The Center for Drug Evaluation (CDE) website has indicated that Visirna Therapeutics’ novel siRNA therapy VSA001, Sperogenix Therapeutics’ vamorolone, and Zai Lab’s (HKG: 9688) efgartigimod are on track for breakthrough therapy designation (BTD) status awards in China.
VSA001 for Familial Chylomicronemia Syndrome (FCS)
VSA001 is designed to reduce triglyceride levels in adult patients with familial chylomicronemia syndrome (FCS), a serious and extremely rare genetic disease characterized by extremely high levels of fasting triglycerides. The drug, a liver-targeted small interfering RNA (siRNA), effectively silences the messenger RNA of apolipoprotein C3 (APOC3 mRNA), leading to a significant reduction in serum triglyceride (TG) and triglyceride-rich lipoprotein (TRL) levels. With no current treatment available for FCS, VSA001 could offer a significant advancement in patient care.
Vamorolone for Duchenne Muscular Dystrophy (DMD)
Vamorolone, a first-in-class Duchenne muscular dystrophy (DMD) therapy, is a dissociative agonist that binds to the same receptor as glucocorticoids but modifies their downstream activity. This mechanism may offer a safer alternative to the standard therapy for children and adolescents with DMD. Sperogenix secured exclusive development and commercialization rights to vamorolone in Greater China through a USD124 million licensing deal with Santhera Pharmaceuticals in January 2022.
Efgartigod for Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
Zai Lab’s efgartigimod is under development to treat chronic inflammatory demyelinating polyneuropathy (CIDP). Zai Lab holds exclusive rights to develop and commercialize efgartigimod in Greater China, following a license agreement with argenx signed in July 2022. Efgartigod is the first approved FcRn blocker in the United States, EU, and China for the treatment of adults with gMG who are anti-acetylcholine receptor (AChR) antibody positive, and in Japan for adults with gMG who do not respond sufficiently to steroids or non-steroidal immunosuppressive therapies (ISTs). Vyvgart, available in IV injection form, was approved in China in July.-Fineline Info & Tech
