FDA Expands Access to Lumizyme, Drops Risk Mitigation Requirements

With its expanded approval of Lumizyme (alglucosidase alfa) to treat all Pompe disease patients, regardless of age or time of disease onset, the FDA has eliminated the Risk Evaluation and Mitigation Strategy (REMS) program that restricted the drug’s use to those aged 8 years or older with late-onset disease.

According to the FDA, Pompe disease is a rare disorder that “causes gene mutations to prevent the body from making enough of the functional form of an enzyme called acid alpha-glucosidase (GAA),” which is necessary for proper muscle functioning. Consequently, the primary symptom of Pompe disease is heart and skeletal muscle weakness that can progress to respiratory weakness and even death from respiratory failure.

As a lysosomal glycogen-specific enzyme, Genzyme’s Lumizyme is believed to resolve GAA deficiency in Pompe patients, though the drug lacked sufficient data to support its safety and efficacy in the infantile-onset disease population when it was originally approved in 2010. Until that approval was expanded, patients younger than 8 years continued treatment with Myozyme (alglucosidase alfa), another Pompe treatment manufactured by Genzyme, while the FDA operated a REMS program for Lumizyme to “mitigate the potential risk of rapid disease progression … and to communicate the risks of anaphylaxis, severe allergic reactions, and severe skin and systemic immune mediated reactions to prescribers and patients,” the agency stated.

However, the FDA recently reviewed new clinical data that demonstrated Lumizyme and Myozyme have comparable chemical compositions and risks, which supported the use of Lumizyme in all Pompe patients and ultimately led to the drug’s label expansion. Because the distribution of Lumizyme is no longer restricted to certain Pompe patients, the FDA said health care professionals, health care facilities, and patients are no longer required to enroll in the Lumizyme REMS program in order to prescribe, dispense, or receive the drug.

“We are thankful to the entire Pompe community who has been on this journey to provide a sustainable, long-term option for Pompe patients in the United States,” Genzyme President and CEO, David Meeker, MD, said in a company statement. “We are pleased we can now offer alglucosidase alfa produced at the 4000-L scale to all patients.”