UK’s MHRA grants Promising Innovative Medicine designation to argenx’ investigational therapy, subcutaneous efgartigimod alfa for the treatment of chronic inflammatory demyelinating polyneuropathy (CIDP)
November 28, 2024 – Biotechnology, Drug Discovery, Other, Pharmaceutical – CIDP, MHRA, Promising Innovative Medicine, argenx, autoimmune diseases
28 November 2024 — Gerrards Cross, UK — argenx, a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, confirmed today that its investigational therapy, subcutaneous efgartigimod alfa has been granted Promising Innovative Medicine (PIM) designation by the UK Medicines and Healthcare products Regulatory Agency (MHRA) for the treatment of adults with chronic inflammatory demyelinating polyneuropathy (CIDP). PIM designation is an early indication that a medicinal product is a candidate for the UK’s Early Access to Medicines Scheme (EAMS).
David Knechtel, General Manager of argenx UK & Ireland commented: “We welcome the MHRA’s recognition of efgartigimod alfa’s potential to address the significant unmet need in CIDP, a disease in which patients endure life-altering symptoms but have few options to treat the underlying cause.”
CIDP is an autoimmune disease of the peripheral nervous system that can lead to severe disability from muscle weakness and sensory disturbances. It can affect any age group, and the onset of the disorder may begin during any decade of life. CIDP affects males twice as often as females and the average age of onset is 50. Around 650 people are diagnosed with CIDP each year in the UK.
Between 20 and 30% of patients with CIDP do not adequately respond to any currently available treatments and many patients with a partial response experience remaining neurological impairment, highlighting the need for effective alternatives. It is believed CIDP is caused by an autoimmune response, which may include involvement of IgG antibodies. Efgartigimod alfa is a human antibody fragment, specifically designed to selectively attach to, and block, a receptor, called FcRn, present within certain cells that line blood vessels. Blocking this receptor results in the reduction of circulating IgG antibodies, including harmful ‘autoantibodies’ that attack healthy nerve cells and cause CIDP symptoms.
Efgartigimod alfa is currently licensed in the UK to treat eligible patients with a different autoimmune disease, generalised myasthenia gravis (gMG). This is the second PIM designation for efgartigimod, which wasgranted a PIM designation for gMG. In this indication it is branded as VYVGART®▼(efgartigimod alfa). Its use in CIDP remains investigational.
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) fragment in the U.S., the EU and Japan. Visit: argenx.com.
