Both rheumatologists and dermatologists are watching this game-changing drug closely—here’s why
Industry Buzz
Any medication for dermatomyositis that demonstrates efficacy in clinical trials would be a welcome addition to the options available to dermatologists and rheumatologists for treating this rare, multisystem disease.
—Richard Krathen, MD, FAAD, FACMS
In many ways, [a brepocitinib approval] reflects a broader movement in autoimmune disease toward mechanism-based therapy rather than nonspecific immunosuppression.
—Tanya Evans, MD
Patients with dermatomyositis, a disease resulting in high patient burden, may have more hope now that brepocitinib—a first-in-class, oral, selective TYK2/JAK1 inhibitor from Priovant Therapeutics—has been granted a Prescription Drug User Fee Act date for August 29.[]
In the 52-week VALOR phase 3 trial, the drug outperformed placebo in several critical categories, including total improvement score, skin disease activity, glucocorticoid tapering, and functional disability.[]
Of the 241 randomized trial participants, 81 received brepocitinib 30 mg, 81 received brepocitinib 15 mg, and 79 received a placebo. The findings showed that brepocitinib 30 mg was superior to placebo across every endpoint, with improvements observed very quickly—as early as week 4.[]
Richard Krathen, MD, FAAD, FACMS, Founder at Florida Dermatology Specialists, says that he diagnoses and treats dermatomyositis, but that’s quite rare in practice. “Any medication for dermatomyositis that demonstrates efficacy in clinical trials would be a welcome addition to the options available to dermatologists and rheumatologists for treating this rare, multisystem disease,” he says. “Options for other effective therapeutic options outside of systemic steroids are great progress."
A targeted therapy with multiple mechanisms
Tanya Evans, MD, dermatologist and medical director of the Skin Cancer Program at the Melanoma Clinic at MemorialCare Saddleback Medical Center, agrees wholeheartedly, saying brepocitinib could be one of the most important advances in dermatomyositis management in years.
“Dermatomyositis has long been a frustrating disease to treat because patients often have persistent skin disease, progressive muscle weakness, and prolonged dependence on corticosteroids despite multiple immunosuppressive therapies,” she says. “The fact that brepocitinib demonstrated improvement across muscle, skin, physical function, and steroid reduction makes this particularly meaningful.”
What makes brepocitinib especially exciting, Dr. Evans says, is that the drug is targeted at the pathways responsible for dermatomyositis. “By selectively inhibiting TYK2 and JAK1, the drug suppresses signaling from type I and II interferons, IL-6, IL-12, and IL-23 cytokine pathways strongly implicated in the inflammatory cascade driving dermatomyositis. In many ways, this reflects a broader movement in autoimmune disease toward mechanism-based therapy rather than nonspecific immunosuppression.”
Rapid response, reduced steroid use
But that’s not all. The rapidity and durability of response are worth celebrating. “That is highly relevant in a disease where patients can experience substantial functional impairment, pain, fatigue, and visible skin involvement that significantly affects quality of life,” Dr. Evans notes.
But for clinicians, the most practice-changing aspect of the study is the turn away from long-term glucocorticoid exposure.
“[It] contributes to osteoporosis, diabetes, infection risk, cardiovascular disease, weight gain, mood changes, and muscle damage itself,” Dr. Evans stresses. “Seeing that 62% of patients on the 30-mg dose tapered to ≤2.5 mg prednisone daily, and 42% discontinued steroids altogether, is remarkable. For many patients, reducing chronic steroid exposure could be life-changing.”
There’s yet another all-important point worth considering: “For dermatologists and rheumatologists, this may also strengthen the collaborative derm-rheum model of care,” Dr. Evans says. “Dermatomyositis sits directly in the middle of both specialties, and a drug proving efficacy in both skin and muscle domains reinforces the importance of integrated management.”