Roche’s Actemra/RoActemra Becomes First FDA-Approved Biologic for Systemic Sclerosis-Associated Interstitial Lung Disease (SSc-ILD)

March 08, 2021 News /Bio ValleyBIOON/ -- Roche recently announced that the U.S. Food and Drug Administration (FDA) has approved the subcutaneous injection of the IL-6 receptor inhibitor Actemra/RoActemra (Chinese brand name: Yameiluo®; generic name: tocilizumab) to slow the rate of decline in lung function in adult patients with systemic sclerosis-associated interstitial lung disease (SSc-ILD). SSc-ILD is a debilitating condition with very limited treatment options. Notably,Actemra/RoActemra is the first biologic therapy approved by the U.S. FDA for the treatment of SSc-ILDSSc-ILD is also the sixth FDA-approved indication for Actemra/RoActemra. Previously,FDAPriority review has been granted for this drug in the treatment of SSc-ILD.

Systemic sclerosis (SSc), also known as scleroderma, is a devastatingAutoimmunitySystemic sclerosis is a progressive, incurable disease that worsens over time. It occurs when immune system dysfunction leads to thickening and hardening of the skin and lung tissues. Approximately 2.5 million people worldwide are affected by systemic sclerosis (SSc). Interstitial lung disease (ILD) may develop in approximately 80% of patients with SSc, causing pulmonary inflammation and fibrosis, and can be life-threatening.。

In September 2019, Boehringer Ingelheim’s oral small-molecule tyrosine kinase inhibitor Ofev (nintedanib) capsules received U.S. FDA approval for slowing the rate of decline in lung function in adult patients with systemic sclerosis-associated interstitial lung disease (SSc-ILD). This approval makesOfev became the first drug approvedFDADrug Approved for the Treatment of This Rare Lung Disease.

Actemra/RoActemra is the first humanized monoclonal antibody targeting the interleukin-6 (IL-6) receptor antagonist, which can be administered via two routes: intravenous infusion (IV) and subcutaneous injection (SC). It has been approved for multiple indications, including: rheumatoidRheumatoid Arthritis(RA), polyarticular juvenile idiopathic arthritis (pJIA), systemic juvenile idiopathic arthritis (sJIA), giant cell arteritis (GCA), cytokine release syndrome (CRS), Castleman disease, and Takayasu arteritis. Actemra/RoActemra includes intravenous (IV) formulations and subcutaneous (SC) pre-filled syringe (PFS) formulations, with specific indications varying across different countries and regions. To date, Actemra/RoActemra has been approved in more than 110 countries worldwide.

Dr. Levi Garraway, Chief Medical Officer and Global Head of Product Development at Roche, stated: “We are honored to provide the first FDA-approved biologic therapy for patients with systemic sclerosis-associated interstitial lung disease. We, together with”FDA“Working closely together to evaluate the impact of Actemra/RoActemra on lung function in this disease. This landmark approval provides a much-needed new treatment option for patients suffering from this rare and debilitating condition.”

SSc-ILD (Image source: europeanpharmaceuticalreview.com)

This approval is based on data from the focuSSced trial, a randomized, double-blind, placebo-controlled Phase 3Clinical Trial, 212 adult patients with systemic sclerosis (SSc) were enrolled. Supportive information also comes from the faSScinate trial, a randomized, double-blind, placebo-controlled Phase II/III trial in patients with SSc. The focuSSced trial did not meet its primary endpoint of change from baseline to Week 48 in the modified Rodnan Skin Score (mRSS). The mRSS is a standard outcome measure for skin fibrosis (skin scarring or hardening) in SSc. The faSScinate trial also did not show statistical significance on the mRSS primary endpoint.

However, in the overall population of the focuSSced trial, the Actemra/RoActemra treatment group exhibited a smaller decline from baseline to Week 48 in forced vital capacity (FVC) and percent predicted forced vital capacity (ppFVC) compared with the placebo group. FVC is a commonly used measure of lung function, defined as the volume of air that can be forcibly exhaled at maximum speed following a maximal inhalation. The ppFVC compares the observed FVC with the expected FVC for healthy individuals of the same age, sex, race, and height. Similar FVC results were observed in the faSScinate trial.

Among the 212 patients randomized into the focuSSced study, 68 patients (65%) in the Actemra/RoActemra group and 68 patients (64%) in the placebo group had SSc-ILD at baseline, as confirmed by visual assessment of high-resolution computed tomography (HRCT) scans by blinded chest radiologists. Post hoc exploratory analyses were conducted to evaluate outcomes in patient subgroups with and without SSc-ILD. The ppFVC and FVC results in the overall population were primarily driven by the outcomes in the SSc-ILD subgroup. In this subgroup, patients in the Actemra/RoActemra group exhibited a smaller mean decline in ppFVC compared with the placebo group (0.07% vs. -6.4%; mean difference: 6.47%) and a smaller decline in FVC (mean change: -14 mL vs. -255 mL; mean difference: 241 mL). Compared with the placebo group, patients in the Actemra/RoActemra group showed a greater mean change from baseline to Week 48 in the modified Rodnan skin score (mRSS) (-5.88 vs. -3.77; mean difference: -2.11).

In the focuSSced study, the safety profile of Actemra/RoActemra after 48 weeks of treatment was generally comparable between patients with SSc-ILD and those with SSc. In both the focuSSced and faSScinate studies, the safety profile of Actemra/RoActemra was consistent with its known safety profile. The most common adverse events in patients treated with Actemra/RoActemra were infections.(Bioon.com)

Original Source: Roche’s Actemra/RoActemra becomes the first biologic therapyapproved by the FDA for slowing the rate of decline in pulmonary function in adults with systemic sclerosis-associated interstitial lung disease, a rare, debilitating condition