Sanofi wins approval for Lumizyme successor, although uptake remains a mystery
It’s not yet clear how many patients will actually take it, but for the first time in 15 years, there is a new treatment available for the rare and deadly genetic condition Pompe disease.
The FDA approved avalglucosidase alfa, the drug Sanofi has been developing to succeed their best-selling Pompe disease treatment Myozyme/Lumizyme, a therapy that transformed the devastating disorder when it was first approved in 2006. Like Lumizyme, the new treatment is an enzyme-replacement therapy, a lab-grown version of the enzymes patients lack and whose absence can destroy muscle cells.
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