alglucosidase alfa alglucosidase alfa alglucosidase alfa, sold under the brand name myozyme among others, is an enzyme replacement therapy (ert) orphan drug for treatment of pompe disease (glycogen storage disease type ii), a rare lysosomal storage disorder (lsd). chemically, the drug is an analog of the enzyme that is deficient in patients affected by pompe disease, alpha-glucosidase. it is the first drug available to treat this disease. not to be confused with avalglucosidase alfa. alglucosidase alfaclinical datatrade namesmyozyme, lumizyme, othersahfs/drugs.commonographlicense data eu ema: by inn us dailymed: alglucosidase alfa routes ofadministrationintravenousatc codea16ab07 (who) legal statuslegal status au: s4 (prescription only) us: ℞-only identifiers iupac name human glucosidase, prepro-α- cas number420784-05-0 ydrugbankdb01272 ychemspidernoneuniidti67o9503keggd03207 nchemical and physical dataformulac4758h7262n1274o1369s35molar mass105338 g·mol−1 ny (what is this?) (verify) it was approved for medical use in the united states in april 2006, as myozyme and in may 2010, as lumizyme. medical uses alglucosidase alfa is indicated for people with pompe disease (gaa deficiency). in 2014 the u.s. food and drug administration announced the approval of alglucosidase alfa for treatment of people with infantile-onset pompe disease, including people who are less than eight years of age. in addition, the risk evaluation and mitigation strategy (rems) is being eliminated. side effects common observed adverse reactions to alglucosidase alfa treatment are pneumonia, respiratory complications, infections and fever. more serious reactions reported include heart and lung failure and allergic shock. myozyme boxes carry warnings regarding the possibility of life-threatening allergic response. society and culture economics some health plans have refused to subsidize myozyme for adults because it lacks approval for treatment in adults, as well as its high cost (300,000 per year for life). in 2015, lumizyme was ranked the costliest drug per patient, with an average charge of $630,159.