AstraZeneca has announced its drug Saphnelo (anifrolumab-fnia)—a first-in-class type I interferon receptor antibody—has landed approval by the US Food and Drug Administration (FDA). According to the pharmaceutical firm, the treatment is the first novel treatment for systemic lupus erythematosus (SLE) to receive the go-ahead from the agency in more than 10 years.
The FDA reportedly based its approval on the efficacy and safety data from the Saphnelo clinical development program, including two TULIP Phase III trials and the MUSE Phase II trial. In the trials, results indicate more patients treated with Saphnelo experienced a reduction in overall disease activity across organ systems, including skin and joints, and achieved sustained reduction in oral corticosteroid (OCS) use compared to placebo, with both groups receiving standard therapy.
The approval is the first for a type I interferon (type I IFN) receptor antagonist. Type I IFN plays a central role in the pathophysiology of lupus and increased type I IFN signaling is associated with increased disease activity and severity.
Richard Furie (chief of the Division of Rheumatology at Northwell Health and a principal investigator in the Saphnelo clinical development program) said the approval offers a ray of hope for lupus patients worldwide.
“Our treatment goals in systemic lupus erythematosus are to reduce disease activity, prevent organ damage from either the illness itself or the medications, especially steroids, and improve one’s quality of life,” Furie commented. “Today’s approval of anifrolumab represents a big step forward for the entire lupus community; physicians will now be able to offer an effective new treatment that has produced significant improvements in overall disease activity while reducing corticosteroid use.”
Stevan Gibson, president and CEO of the Lupus Foundation of America, said the approval is good news for lupus patients.
"After having only one therapy approved for lupus during the past 60 years, it is a cause for celebration to have two new treatment options approved in 2021 alone for this life-threatening autoimmune disease that affects an estimated 1.5m Americans," Gibson said. “The pipeline of potential new treatments for lupus remains vibrant, and the Lupus Foundation of America continues its work to bring down barriers to lupus drug development and ensure that people with lupus will have access to the medications they need to improve their quality of life.”
Mene Pangalos, AstraZeneca’s executive vice president of biopharmaceuticals R&D, said, “Today’s landmark approval of Saphnelo is the culmination of years of AstraZeneca’s pioneering research in the type I interferon pathway, a central driver in systemic lupus erythematosus pathophysiology. This ground-breaking medicine has the potential to meaningfully improve the lives of patients living with this often debilitating disease.”
SLE is the most common form of lupus, impacting up to 300,000 in the US alone, and it disproportionately affects minority populations, including African-American, Hispanic, and Asian people. The complex autoimmune condition stands to impact nearly any organ, and patients commonly experience debilitating symptoms, long-term organ damage, and poor health-related quality of life.
Saphnelo currently is under regulatory review for the treatment of SLE in the EU and Japan. The Phase III trial in SLE using subcutaneous delivery has been initiated and additional Phase III trials are planned for lupus nephritis, cutaneous lupus erythematosus, and myositis.