Roche biologic gets first FDA nod for rare lung disease

March 5, 2021

The U.S. FDA has approved Roche's Actemra/RoActemra for the treatment of systemic sclerosis making the drug the first biologic therapy approved by the agency for the treatment of the rare debilitating autoimmune disease.

Actemra is a subcutaneous injection for slowing the rate of decline in pulmonary function in adult patients with systemic sclerosis-associated interstitial lung disease (SSc-ILD).

The FDA approval is based on data from the focuSSced trial, a phase 3 randomized, double-blind, placebo-controlled clinical trial of 212 adults with systemic sclerosis. Supportive information was also used from the faSScinate trial, a phase 2/3, randomized, double-blind, placebo-controlled study in patients with SSc.

However, the trials failed to meet primary endpoints based on skin fibrosis (the scarring or hardening of the skin). But in the overall population of the focuSSced study, patients treated with Actemra were observed to have less decline from baseline to week 48 in observed forced vital capacity (FVC), a common measure of lung function that assesses how much air can be exhaled, and percent predicted forced vital capacity.

Actemra was previously granted Priority Review for SSc-ILD by the FDA. This is the sixth FDA approved indication for the drug since it was launched in the U.S. in 2010 as treatment for rheumatoid arthritis.

Actemra has also been explored with varying success as a treatment for patients hospitalized with severe COVID-19. Recently, results from the UK RECOVERY trial showed benefit of the drug, together with the steroid dexamethasone, in improving survival, shortening hospital stay, and reducing the need for mechanical ventilators.

Read the press release