Rare Disease Treatment Approved: FDA Clears Argenx’s Vyvgart Hytrulo for CIDP

Rare disease chronic inflammatory demyelinating polyneuropathy (CIDP) is the focus of Netherlands biotech company Argenx’s second the Food and Drug Administration (FDA) approval for Vyvgart Hytrulo, an FcRn inhibitor. This approval represents a significant milestone for a drug that Argenx hopes could be beneficial for more than a dozen diseases.

Vyvgart Hytrulo (efgartigimod alfa/hyaluronidase) is the first drug in the FcRn blocker class approved for CIDP, a rare autoimmune disease affecting the peripheral nervous system. CIDP causes symptoms like fatigue, muscle weakness, and loss of sensation, which can lead to severe disability and necessitate the use of a wheelchair. Argenx highlighted that this subcutaneously administered drug is the first “meaningful” new treatment for the approximately 24,000 people in the US suffering from CIDP in over three decades.

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Rare Disease CIDP: Current Treatment Options and FDA Approval Updates

Current treatments for CIDP include corticosteroids, high-dose intravenous immune globulins (such as Takeda’s HyQvia, approved by the FDA for CIDP in January), and plasmapheresis. Plasmapheresis involves removing a patient’s blood, filtering out plasma containing auto-antibodies that cause the disease, and returning the whole cells to the patient.

The FDA’s approval of Vyvgart Hytrulo for CIDP was based on the ADHERE trial, which demonstrated a 61% lower risk of relapse compared to placebo. Additionally, 69% of patients in the trial responded positively to Argenx’s drug. This builds on the success of its intravenously dosed counterpart, Vyvgart (efgartigimod alfa), which was the first FcRn blocker approved by the FDA in December 2021 for generalized myasthenia gravis (gMG).

Vyvgart Hytrulo was also approved for gMG in the US a year ago. Argenx used a priority review voucher (PRV) to expedite the CIDP approval process, underscoring the significance of this new indication.

Rare Disease CIDP: Vyvgart Hytrulo’s Expansion Challenges and Future Potential

Despite setbacks in extending Vyvgart Hytrulo to other diseases like pemphigus vulgaris/pemphigus foliaceus and primary immune thrombocytopenia (ITP), the drug was recently approved in Japan for ITP. Overall, Vyvgart remains in development for about 15 indications and is anticipated by analysts to achieve multibillion-dollar sales at its peak, alongside FcRn rival nipocalimab from Johnson & Johnson.

Last year, the Vyvgart franchise generated $1.2 billion in sales. Future growth is expected to be driven primarily by the subcutaneous version of the drug, which offers the convenience of a once-weekly injection administered by a healthcare professional, taking only 30 to 90 seconds, compared to the weekly, hour-long IV infusion required with Vyvgart.

The introduction of Vyvgart Hytrulo provides an essential new treatment option for patients with CIDP, offering a more convenient and potentially more effective alternative to existing therapies. This approval not only enhances Argenx’s portfolio but also brings hope to thousands of patients living with this debilitating condition. The success of Vyvgart Hytrulo in clinical trials highlights the potential of FcRn inhibitors in treating autoimmune diseases and sets the stage for further research and development in this promising therapeutic area.

Argenx’s commitment to expanding the indications for Vyvgart Hytrulo demonstrates the company’s dedication to addressing unmet medical needs and improving patient outcomes. With ongoing clinical trials and future regulatory submissions, Vyvgart Hytrulo is poised to become a cornerstone treatment for a variety of autoimmune diseases, offering new hope to patients and healthcare providers alike.