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Generic Name
Miglustat
Brand Names
Opfolda, Yargesa, Zavesca
FDA approval date: July 31, 2003
Classification: Glucosylceramide Synthase Inhibitor
Form: Capsule
What is Opfolda (Miglustat)?
OPFOLDA is indicated, in combination with Pombiliti, for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase deficiency) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy . OPFOLDA is an enzyme stabilizer indicated, in combination with Pombiliti, a hydrolytic lysosomal glycogen-specific enzyme, for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase deficiency) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy .
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Brand Information
OPFOLDA (miglustat)
1INDICATIONS AND USAGE
OPFOLDA is indicated, in combination with Pombiliti, for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT).
2DOSAGE FORMS AND STRENGTHS
Capsules: 65 mg of miglustat, white opaque hard gelatin capsule with grey opaque cap with “AT2221” printed in black on the body.
3CONTRAINDICATIONS
OPFOLDA in combination with Pombiliti is contraindicated in Pregnancy
4DESCRIPTION
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
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.
5CLINICAL STUDIES
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.S.-approved alglucosidase alfa product (ERT‑experienced) and 28 (23%) were ERT‑naïve. More than two thirds (n=64, 67%) of ERT‑experienced patients had been on ERT treatment for more than 5 years prior to entering Trial 1 (mean of 7.4 years).
Demographics, baseline sitting forced vital capacity (FVC) (% predicted), and 6-minute walk distance (6MWD) were generally similar between the 2 treatment groups (see
Key efficacy endpoints included assessment of sitting FVC (% predicted) and 6MWD (
Sitting FVC (Percent‑predicted) at 52 Weeks
Patients treated with OPFOLDA in combination with Pombiliti showed a mean change in sitting FVC from baseline at Week 52 of ‑1.1% as compared with patients treated with a non-U.S.-approved alglucosidase alfa product with placebo of ‑3.3%; the estimated treatment difference was 2.3% (95% CI: 0.02, 4.62).
Patients treated with OPFOLDA in combination with Pombiliti showed a mean change in sitting FVC from baseline at Week 52 of ‑1.1% as compared with patients treated with a non-U.S.-approved alglucosidase alfa product with placebo of ‑3.3%; the estimated treatment difference was 2.3% (95% CI: 0.02, 4.62).
The ERT‑experienced patients treated with OPFOLDA in combination with Pombiliti showed a numerically favorable change in sitting FVC from baseline at Week 52 (
Table 3. Summary of Sitting FVC in Adults with LOPD by ERT Status at 52 Weeks in Trial 1
Figure 2. Mean Change (± SE) in Sitting FVC (% predicted) from Baseline to Week 52 in ERT-experienced Adults with LOPD in Trial 1
SE: standard error; FVC: forced vital capacity; ERT: enzyme replacement therapy; LOPD: late-onset Pompe disease
6-Minute Walk Distance (6MWD) at 52 Weeks
Patients treated with OPFOLDA in combination with Pombiliti walked on average 21 meters farther from baseline as compared to those treated with a non-U.S.-approved alglucosidase alfa product with placebo who walked 8 meters farther from baseline; the estimated treatment difference was 14 meters (95% CI: -1, 28).
Patients treated with OPFOLDA in combination with Pombiliti walked on average 21 meters farther from baseline as compared to those treated with a non-U.S.-approved alglucosidase alfa product with placebo who walked 8 meters farther from baseline; the estimated treatment difference was 14 meters (95% CI: -1, 28).
The ERT-experienced patients treated with OPFOLDA in combination with Pombiliti showed a numerically favorable change in 6MWD from baseline at Week 52 (
Table 4. Summary of 6MWD in Adults with LOPD by ERT Status at 52 Weeks in Trial 1
Figure 3. Mean Change (± SE) of 6MWD from Baseline to Week 52 in ERT-experienced Adults with LOPD in Trial 1*
SE: standard error; 6MWD: 6-minute walk distance; ERT: enzyme replacement therapy; LOPD: late-onset Pompe disease
6HOW SUPPLIED/STORAGE AND HANDLING
How Supplied
OPFOLDA (miglustat) capsules are supplied as 65 mg, white opaque hard gelatin capsules with a grey opaque cap with “AT2221” printed in black on the body, and are available in bottles with child resistant caps. See Table 5 for the available OPFOLDA packages.
OPFOLDA (miglustat) capsules are supplied as 65 mg, white opaque hard gelatin capsules with a grey opaque cap with “AT2221” printed in black on the body, and are available in bottles with child resistant caps. See Table 5 for the available OPFOLDA packages.
Table 5. OPFOLDA Packages
Storage and Handling
Store at 20°C to 25°C (68°F to 77°F). Excursions are permitted between 15°C to 30°C (59°F to 86°F), [see USP Controlled Room Temperature].
Store at 20°C to 25°C (68°F to 77°F). Excursions are permitted between 15°C to 30°C (59°F to 86°F), [see USP Controlled Room Temperature].
Store in the original container to protect from light.
7PATIENT COUNSELING INFORMATION
Advise the patient to read the FDA‑approved patient labeling (Patient Information).
OPFOLDA must be administered in combination with Pombiliti. Refer to the Pombiliti Prescribing Information for Pombiliti patient counseling information.
Administration
Advise the patient and caregiver to follow the timeline recommendations for taking OPFOLDA prior to the intravenous infusion with Pombiliti and to follow the fasting recommendation. Advise the patient and caregiver that OPFOLDA should be swallowed only with unsweetened beverages [see .
Advise the patient and caregiver to follow the timeline recommendations for taking OPFOLDA prior to the intravenous infusion with Pombiliti and to follow the fasting recommendation. Advise the patient and caregiver that OPFOLDA should be swallowed only with unsweetened beverages [see .
Embryo-Fetal Toxicity
OPFOLDA in combination with Pombiliti may cause embryo-fetal harm. Advise a female patient and caregiver to inform their healthcare provider of a known or suspected pregnancy [see .
OPFOLDA in combination with Pombiliti may cause embryo-fetal harm. Advise a female patient and caregiver to inform their healthcare provider of a known or suspected pregnancy [see .
Advise a female of reproductive potential to use effective contraception during treatment with OPFOLDA in combination with Pombiliti and for at least 60 days after the last dose
Lactation
Advise a lactating female not to breastfeed during treatment with OPFOLDA in combination with Pombiliti [see .
Advise a lactating female not to breastfeed during treatment with OPFOLDA in combination with Pombiliti [see .
Infertility
Advise the male or female of reproductive potential that OPFOLDA in combination with Pombiliti may impair fertility [see .
Advise the male or female of reproductive potential that OPFOLDA in combination with Pombiliti may impair fertility [see .
Manufactured for:
OPFOLDA and Pombiliti are registered trademarks of Amicus Therapeutics, Inc.
8PRINCIPAL DISPLAY PANEL - NDC: 71904-300-01 - 4 Count Bottle Label
9PRINCIPAL DISPLAY PANEL - NDC: 71904-300-02 - 24 Count Bottle Label
10PRINCIPAL DISPLAY PANEL - NDC: 71904-300-03 - 100 Count Bottle Label
