Opfolda (miglustat) — Highmark

Late-onset Pompe disease (LOPD)

Initial criteria

age ≥ 18 years
member weighs ≥40 kg
diagnosis of late-onset Pompe disease (LOPD)
member is not improving on current enzyme replacement therapy (ERT)
Opfolda is used in combination with Pombiliti (cipaglucosidase alfa-atga)
confirmation that the member meets one of the following: (1) has coverage of Pombiliti through medical insurance OR (2) has coverage of Pombiliti through another form of coverage (e.g., out-of-pocket expense)

member has experienced improvement in signs or symptoms for Pompe disease from baseline (e.g., decreased heart size, normal heart function, improvement of muscle strength, improvement in walking distance, improvement in respiratory function) OR has reduced glycogen accumulation from baseline
member continues to use Opfolda in combination with Pombiliti
confirmation that the member has coverage of Pombiliti through medical insurance or another form of coverage

12 months