughly one-fourth (20% in DELTA 1 and 29% in DELTA 2) of patients treated with Anzupgo met the primary end point of an Investigator's Global Assessment for Chronic Hand Eczema score of 0 (clear) or 1 (almost clear) compared with 8% (10% in DELTA 1 and 7% in DELTA 2) of patients receiving the cream vehicle.

The wholesale cost in the U.S. of Anzupgo is $804.18 per tube.

Oral BTK Inhibition for Chronic Spontaneous Urticaria

In October, the FDA approved Rhapsido (remibrutinib), an oral treatment for adult patients with chronic spontaneous urticaria (CSU). CSU is characterized by recurrent, unpredictable hives and intense itching, affecting approximately 1.7 million individuals in the U.S., with a substantial portion remaining symptomatic despite high-dose antihistamines.

Rhapsido is the first and only oral BTK inhibitor approved for this indication. The approval was based on the results from the phase 3 REMIX-1 and REMIX-2 trials. Patients treated with Rhapsido had a significantly greater decrease in their urticaria activity score during a 7-day period (UAS7) at week 12. Close to half of patients (49.8% in REMIX-1 and 46.8% in REMIX-2) treated with Rhapsido had a UAS7 of 6 or lower compared with only 24.8% of patients in REMIX-1 and 19.6% of patients on the placebo. In addition, 31.1% in REMIX-1 and 27.9% in REMIX-2 in the Rhapsido group achieved a UAS7 score of 0 compared with only 10.5% and 6.5%, respectively, on placebo.

“The approval of remibrutinib is an important development in CSU care. It quickly reduces symptoms, offering patients control of the hives and itching that they experience on a daily basis,” Giselle Mosnaim, M.D., MS, an allergist and immunologist from Endeavor Health, clinical associate professor at the University of Chicago Pritzker School of Medicine, and REMIX trial investigator, said in a statement. “This is significant because it expands beyond existing injectable treatments and gives patients an oral option that can easily be incorporated into their daily lives.”

Rhapsido costs $4,521 for a 30-day supply.