New agent approved for late-onset Pompe disease
FDA has approved alglucosidase for patients aged 8 years and older for the treatment of late-onset Pompe disease.
FDA has approved alglucosidase alfa (Lumizyme, Genzyme) for patients aged 8 years and older for the treatment of late-onset (noninfantile) Pompe disease.
The agent was approved with a risk evaluation and mitigation strategy and will be available only through a restricted distribution system. The product labeling will also contain a boxed warning regarding an increased risk of anaphylaxis and severe allergic and immune-mediated reactions.
The approval of this agent was based on a clinical study in 90 patients aged 10 to 70 years with late-onset Pompe disease. In this study, the most common adverse events included infusion-related reactions.
Major congenital malformations not linked to first trimester tetracycline use
November 22nd 2024A large population-based study found that first-trimester tetracycline exposure does not elevate the risk of major congenital malformations, though specific risks for nervous system and eye anomalies warrant further research.
