OPFOLDA

Miglustat maximum concentration (C max ) and area under the plasma concentration-time curve (AUC) increases proportionally over a dosage range from 130 mg to 260 mg (0.5 to 1 times the approved recommended dosage of 260 mg in patients weighing ≥50 kg). At the recommended 260 mg dose, the mean (SD) C max was approximately 3 (0.9) mcg/mL and the mean AUC was approximately 25 (6.5) mcg∗hr/mL.

AbsorptionThe mean time to reach the maximum concentration (T max ) of miglustat ranged from 2 hours to 3 ...

Trial 1 was a randomized, double‑blind, active‑controlled, international, multi-center clinical trial (NCT#03729362) in patients ≥18 years old diagnosed with LOPD. Patients were randomized 2:1 to receive OPFOLDA (260 mg orally for those ≥50 kg or 195 mg orally for those ≥40 kg to <50 kg) in combination with Pombiliti (20 mg/kg by intravenous infusion) or a non-U.S.-approved alglucosidase alfa product with placebo every other week for 52 weeks. The efficacy population included a total of 123 patients of whom 95 (77%) had received prior treatment with U.S.-approved alglucosidase alfa or a non-U....

Most common adverse reactions ≥5% are headache, diarrhea, fatigue, nausea, abdominal pain, and pyrexia. (6.1) To report SUSPECTED ADVERSE REACTIONS, contact Amicus Therapeutics at 1-877-4AMICUS or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not r...

OPFOLDA in combination with Pombiliti is contraindicated in Pregnancy [see Use in Specific Populations (8.1 )] Pregnancy. ( 4, 5.1, 8.1 )

Embryo-Fetal Toxicity: May cause embryo-fetal harm. Advise females of reproductive potential of the potential risk to a fetus and to use effective contraception during treatment and for at least 60 days after the last dose. ( 4, 5.1, 8.1, 8.3 ) Risks Associated with Pombiliti: Refer to the Pombiliti Prescribing Information for a description of additional risks for Pombiliti. (5.2) 5.1 Embryo-Fetal Toxicity Based on findings from animal reproduction studies, OPFOLDA in combination with Pombiliti ...