Nexviazaim(component name: Avalglucosidase Alpha), a treatment for Sanofi Pompeo's disease, received an item license from the Ministry of Food and Drug Safety on the 29th. The indication is "a patient confirmed with pompe disease (acid alpha-glucosidase deficiency)" and can be used as a long-term enzyme replacement therapy.
NexviaZaim is the first rare drug to be licensed as an improved biopharmaceutical in recognition of its clinical improvement and advances in pharmaceutical technology. Prior to Nexvia-Zaym's approval, myoZaym was the only treatment available to patients with Pompeii.
Pompeo's disease is a progressive and hereditary neuromuscular disease that can affect patients of all ages due to a lack of acidic alpha glucosidase (GAA), an enzyme that breaks down glycogen in muscles. If the disease is left unattended, it can lead to irreversible muscle damage, respiratory function and decreased mobility, and there is also a possibility of early death.
Improved biopharmaceuticals refer to drugs recognized by the Ministry of Food and Drug Safety that improve safety, effectiveness, and usefulness (dose compliance, convenience, etc.) or are progressive in drug technology compared to already licensed biopharmaceuticals. NexviaZaim has been recognized for its pharmaceutical improvement and progressiveness over the past few years compared to myoZime, the only treatment licensed in Pompeii.
Nexviazyme has increased the amount of M6P, which plays an important role in the intracellular absorption of drugs, by about 15 times compared to conventional myozyme through innovative glyco-engineering technology. Increased M6P of nexviazyme can increase the absorption of drugs compared to myozyme, improve GAA activity, and reduce damage to muscle cells through effective glycogen degradation. In addition, increased surface M6P contributes to the improvement of immunogenicity and is advantageous in safety.
NexviaZaim was recognized as the third domestic specialty drug and the first rare drug to be approved as an improved biopharmaceutical. In particular, it was designated as a Breakthrough Therapy Designation by the U.S. FDA prior to domestic approval, approved in August 2021, and approved for an item license in Europe in June last year.
