Lupus Research Alliance Applauds U.S. FDA Approval of AstraZeneca's anifrolumab-fnia (Saphnelo™) for Systemic Lupus Erythematosus (SLE)

First in New Class of Drugs in More than a Decade for SLE and Targeting Type 1 Interferons

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Aug 02, 2021, 07:12 ET

NEW YORK, Aug. 2, 2021 /PRNewswire/ -- The Lupus Research Alliance (LRA) is particularly excited about the U.S. Food and Drug Administration (FDA) approval of anifrolumab-fnia (Saphnelo™), a first in class, type I interferon receptor antagonist, indicated for adults with moderate to severe systemic lupus erythematosus (SLE). The approval represents the first new treatment for generalized SLE in more than a decade and is the result of significant seminal research funded originally by the Lupus Research Alliance.

An autoimmune disease, SLE is the most common form of lupus, in which the immune system creates antibodies that can attack any part of the body. Affecting multiple organs, SLE is associated with major clinical burden for the patient and organ damage. It affects a diverse patient population and is more prevalent in Black and Hispanic ethnicities. Heterogeneity in SLE is evident as the condition presents differently across diverse populations, and symptoms vary from patient to patient.

Given by intravenous infusion, anifrolumab-fnia works by blocking immune system molecules called type I interferons that are one of the main factors that leads to tissue damage and disease symptoms. Type I interferons are a family of 17 similar molecules that help protect us from infections.ⁱ Research has shown that the vast majority -- 60-80 percent of adultsⁱⁱ -- have high levels of type I interferons.

The Lupus Research Alliance has invested $16 million in 40 studies on type I interferons, including pioneering work identifying a unique combination of genes that had been switched on by type I interferons in lupus patients; these results galvanized research into the molecules' role in the disease. AstraZeneca plc led its further development.

One of the trailblazers in interferon research is former chair of the Lupus Research Alliance Scientific Advisory Board Dr. Mary Crow. "This approval represents a critical milestone for lupus research and an exciting new therapeutic option for patients with lupus," noted Dr. Crow. "It is personally gratifying to have discovered the activation of the type I interferon pathway as a significant mechanism in lupus disease and to see the fruition of interferon research and the related drug development program."

The safety and effectiveness of anifrolumab-fnia was demonstrated by two large Phase 3 clinical trials, TULIP-1ⁱⁱⁱ and TULIP-2^iv showing that it reduced flares in people with moderate to severe lupus while allowing lowered steroid use. Further analysis of these two studies also found that the drug produced long-term reductions in the severity of skin rash and arthritis symptoms. TULIP-1 and TULIP-2 were led by top lupus researchers, many of whom are leaders in the Lupus Clinical Investigators Network (LuCIN) managed by the Lupus Research Alliance affiliate Lupus Therapeutics including TULIP-1 principal investigator Dr. Richard Furie along with TULIP-2 lead investigator Professor Eric Morand of Monash University in Australia, also an LRA Distinguished Innovator Award recipient.

Northwell Health Chief of Rheumatology Dr. Furie explained, "The lupus community has long been confident in the successful development of anifrolumab-fnia; most recently the clinical benefit was strongly observed in the phase 3 TULIP trials, and we confirmed a hypothesis dating back many years that interferons play an important role in SLE. The FDA approval of anifrolumab-fnia, coming 10 years after the FDA approval of belimumab, represents another milestone in our quest to make more therapies available and improve the lives of our patients with SLE."

"The role of interferon in lupus has been supported by the science for years but has long awaited this formal proof of benefit in a clinical trial," noted Professor Morand. "The FDA approval of anifrolumab-fnia potentially allows thousands of lupus patients to benefit. Decreased disease activity along with reduced steroid use form the pillars of treatment goals in lupus, and anifrolumab-fnia has shown these effects. As an LRA investigator, I am extremely proud to have been involved in this landmark journey to improve the lives of people with lupus."

Lupus Research Alliance President and CEO Kenneth M. Farber said, "The approval of anifrolumab-fnia is especially gratifying to us. Over the past two decades, the Lupus Research Alliance has made significant investments in research focused on the role of type I interferons in lupus. Particularly influential work we funded was conducted by renowned scientific leaders, and it is particularly exciting to see that work realized with an urgently needed new treatment option for people with lupus."

"This approval exemplifies the importance of foundational research which seeds and informs the drug development pipeline" said Rachele Berria, MD, PhD, Vice President, US Medical, Biopharmaceuticals at AstraZeneca. "Numerous early studies supported by the Lupus Research Alliance provided robust evidence on the central role the type I interferon pathway has in SLE pathophysiology. This research has helped in paving the way for the development of Saphnelo, a much-needed new treatment for people living with lupus."

Click here for the announcement from AstraZeneca plc.

About Lupus
Lupus is a chronic, complex autoimmune disease that affects millions of people worldwide. More than 90 percent of people with lupus are women; lupus most often strikes during the childbearing years of 15-45. African Americans, Latinx, Asians and Native Americans are two to three times at greater risk than Caucasians. In lupus, the immune system, which is designed to protect against infection, creates antibodies that can attack any part of the body impacting a wide range of tissues and organs including kidneys, brain, heart, lungs, blood, skin, and joints. Its treatment can also have serious side effects; one of the common medications for lupus, oral corticosteroids, for example, can lead to organ damage, infections, heart disease, osteoporosis weakening bones and cataracts affecting vision.^v,vi

About the Lupus Research Alliance
The Lupus Research Alliance is the largest non-governmental, non-profit funder of lupus research worldwide. The organization aims to transform treatment by funding the most innovative lupus research, fostering diverse scientific talent, and driving discovery toward better diagnostics, improved treatments and ultimately a cure for lupus. Because the Lupus Research Alliance's Board of Directors fund all administrative and fundraising costs, 100% of all donations goes to support lupus research programs.

ⁱ McNab, F., Mayer-Barber, K., Sher, A. et al. Type I interferons in infectious disease. Nat Rev Immunol 15, 87–103 (2015)
ⁱⁱ Becker AM. SLE peripheral blood B cell, T cell and myeloid cell transcriptomes display unique profiles and each subset contributes to the interferon signature. PLoS One 2013 Jun 24;8(6):e67003.
ⁱⁱⁱ Furie RA, et al. Type I interferon inhibitor anifrolumab in active systemic lupus erythematosus (TULIP-1): a randomised, controlled, phase 3 trial. The Lancet Rheumatology. Vol. 1, Issue 4, E208-E219, December 1, 2019.
^iv Morand EF, et al. Trial of Anifrolumab in Active Systemic Lupus Erythematosus.
^v Ruiz-Arruza I, Ugarteet A, Cabezas-Rodriguez I, et al. Glucocorticoids and irreversible damage in patients with systemic lupus erythematosus. Rheumatology (Oxford). 2014;53(8):1470-1476.
^vi Al Sawah S, Zhang X, Zhu B, et al. Effect of corticosteroid use by dose on the risk of developing organ damage over time in systemic lupus erythematosus—the Hopkins Lupus Cohort. Lupus Sci Med. 2015;2(1):e000066.