Targeted therapies are medications that interfere with specific pathways involved in the immune response. Most targeted therapies used in myositis belong to a group known as Janus kinase (JAK) inhibitors. Unlike biologics, which are typically given by infusion or injection, targeted therapies are small-molecule medications taken orally. These treatments have shown promise for some patients with dermatomyositis, particularly those with difficult-to-control skin disease or interstitial lung disease.

Tofacitinib (Xeljanz)

Tofacitinib is a JAK inhibitor that blocks signaling pathways involved in inflammation and immune system activation. Studies have suggested that it may improve skin symptoms and overall disease activity in some patients with refractory dermatomyositis.

How it is administered

  • Taken orally as a tablet, usually once or twice daily depending on the formulation

Common side effects

  • Upper respiratory infections
  • Headache
  • Nausea
  • Elevated cholesterol levels

Important considerations

  • May increase the risk of serious infections
  • Blood tests are typically performed to monitor blood counts, cholesterol, and liver function
  • Patients should discuss any history of blood clots, cardiovascular disease, or cancer with their healthcare provider

Baricitinib (Olumiant)

Baricitinib is a JAK inhibitor that targets specific proteins involved in inflammatory signaling. Early studies and case reports have shown potential benefits in some patients with difficult-to-treat dermatomyositis, including improvements in skin manifestations and muscle weakness.

How it is administered

  • Taken orally as a once-daily tablet

Common side effects

  • Upper respiratory infections
  • Nausea
  • Headache
  • Elevated cholesterol levels

Important considerations

  • Requires monitoring of blood counts and laboratory values
  • May increase the risk of serious infections and blood clots
  • Patients should promptly report unusual swelling, chest pain, or shortness of breath

Ruxolitinib (Jakafi)

Ruxolitinib is a JAK inhibitor that has been used in selected patients with refractory dermatomyositis and interferon-driven disease. Emerging evidence suggests it may help reduce inflammation and improve skin symptoms in certain patients, although experience remains limited and it is not approved specifically for myositis.

How it is administered

  • Taken orally as a tablet

Common side effects

  • Anemia
  • Low platelet counts
  • Dizziness
  • Headache
  • Increased risk of infection

Important considerations

  • Routine blood tests are recommended to monitor blood counts
  • Patients should discuss any signs of infection with their healthcare provider
  • Dose adjustments may be necessary based on laboratory results

Brepocitinib (Lisraya)

Brepocitinib is a targeted therapy that inhibits both TYK2 and JAK1 signaling pathways, which play important roles in inflammation. In August 2026, brepocitinib became the first oral therapy approved by the US Food and Drug Administration for the treatment of adults with dermatomyositis. Clinical studies demonstrated improvements in overall disease activity, physical function, and skin disease severity.

How it is administered

  • Taken orally as a once-daily tablet

Common side effects

  • Upper respiratory tract infection
  • Headache
  • Fatigue
  • Urinary tract infection
  • Nausea

Important considerations

  • Carries warnings similar to other JAK inhibitors regarding serious infections
  • Regular monitoring of blood counts and other laboratory parameters may be recommended

Patients should discuss vaccination status and infection risk with their healthcare provider before starting treatment

Disclaimer: Information provided on this website is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always consult a qualified healthcare provider with any questions you may have regarding a medical condition.