amicus therapeutics us, llc - Medication Listings

Migalastat, an alpha-galactosidase A (alpha-Gal A) pharmacological chaperone, is a low molecular weight iminosugar and an analogue of the terminal galactose of globotriaosylceramide (GL-3). Migalastat is present in the form of a hydrochloride salt in GALAFOLD. The chemical name for migalastat hydrochloride is (+)-(2 R ,3 S ,4 R ,5 S )-2-(hydroxymethyl) piperidine-3,4,5-triol hydrochloride. Its molecular formula is C 6 H 13 NO 4 •HCl, molecular mass is 199.63 g/mol, and its chemical structure is depicted below. Migalastat hydrochloride is a white to almost white crystalline solid. It is freely soluble in aqueous media within the pH range of 1.2 to 7.5. GALAFOLD (migalastat) capsules for oral administration contain 123 mg of migalastat (equivalent to 150 mg migalastat hydrochloride) as a white to pale brown powder and are supplied in a size “2” hard gelatin capsule with an opaque blue cap and an opaque white body imprinted with “A1001” in black ink. The inactive ingredients are magnesium stearate and pregelatinized starch. Capsule shells consist of gelatin, indigotine - FD&C Blue 2, and titanium dioxide. The black ink consists of black iron oxide, potassium hydroxide, and shellac. Chemical Structure

Miglustat is an N-alkylated iminosugar, a synthetic analog of D‑glucose. The pharmacologic class is enzyme stabilizer. The chemical name is 1,5-(butylimino)-1,5-dideoxy-D-glucitol with the molecular weight of 219.28 g/mol, the molecular formula C 10 H 21 NO 4 , and the following chemical structure: Miglustat is a white to off-white crystalline solid (powder). It is highly soluble in water (>1,000 mg/mL as a free base). OPFOLDA (miglustat) capsules each contain 65 mg of miglustat for oral administration. The inactive ingredients are: colloidal silicon dioxide, magnesium stearate, microcrystalline cellulose, pregelatinized maize starch, and sucralose. The capsule shells include black iron oxide, gelatin, and titanium dioxide. Edible printing ink includes black iron oxide, and shellac. Chemical Structure

Cipaglucosidase alfa-atga is a hydrolytic lysosomal glycogen‑specific recombinant human α‑glucosidase (rhGAA) enzyme derived from a Chinese Hamster Ovary (CHO) cell line using perfusion methodology, resulting in cellularly (CHO)‑derived N‑glycans. Cipaglucosidase alfa‑atga is a glycoprotein with 1.3 mols of bis‑mannose‑6‑phosphate (bis‑M6P) per mol of enzyme. Cipaglucosidase alfa-atga has a molecular weight of approximately 110 kDa. POMBILITI (cipaglucosidase alfa-atga) for injection is a sterile, white to slightly yellowish lyophilized powder with a cake-like appearance for intravenous use after reconstitution and dilution. Each single-dose vial contains 105 mg of cipaglucosidase alfa-atga and the inactive ingredients citric acid monohydrate (4.57 mg), mannitol (140 mg), polysorbate 80 (3.5 mg), and sodium citrate (39 mg). After reconstitution with Sterile Water for Injection, USP, the resultant concentration is 15 mg/mL with a pH of between 5.7 to 6.3.