Regulator Oks Sanofi's improved Pompe disease treatment

Sanofi Aventis Korea said that the Ministry of Food and Drug Safety approved Nexviazyme (ingredient: avalglucosidase alfa-ngpt), its Pompe disease treatment on Thursday.

The approved indication in Korea is for long-term enzyme replacement therapy in patients with confirmed Pompe disease (acid alpha-glucosidase deficiency). 

Nexviazyme is the first orphan drug to receive marketing authorization as an improved biologic. Prior to the approval of Nexviazyme, the only treatment available for patients with Pompe disease was Myozyme, developed by Genzyme Corp.

Pompe disease is a progressive, inherited neuromuscular disease caused by a deficiency of acid alpha-glucosidase (GAA), an enzyme that breaks down glycogen in muscle, and can affect patients of all ages.

If left untreated, the disease can lead to irreversible muscle damage, decreased respiratory function and mobility, and potentially premature death. 

An improved biologic drug is a drug that has received recognition from the Minister of Food and Drug Safety as having improved safety, efficacy, usefulness, or advances in medical technology compared to an already licensed biologic drug.

Nexviazyme was approved as an improved biologic for its formulation improvements and advancements over Myozyme.

Through innovative glycoengineering techniques, Nexviazyme has increased the amount of M6P, which plays a critical role in the drug's intracellular uptake, by approximately 15-fold compared to Myozyme.

This increase in M6P in Nexviazyme may increase drug uptake and improve GAA activity compared to Myozyme, resulting in more effective glycogenolysis and less damage to muscle cells. 

Increased surface M6P also contributes to improved immunogenicity, which has safety benefits. 

"Pompe disease is an irreversible, progressive, rare disease with a positive prognosis if treated early, and we are pleased to be able to offer our innovative new drug Nexviazyme to patients who are struggling with treatment," Sanofi Aventis Specialty Care Business Division General Manager Park Hee-kyung said.