Subjects ” Neuromuscular Disorders
The Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) for efgartigimod (ARG-113; Argenx) for the treatment of generalized myasthenia gravis for review.
Efgartigimod is an Fc receptor antagonist designed to lower immunoglobulin G (IgG) antibody levels and block the IgG recycling process. Use is supported by data from the multicentre, randomized, double-blind, phase 3 ADAPT study (ClinicalTrials.gov: NCT03669588), which compared the efficacy and safety of efgartigimod with placebo given as an intravenous infusion in 167 adults with generalized Myasthenia gravis was administered. The primary endpoint was the percentage of responders to the MG-ADL (Myasthenia Gravis Activities of Daily Living) score in patients with acetylcholine receptor antibody-positive (AChR-Ab +) generalized myasthenia gravis. It was defined that the responders had at least a 2-point improvement in the MG-ADL score for at least 4 consecutive weeks.
The study results showed that a significantly higher proportion of efgartigimod-treated AChR-Ab + patients met the primary endpoint compared to placebo (67.7% versus 29.7%; P <0.0001). In addition, a greater proportion of efgartigimod-treated AChR-Ab + patients achieved minimal symptom expression, defined as an MG-ADL score of 0 (symptom-free) or 1, and responded to the quantitative myasthenia gravis score compared to placebo.
The company is currently evaluating the long-term safety and tolerability of efgartigimod in the 3-year ADAPT + open-label expansion study (ClinicalTrials.gov: NCT03770403) in 118 patients who completed the ADAPT study.
A target date of the Prescription Drug User Fee Act (PDUFA) of December 17, 2021 has been set for this application.
reference
argenx Announces FDA Approval of BLA Filing for efgartigimod for the Treatment of Generalized Myasthenia Gravis. [press release]. Breda, Netherlands: argenx; March 2, 2021.
This article originally appeared on MPR
Subjects:
Treatment of neuromuscular diseases with myasthenia gravis
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