China Agrees to Review Efgartigimod for Generalized MG
Zai Lab’s application seeking approval of efgartigimod in China to treat adults with generalized myasthenia gravis (gMG) has been accepted by the country’s regulatory agency, the National Medical Products Administration (NMPA).
“We are pleased to have the NMPA’s acceptance of the [application] filing for efgartigimod alfa injection for intravenous use,” Harald Reinhart, MD, Zai Lab’s president and head of global development, neuroscience, autoimmune and infectious diseases, said in a press release.
“We look forward to collaborating with the NMPA during the review process in a joint effort to address unmet needs of gMG patients in Greater China,” he added.
Zai Lab is leading development and potential commercialization of efgartigimod in Greater China — including the mainland, Hong Kong, Taiwan, and Macau — under terms of a 2021 agreement with the therapy’s developer, Argenx. The company did not specify when a decision from the NMPA is expected.
“For the estimated 200,000 people in China living with MG, there is a lack of innovative treatment options, representing significant unmet needs in clinical practice,” said Chongbo Zhao, MD, PhD, deputy director of the department of neurology at Huashan Hospital, which is affiliated with Fudan University.
“First, current treatment options may fail to address refractory gMG or those likely to progress with life-threatening conditions. Second, long-term use of glucocorticoids or other immunosuppressants may lead to various intolerable side effects. Third, rescue therapies such as intravenous immune globulin (IVIg) or plasma exchange (PLEX) are limited in supply or require patients to go to specialty medical facilities,” Zhao said. “Therefore, there is an urgent need for a novel, safe, effective therapy to improve the treatment outcome and quality of life for patients.”
In MG, symptoms are driven by self-reactive antibodies that interfere with the communication between nerve and muscle cells. Efgartigimod is designed to lower the levels of disease-driving antibodies by blocking the activity of a protein that normally helps prevent the breakdown of antibodies circulating in the bloodstream.
The therapy was approved in recent months in the U.S. and Japan, where it is being marketed under the brand name Vyvgart, and it is currently under review in Europe.
The safety and efficacy of efgartigimod was demonstrated in a Phase 3 trial called ADAPT (NCT03669588), which enrolled 167 adults with gMG who were randomized to receive efgartigimod or a placebo for about six months. Participants then had the option to start or continue treatment with egartigimod in the open-label extension study ADAPT+ (NCT03770403).
Results from ADAPT showed that efgartigimod outperformed placebo at reducing gMG symptoms, irrespective of the presence of the most common type of MG-driving antibody, called anti-AChR. Long-term data from ADAPT+, which has recently been completed, have continued to show that Vyvgart eased symptoms up to at least a year of treatment.
“In clinical studies, efgartigimod has shown that it can provide fast, deep, and durable clinical response with a good safety profile,” Zhao said. “We appreciate Zai Lab’s investment and continued support of gMG patients in China.”
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