FDA Approves First Biologic for Rare, Debilitating Lung Disease

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The U.S. Food and Drug Administration (FDA) approved Genentech’s Actemra (tocilizumab) for slowing the rate of decline in pulmonary function in adults with systemic sclerosis-associated interstitial lung disease (SSc-ILD). It is the first biologic approved by the FDA for this indication.

Systemic sclerosis (SSc) is also called scleroderma. It is an autoimmune disease that progressively worsens. There is no cure. It is caused by the immune system malfunctioning, causing skin and lung tissues to thicken and harden. It affects up to 75,000 people in the U.S. Interstitial lung disease (ILD) occurs in about 80% of SSc patients, causing inflammation and scarring of the lungs. It can be life-threatening.

“We are honored to offer the very first FDA-approved biologic treatment option to people living with systemic sclerosis-associated interstitial lung disease,” said Levi Garraway, chief medical officer and head of Global Product Development for Genentech. “We worked closely with the FDA to evaluate Actemra’s impact on lung function in this setting. This milestone approval provides a much-needed new treatment option for people living with this rare, debilitating disease.”

The drug was recently reported to decrease deaths and the need for mechanical ventilation in severe COVID-19 patients. Typically approved for rheumatoid arthritis and cytokine release syndrome associated with CAR-T therapy, the University of Oxford evaluated it in COVID-19 in its RECOVERY clinical trial.

In the study, about half of the 4,116 adults in the trial were given Actemra with standard of care, while the other half received standard of care alone. Mortality in the Actemra group was 39% compared to 33% in the standard of care only group. Mortality decreased from 22% to 19% in patients not requiring ventilation and from 48% to 47% in those who did require mechanical ventilation. The biggest benefit was in patients who received corticosteroids as part of standard of care, primarily dexamethasone.

The FDA approval for SSc-ILD was based on data from the focuSSced Phase III trial of 212 adults with systemic sclerosis. Additional data from the fascinate Phase II/III trial was also included. The focuSSced trial did not meet the primary endpoint of change from baseline to week 48 in the modified Rodnan Skin Score (mRSS), a standard outcome measure for skin fibrosis (scarring) in SSc, nor was there a statistically significant effect on the primary endpoint of mRSS in the faSSscinate trial.

But, in the overall patient population of the focuSSced trial, patients receiving Actemra compared to the placebo cohort, had less decline from baseline to week 48 in observed forced vital capacity (FVC), a measure of lung function. FVC results were similar in the faSScinate study.

The drug had previously received Priority Review from the FDA for this indication. It is the sixth FDA approved indication for Actemra since its U.S. launch in 2010.

Actemra was the first humanized interleukin-6 (IL-6) receptor antagonist approved for moderately to severely active rheumatoid arthritis. The drug is also approved for adults with giant cell arteritis (GCA), for patients two years and older with active polyarticular juvenile idiopathic arthritis (PJIA) or active systemic juvenile idiopathic arthritis (SJIA), and now for SSc-ILD.

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