New Treatment Approved for Adults and Children With Pompe Disease | Pediatrics | JAMA | JAMA Network
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News From the Food and Drug Administration
September 21, 2021

New Treatment Approved for Adults and Children With Pompe Disease

JAMA. 2021;326(11):1000. doi:10.1001/jama.2021.15588

A second enzyme replacement treatment has received approval for the treatment of late-onset Pompe disease in patients aged 1 year or older.

People with late-onset Pompe disease have genetic variants that create a deficiency of acid α-glucosidase (GAA), an enzyme that helps break down glycogen into glucose for energy. Without adequate GAA, the glycogen buildup damages muscles, especially heart and skeletal muscle, which can lead to death from heart or respiratory failure. The first enzyme replacement product for Pompe disease, alglucosidase alfa, received approval in 2010.

The new product, avalglucosidase alfa-ngpt, was approved based on results from a randomized phase 3 clinical trial involving 100 patients. Compared with patients who took alglucosidase alfa, those who received avalglucosidase alfa-ngpt had slightly better lung function at week 49. Patients who received avalglucosidase alfa-ngpt also could walk 30 m farther in a 6-minute walking test than those in the comparator group.

Both drugs come with a warning about the potential for severe allergic reactions. Common adverse effects from avalglucosidase alfa-ngpt included headache, fatigue, diarrhea, nausea, vomiting, joint pain, dizziness, muscle pain, itching, difficulty breathing, welts, and a “pins and needles” sensation.

Avalglucosidase alfa-ngpt will have the same price as alglucosidase alfa, according to a statement from Sanofi, which makes both drugs. Alglucosidase alfa, marketed as Lumizyme, is reportedly one of the most expensive drugs on the market at about $630 000 a year, a price that has more than doubled since 2017.

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