Long-Term Observation of the Safety and Effectiveness of Enzyme Replacement Therapy in Japanese Patients with Pompe Disease: Results From the Post-marketing Surveillance

INTRODUCTION: Alglucosidase alfa received marketing approval for the treatment of Pompe disease in Japan in 2007. We conducted a post-marketing surveillance study to monitor the long-term safety and efficacy of alglucosidase alfa therapy among Japanese patients with Pompe disease. METHODS: The safety and efficacy outcomes were collected as real-world data for up to 9 years following the initiation of treatment with alglucosidase alfa, without any intervention to treatment strategies. The safety of the drug was assessed in 73 patients in terms of the rate of drug-related adverse events, infusion-associated reactions, and antibody titers. The efficacy was evaluated in 72 patients on the basis of subjective evaluation of their general condition after treatment, pulmonary function, 6-min walk test, and survival rate. RESULTS: Drug-related adverse events were observed in 29 of 73 (39.7%) cases, and the cumulative adverse event rate during the 9 years of the study was 45.7%. Immunoglobulin G antibodies against alglucosidase alfa were positive in 59 of 61 cases in which the titers were not correlated with drug-related adverse events or infusion-associated reactions. After the final dosing, the treating physicians determined that the disease was at least stabilized in 62 of 72 cases (86.1%), while the results of the physical function tests suggested that disease progression was actually not stopped completely. Survival of infantile-onset cases was sustained for 9 years. CONCLUSION: The drug was generally well tolerated, and treatment with alglucosidase alfa was able to suppress disease progression in the majority of Japanese patients with Pompe disease included in this study. FUNDING: Sanofi ELECTRONIC SUPPLEMENTARY MATERIAL: The online version of this article (10.1007/s40120-019-00157-4) contains supplementary material, which is available to authorized users.