Long-Term Efgartigimod Tied to Improved Quality of Life in Myasthenia Gravis

Among patients with myasthenia gravis, the long-term use of efgartigimod was tied to improvements in activities of daily living and quality of life and was well-tolerated.

Long-term use of efgartigimod is well-tolerated and demonstrates improvements in activities of daily living and quality of life in patients with myasthenia gravis (MG), according to study results presented at the 2023 American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting, held from November 1 to 4 in Phoenix, Arizona.

The ADAPT-SC open label trial (ClinicalTrials.gov Identifier: NCT04735432) was conducted to compare the safety and efficacy of subcutaneous vs multiple infusions of efgartigimod coformulated with PH20 in patients with generalized MG.

For the current study, researchers assessed the safety and efficacy of efgartigimod in patients with anti-acetylcholine receptor antibody-negative (AChR-Ab-) generalized MG in the ADAPT-SC study population.

The researchers included patients with MG from the ADAPT-SC study who received at least 1 dose of subcutaneous efgartigimod PH20 and were AChR-Ab- in the analysis.

Long-term treatment with efgartigimod PH20 SC was associated with consistent and repeatable improvements in MG-ADL and MG-QoL15r scores in AChR-Ab– patients in ADAPT-SC+ and was well tolerated.

Of 164 patients who received efgartigimod in the ADAPT-SC study, 30 were AChR-Ab-. Median follow-up was 204 days.

Patients with MG who were AChR-Ab- showed improvements from study baseline in scores of activities of daily living and quality of life (-3.6±0.53 and -3.3±0.80, respectively) at week 4. The results were consistent with those observed among AChR-Ab+ patients (-4.1±0.29 and -5.2±0.47, respectively).

With regard to safety of efgartigimod, 76.2% of study participants had 1 or more mild/moderate adverse events, including injection site erythema, headache, and COVID-19. None of the adverse events led to study discontinuation.

The researchers concluded, “Long-term treatment with efgartigimod PH20 SC [subcutaneous] was associated with consistent and repeatable improvements in MG-ADL [activities of daily living and quality of life] scores in AChR-Ab- patients in ADAPT-SC+ and was well tolerated.”

Disclosure: Multiple study authors declared affiliations with biotech, pharmaceutical, and/or device companies. Please see the original reference for a full list of the authors’ disclosures.

References:

Howard Jr JF, Vu T, De Bleecker JL, et al. Long-term efficacy and safety of efgartigimod PH20 SC in anti-acetylcholine receptor autoantibody seronegative patients with generalized myasthenia gravis: interim analysis of the ADAPT-SC+ study. Abstract presented at: AANEM 2023; November 1-4, 2023; Phoenix, AZ. Abstract #151.