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FDA Grants Breakthrough Therapy Designation to Actemra for Systemic Sclerosis

US health regulators have granted Breakthrough Therapy designation to Genentech’s Actemra (tocilizumab) for systemic sclerosis.

Genentech, a member of the Roche Group, said that the US Food and Drug Administration (FDA) has granted Breakthrough status to Actemra for systemic sclerosis, also known as scleroderma, a rare, chronic disorder characterized by blood vessel abnormalities, as well as degenerative changes and scarring in the skin, joints, and internal organs.

“The development of Actemra in systemic sclerosis represents our commitment to severe rheumatic diseases,” said Sandra Horning, MD, chief medical officer and head of Global Product Development. “This Breakthrough Therapy Designation underscores the unmet need in systemic sclerosis and the potential of Actemra to help patients with this debilitating autoimmune disorder.”

Breakthrough status is intended to expedite the development and review of drugs that demonstrate potential clinical benefit in serious diseases, in order for patients to gain access to them as soon as possible. Genentech said that it has also started a late-stage study in systemic sclerosis.

Systemic sclerosis affects approximately 75,000 to 100,000 people in the US. More than 75 percent of systemic sclerosis patients are women, primarily aged 30 to 50. It has the highest mortality of any autoimmune rheumatic disease. Currently, there are no drugs approved by the FDA for treatment of systemic sclerosis. While there are treatments for some aspects of this disease, none are used to stop or reverse the key symptom of skin thickening and hardening.

The Breakthrough designation for Actemra was granted based on results from a mid-stage study, known as fascinate. While the drug failed to meet the primary endpoint of improvement in skin thickening at 24 weeks, a meaningful trend was observed in the Phase II study. In this second part of the study, there was continued improvement in skin thickening between weeks 24 and 48.

Additionally, Roche said that new data from the U-ACT-EARLY and TENDER studies in patients with early rheumatoid arthritis (RA) and systemic juvenile idiopathic arthritis (sJIA), respectively, as well as results from the fascinate study will be presented at the European League Against Rheumatism (EULAR) meeting.

Actemra is already approved for treatment of adult patients with moderately to severely active RA who have used one or more disease-modifying antirheumatic drugs (DMARDs) that did not provide enough relief. Additionally, the drug is used as an IV formulation for patients with active polyarticular juvenile idiopathic arthritis (PJIA) or sJIA two years of age and older.

“Close to 500,000 people worldwide have benefited from treatment with Actemra/RoActemra since initial approval over a decade ago. The breadth of our study results at EULAR, ranging from arthritis in adults and children to a rare inflammatory disorder, underscores our commitment to helping people with debilitating autoimmune diseases,” said Sandra Horning, MD, Roche’s Head of Global Product Development and Chief Medical Officer. “These new further demonstrate the efficacy and safety of Actemra/RoActemra in multiple diseases, including use as a single therapy in early RA.”

Sources: Genentech; Roche

Last updated: 6/10/15; 5:20pm EST

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