Rheumatoid Arthritis Drug Awarded Breakthrough Status for Systemic Sclerosis

The FDA has granted Breakthrough Therapy Designation to Roche’s tocilizumab (Actemra/RoActemra) for the treatment of systemic sclerosis (SSc), also known as scleroderma.

Tocilizumab is currently approved to treat moderate-to-severe rheumatoid arthritis (RA), as well as polyarticular juvenile idiopathic arthritis (PJIA) and systemic juvenile idiopathic arthritis (SJIA).

The interleukin-6 receptor antagonist’s breakthrough status for SSc was based on the phase 2 faSScinate study that showed continued improvement in skin thickening with active treatment. To further examine the drug in SSc, Roche has launched a global phase 3 clinical trial.

“Close to 500,000 people worldwide have benefited from treatment with Actemra/RoActemra since initial approval over a decade ago,” stated Sandra Horning, MD, Roche’s head of global product development and chief medical officer. “…These new data further demonstrate the efficacy and safety of Actemra/RoActemra in multiple diseases.”

SSc is a rare, chronic disorder that causes the skin and joints to harden and scar, due to accumulations of collagen and blood vessel abnormalities. Once it reaches the internal organs, SSc can be life threatening. In fact, it has the highest mortality rate of any rheumatic disease.

Approximately 2.5 million individuals worldwide are believed to have SSc. Currently, there are no FDA-approved disease-modifying therapeutic options to reverse or stop its effects.

Common side effects of tocilizumab include upper respiratory tract infections, headache, hypertension, and injection site reactions. More serious side effects include stomach tears, low white blood cell and platelet counts, and hepatitis B infection.