Actemra is FDA approved by the FDA for slowing the rate of decline in pulmonary function in adults with systemic sclerosis-associated interstitial lung disease.

Genentech/Roche announced that the FDA approved Actemra (tocilizumab) subcutaneous injection for slowing the rate of decline in pulmonary function in adult patients with systemic sclerosis-associated interstitial lung disease (SSc-ILD), a debilitating condition with limited treatment options. Actemra is the first biologic therapy approved by the FDA for the treatment of the disease. Systemic sclerosis (SSc), also known as scleroderma, is an often devastating autoimmune disease that worsens over time and has no cure. It occurs when the immune system malfunctions causing tissues of the skin and lungs to thicken and harden. SSc impacts up to 75,000 people in the United States. Interstitial lung disease (ILD), which may occur in approximately 80% of SSc patients, causes inflammation and scarring of the lungs and can be life-threatening. The FDA approval is based on data from the focuSSced trial, a Phase III randomized, double-blind, placebo-controlled clinical trial of 212 adults with systemic sclerosis. Supportive information was also used from the faSScinate trial, a Phase II/III, randomized, double-blind, placebo-controlled study in patients with SSc . The trials did not meet their primary endpoint of change from baseline to week 48 in the modified Rodnan Skin Score (mRSS), which is a standard outcome measure for skin fibrosis (the scarring or hardening of the skin) in SSc. However, in the overall population of the focuSSced study, patients treated with Actemra, as compared to placebo-treated patients, 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 (ppFVC), which compares the observed FVC to that expected for a healthy person of the same age, gender, race and height. FVC results were similar in the faSScinate study . Of the 212 patients who were randomized into the focuSSced study 68 patients (65%) in the Actemra arm and 68 patients (64%) in the placebo arm had SSc-ILD at baseline, as confirmed by a visual read of high resolution computed tomograph (HRCT) by blinded thoracic radiologists. Post-hoc exploratory analyses were performed to evaluate the results within the subgroups of patients with and without SSc-ILD. The ppFVC and FVC results in the overall population were primarily driven by results in the SSc-ILD subgroup. In that subgroup, patients in the Actemra group had a smaller decline in mean ppFVC than patients on placebo (0.07% vs. -6.4%, mean difference 6.47%), and a smaller decline in FVC compared to placebo (mean change -14 mL vs. -255 mL, mean difference 241 mL). The mean change from baseline to week 48 in mRSS in patients receiving Actemra compared to placebo was -5.88 vs. -3.77, mean difference -2.11. The safety profile for Actemra through week 48 was comparable for SSc-ILD and SSc patients overall in the focuSSced study, and was consistent with the known safety profile of Actemra in both focuSSced and faSScinate studies. The most common adverse events in patients treated with Actemra were infections.