argenx Announces the UK MHRA has Granted Early Access to Efgartigimod for Generalized Myasthenia Gravis

• Positive scientific opinion under Early Access to Medicines Scheme (EAMS) will make efgartigimod available to eligible generalized myasthenia gravis (gMG) patients in the UK prior to marketing authorization
• Efgartigimod was granted a Promising Innovative Medicine (PIM) designation by UK’s Medicines and Healthcare products Regulatory Agency (MHRA) in November 2021

Breda, the Netherlands—May 31, 2022—argenx SE (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, today announced that efgartigimod, the Company’s neonatal Fc receptor (FcRn) blocker has been awarded a positive scientific opinion by the MHRA under the EAMS. Within the EAMS, efgartigimod is indicated for the treatment of adult patients with anti-acetylcholine receptor (AChR) antibody seropositive gMG, including patients with refractory gMG who have failed, not tolerated or are ineligible for licensed treatment.

The EAMS program offers early access to innovative medicines for patients who are experiencing a significant unmet medical need, before the MHRA approves a formal marketing authorization application (MAA) and prior to reimbursement. The positive scientific opinion enables healthcare providers to decide whether to prescribe a treatment before licensing is approved, granting eligible gMG patients in the UK the potential for early, pre-license access to efgartigimod while relevant regulatory bodies complete the review of the MAA.

“Despite currently available treatments, gMG patients in the UK face a significant disease burden and many struggle to manage the debilitating symptoms of this rare autoimmune disease. We are committed to addressing this unmet need, and are thrilled that UK healthcare professionals now have a potential new option for the treatment of their eligible gMG patients,” said David Knechtel, UK Country Manager at argenx. “The MHRA’s positive scientific opinion supports our belief in the value efgartigimod can offer to people living with gMG, and marks another advancement toward our goal of serving patients around the world. We look forward to continued collaboration with the agency to make this innovative therapy available to UK patients as soon as possible.”

The European Medicines Agency (EMA) is currently reviewing the MAA for efgartigimod for the treatment of gMG, with a decision expected in the second half of 2022, followed by an anticipated decision from the MHRA on a UK marketing authorization. Efgartigimod was previously granted a PIM designation by the MHRA in November 2021.

About Generalized Myasthenia Gravis

Generalized myasthenia gravis (gMG) is a rare and chronic autoimmune disease, impacting up to 15,000 UK patients, where immunoglobulin G (IgG) autoantibodies disrupt communication between nerves and muscles, causing debilitating and potentially life-threatening muscle weakness. Approximately 85% of people with MG progress to gMG within 24 months1, where muscles throughout the body may be affected. Patients with confirmed AChR antibodies account for approximately 85% of the total gMG population1.

About Efgartigimod

Efgartigimod is an antibody fragment designed to reduce pathogenic immunoglobulin G (IgG) antibodies by binding to the neonatal Fc receptor and blocking the IgG recycling process. Efgartigimod is being investigated in several autoimmune diseases known to be mediated by disease-causing IgG antibodies, including neuromuscular disorders, blood disorders, and skin blistering diseases. It is currently approved in the United States for the treatment of adult patients with gMG who are anti-acetylcholine receptor antibody positive, and Japan for adult patients with gMG who do not have sufficient response to steroids or non-steroidal immunosuppressive therapies.

About argenx

argenx is a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases. Partnering with leading academic researchers through its Immunology Innovation Program (IIP), argenx aims to translate immunology breakthroughs into a world-class portfolio of novel antibody-based medicines. argenx developed and is commercializing the first-and-only approved neonatal Fc receptor (FcRn) blocker in the U.S. and Japan.

References
1. Behin et al. New Pathways and Therapeutics Targets in Autoimmune Myasthenia Gravis. J Neuromusc Dis 5. 2018. 265-277