Pombiliti™ (cipaglucosidase alfa-atga)

Pombiliti (cipaglucosidase alfa-atga) is authorized up to 12 months for adults ≥18 years with late‑onset Pompe disease weighing ≥40 kg who have laboratory or genetic confirmation of GAA deficiency and who have not improved after ≥1 year of prior ERT (Lumizyme or Nexviazyme), when given 20 mg/kg IV every other week in combination with Opfolda and prescribed by or in consultation with a geneticist, neurologist, metabolic disorder subspecialist, or lysosomal storage disorder specialist. Required documentation includes confirmatory lab/genetic testing, records of ≥1 year prior ERT plus objective measures showing lack of improvement (e.g., FVC or 6‑minute walk), age/weight, specialist involvement, and a treatment/dosing plan; excluded are patients <18 years, <40 kg, without confirmatory testing, improving on prior ERT, or receiving Pombiliti without Opfolda.

"Weight restriction: only for individuals weighing 40 kg or greater."