Pombiliti™ (cipaglucosidase alfa-atga)

Pombiliti (cipaglucosidase alfa‑atga) is covered only 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 demonstrated improvement in objective measures (e.g., FVC or 6MWT) after ≥1 year of prior ERT with Lumizyme or Nexviazyme; therapy must be prescribed by or in consultation with a geneticist, neurologist, metabolic/lysosomal storage disorder specialist, given with Opfolda, dosed 20 mg/kg IV every other week, and is authorized for 12 months. Patients who are improving on current ERT, are <18 years or <40 kg, lack diagnostic confirmation, will not receive Opfolda, or lack required specialist involvement are excluded.

"Pombiliti is indicated, in combination with Opfolda, for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing greater than or e..."