FDA Drug Approvals, Dermatology — 2022 Midyear Review

August 24, 2022

Vtama (tapinarof topical)

Tapinarof is a first-in-class aryl hydrocarbon receptor (AhR) agonist for treatment of plaque psoriasis in adults. Efficacy of tapinarof in psoriasis is attributed to its binding and activation of AhR, a ligand-dependent transcription factor, leading to the downregulation of proinflammatory cytokines, including interleukin-17.

Approval was based on the PSOARING clinical trials, which compared use versus topical placebo. Approximately 35-40% of patients who received active drug had clear or almost clear scores after 12 weeks, as compared to 6% of patients on placebo. N Engl J Med 2021;385:2219-2229

Cibinqo (abrocitinib)

Abrocitinib is an oral Janus kinase (JAK)-1 inhibitor indicated for refractory moderate-to-severe atopic dermatitis in adults whose disease is not adequately controlled with other systemic therapies or for whom those therapies are inadvisable. JAK1 inhibitors reduce interleukin-4 (IL-4) and IL-13 signaling.

Approval was based on the JADE COMPARE trial, which compared abrocitinib 200 mg or 100 mg once daily, dupilumab 300 mg SC every other week (after a 600-mg loading dose), and placebo. Additionally, all patients received topical therapy. An IGA response at week 12 was observed in 48.4% of patients in the 200-mg abrocitinib group, 36.6% in the 100-mg abrocitinib group, 36.5% in the dupilumab group, and 14% in the placebo group (P<0.001 for both abrocitinib doses vs placebo). An eczema area and severity index-75 (EASI-75) response at week 12 was observed in 70.3%, 58.7%, 58.1%, and 27.1%, respectively (P<0.001 for both abrocitinib doses vs placebo). The 200-mg dose, but not the 100-mg dose, of abrocitinib was superior to dupilumab with respect to itch response at week 2. N Engl J Med. 2021 Mar 25;384(12):1101-1112

Vijoice (alpelisib)

Alpelisib is the first drug approved for patients aged 2 years and older with severe manifestations of PIK3CA-related overgrowth spectrum (PROS) who require systemic therapy. Klippel-Trenaunay-Weber syndrome is part of this spectrum of diseases. FDA approval of alpelisib was supported by real-world evidence from the open-label EPIK-P1 trial. A retrospective chart review showed patients treated with alpelisib had reduced target lesion volume and improvement in PROS-related symptoms and manifestations. After 24 weeks, 27% of patients (10/37) achieved a confirmed response to treatment, defined as 20% or greater reduction in the sum of PROS target lesion volume. Also, 23 of 31 patients (74%) showed some reduction in target lesion. Prescribing Information

Olumiant (baricitinib)

Baricitinib is the first systemic treatment to gain FDA approval for adults with severe alopecia areata. Baricitinib is a Janus kinase (JAK) inhibitor that blocks phosphorylation and activation of signal transducers and activators of transcription (STATs), which modulate intracellular activity, including gene expression involved in the inflammatory pathway.

Approval was based on two phase 3 trials, BRAVE-AA1 and BRAVE-AA2. In BRAVE-AA1, 22% of the 184 patients who received baricitinib 2 mg and 35% of the 281 patients who received 4 mg achieved adequate scalp hair coverage, compared to 5% of the 189 patients who received placebo. In BRAVE-AA2, 17% of the 156 patients who received baricitinib 2 mg and 32% of the 234 patients who received 4 mg achieved adequate scalp hair coverage, compared to 3% of the 156 patients who received a placebo. Results were statistically significant for each treatment group compared to placebo (P<0.001). N Engl J Med 2022 May 5;386(18):1687-1699

Opzelura (ruxolitinib topical)

Ruxolitinib topical cream gained approval in July 2022 for treatment of adults and adolescents aged 12 years and older with nonsegmental vitiligo. Approval was based on data from two phase 3 trials (TRuE-V1 and TRuE-V2) that evaluated the safety and efficacy of ruxolitinib cream compared to vehicle in more than 600 people.

Topical ruxolitinib resulted in significant improvements in vitiligo area scoring index (VASI), which represent improvements in facial and total body repigmentation, at week 24 (primary analysis) compared to vehicle and in an open-label extension at week 52.

Results at week 24, which were consistent across both studies, showed approximately 30% of patients treated with topical ruxolitinib achieved at least 75% improvement from baseline in facial repigmentation (F-VASI75), the primary endpoint, compared to approximately 8% and 13% of patients treated with vehicle in TRuE-V1 and TRuE-V2, respectively. At week 52, approximately 50% of ruxolitinib-treated patients achieved F-VASI75.

Additionally, at week 24, more than 15% of patients treated with topical ruxolitinib achieved at least 90% improvement from baseline in F-VASI (F-VASI90), compared to approximately 2% of patients treated with vehicle. At week 52, the percentage of ruxolitinib-treated patients who achieved F-VASI90 doubled to approximately 30%. Medscape Medical News

Other dermatology approvals

Rinvoq (upadacitinib) – JAK inhibitor that gained approval by the FDA for refractory moderate-to-severe atopic dermatitis, including adults and adolescents aged 12 years and older.

Skyrizi (risankizumab) – New indication approved for adults with active psoriatic arthritis.

Zoryve (roflumilast topical) – New dosage form approved for adults and adolescents with plaque psoriasis

Juvederm Volbella XC (hyaluronic acid, non-animal stabilized) – New indication approved for dermal filler for improvement of infraorbital hollowing.


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