Three Novel Therapies from Zai Lab, Shu Pharm and Visirna Therapeutics Designated for Breakthrough Therapy Status in China

On September 8, the CDE website showed that Efgartigimod Injection from Zai Lab, Vamorolone Oral Suspension from Beijing Hanyou Pharmaceutical Technology Co., Ltd., and VSA001 Injection from Visirna were all proposed to be included in the breakthrough therapy designation.

Drug Name: Efgartigimod

Company: Zai Lab

Indications: Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) in adults

Efgartigimod is an FcRn monoclonal antibody developed by Argenx. It can reduce pathogenic IgG antibodies and block IgG recycling by binding to FcRn. In January 2021, Zai Lab reached an exclusive licensing agreement with Argenx for the development and commercialization of Efgartigimod in Greater China, with a total deal value of $175 million. At the end of June this year, the intravenous injection of Efgartigimod was approved for marketing in China to treat patients with generalized myasthenia gravis.

Drug Name: Vamorolone

Company: Shu Fang Pharmaceutical

Indications: Duchenne Muscular Dystrophy (DMD) in 2-year-olds

Vamorolone is a newly synthesized free steroidal hormone drug. It retains the anti-inflammatory effects of traditional hormones (such as prednisone/prednisolone) while reducing some of the side effects associated with conventional steroids, thereby extending the duration of steroid use in DMD patients and further slowing disease progression.

Previously published results from the Phase IIb VISION-DMD study showed that, compared with the placebo group, patients in the treatment group demonstrated statistically significant and clinically meaningful improvement in the time required to rise from supine to standing. Meanwhile, the most common adverse reactions (such as Cushingoid features, vomiting, and vitamin D deficiency) were significantly reduced.

Drug Name: VSA001

Company: Visirna

Indications: To reduce triglyceride levels in adult patients with familial chylomicronemia syndrome (FCS) on the basis of a low-fat diet and other lipid-lowering therapies (e.g., fibrates).

VSA001 is a liver-targeted small interfering RNA (siRNA) drug for the treatment of lipid metabolism disorders. It can effectively and persistently reduce APOC3 protein expression by silencing the messenger RNA of apolipoprotein C3. Through both lipoprotein lipase-dependent and -independent pathways, it significantly lowers serum triglycerides and triglyceride-rich lipoproteins as well as their degradation remnants. Currently, this product is undergoing clinical trials for familial chylomicronemia syndrome, hypertriglyceridemia, and mixed hyperlipidemia.

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