Innovative medicine treats autoimmune disorder

An innovative medicine to treat generalized myasthenia gravis, a chronic autoimmune disease that causes muscle weakness, was recently approved for the domestic market.

New therapies have been rarely available for Chinese patients in the past three decades.

Zai Lab, a Shanghai-based biopharmaceutical company, announced on Monday that China's National Medical Products Administration on June 30 approved Vyvgart (efgartigimod alfa injection) for the treatment of gMG in adults who are anti-acetylcholine receptor antibody positive.

Such patients represent roughly 80 percent of the total population living from this chronic neuromuscular disease characterized by debilitating and potentially life-threatening muscle weakness.

"This important milestone brings forward a novel treatment for gMG patients who face many challenges living with this complex and difficult-to-control autoimmune disease," said Samantha Du, founder, chairperson and CEO of Zai Lab.

Zhao Chongbo, deputy director of the department of neurology at Shanghai Huashan Hospital, and director of the working group at Huashan Rare Disease Center, said that there are over 200,000 people living with MG in China, and there remains a significant unmet medical need despite the current treatment options.

The disease gMG was included in the first batch of rare disease catalogues jointly issued by five national ministries and commissions in 2018.

"In clinical studies, the medicine demonstrated good characteristics in terms of onset of action, efficacy, and safety, helping to improve patients' muscle strength and quality of life," said Zhao.

"The therapy has the potential to revolutionize the treatment landscape for gMG patients in China."

Experts said that more than 85 percent of people with MG progress to gMG within 18 months.

In this generalized form of the disease, skeletal muscles throughout the body may be affected, resulting in weakness and early fatigue.

Difficulties with double vision, facial expression, speech, swallowing, and walking are frequent and difficult to manage for patients and treating physicians.

In more life-threatening cases, gMG can affect the muscles responsible for breathing.

A gMG patient since her high school years, who wanted to be identified by her nickname Jiajia, said that for many patients, including her, the biggest wish is to live and work in the way an ordinary, healthy person does.

"I once relied on ventilators for a whole month to survive and hormone therapy transformed my appearance thoroughly. There was no way that we could take care of ourselves at the onset of the disease, and it was extremely challenging psychologically," said the woman, a native of Anhui province in her 30s.

"I failed to smile when the disease deteriorated, but I hope I can smile whenever I want. I'm a mother of a toddler now and if I don't smile, my child will feel scared," she said.