Pombiliti Atga cipaglucosidase alfa-atga 105 mg Injection, Powder, Lyophilized, For Solution — NDC 71904-200-03 (Billing 71904-0200-03)
This is a package of Pombiliti Atga cipaglucosidase alfa-atga 105 mg Injection, Powder, Lyophilized, For Solution from AMICUS THERAPEUTICS US, LLC, marketed since Oct 2023 and currently FDA-listed.
NDC database record
One package, one record: these facts belong to NDC 71904-200-03 alone.
- Record
- FDA NDC Directory package listing · Human prescription drug
- Code segments
- 71904 labeler · 200 product · 03 package
- Package marketed since
- Oct 11, 2023
- Sample package
- No — commercial package
- Listing certified through
- Dec 31, 2027
- Barcode (UPC)
- 0371904200033, 0371904200019
- FDA record last changed
- Aug 27, 2026
Identity & classification
Regulatory identifiers FDA, NLM and CMS codes for this package
Drug-database identifiers Medi-Span GPI and First Databank GCN / HICL / AHFS classification
- GSN (GCN sequence number): 085350
- GCN: 54808
- HICL (First Databank): 049231
- AHFS class code: 44:00.00.00
- RxCUI (RxNorm): 2667440
Where does this data come from?
- FDA openFDA NDC Directory · synced Oct 1, 2026
- FDA label on DailyMed · label index refreshed Oct 5, 2026
- RxNorm (NLM RxNav) · catalog refreshed Oct 1, 2026
- Medi-Span GPI (licensed)
- First Databank (licensed) · refreshed Oct 1, 2026
RxNorm drug class
This medicine belongs to the Enzymes class.
Where does this data come from?
- RxClass (NLM) · catalog refreshed Oct 1, 2026
Clinical
- Pombiliti is an enzyme replacement therapy for late-onset Pompe disease — a rare condition where your body can't properly break down a stored sugar called glycogen, which then buil...
- What exactly is Pombiliti for, and why do I need to take it with Opfolda?
- Each Pombiliti infusion takes about 4 hours and is given through an IV in a clinical setting every other week. About an hour before the infusion, you'll take Opfolda by mouth. Your...
- What should I expect during my infusion, and how long does it take?
Patient education
Supplement & herbal interactions
Where does this data come from?
- MedlinePlus (NLM) · refreshed Oct 1, 2026
- FDA label on DailyMed · label index refreshed Oct 5, 2026
Ask a licensed pharmacist directly — free, answered by our team.
Pricing
A drug doesn't have one price. Each row is a different public payment system, and none is what you'd pay at the counter — that depends on your insurance. The ⓘ on each row explains what it measures.
| Price system | Per mL | Per package |
|---|---|---|
| Retail pharmacies payNADAC · weekly | Not in the retail survey — common for institutional, discontinued, or low-volume packs. | |
| Medicaid paysCMS SDUD · 12 mo | No recent Medicaid claims on file for this NDC — rare and low-volume NDCs are suppressed in the public data. | |
| Medicare drug plans payPart D · quarterly | No Part D plan price is available for this NDC in our data. | |
| Medicare Part B allowsASP · J1203 | $96.302 / J1203 unit | — |
Where does this data come from?
- CMS NADAC weekly file
- CMS ASP pricing files · refreshed Sep 20, 2026
- CMS Medicaid State Drug Utilization Data · refreshed Oct 5, 2026
- CMS Part D plan pricing files · refreshed Sep 24, 2026
- VA National Acquisition Center price file
Billing & reimbursement
Where does this data come from?
- CMS ASP NDC-HCPCS crosswalk · refreshed Sep 22, 2026
- DMEPDAC NDC-HCPCS crosswalk
- openFDA NSDE billing units · refreshed Sep 7, 2026
Packaging — all sizes for this product
| Package NDC | Description | Marketing start | Marketing end | Status |
|---|---|---|---|---|
| 71904-0200-01 71904-200-01 Main listing | 1 VIAL, GLASS in 1 CARTON / 105 INJECTION, POWDER, LYOPHILIZED, FOR SOLUTION in 1 VIAL, GLASS | 2023-10-11 | — | Active |
| 71904-0200-02 71904-200-02 | 10 VIAL, GLASS in 1 CARTON / 105 INJECTION, POWDER, LYOPHILIZED, FOR SOLUTION in 1 VIAL, GLASS | 2023-10-11 | — | Active |
| 71904-0200-03 You're viewing this | 25 VIAL, GLASS in 1 CARTON / 105 INJECTION, POWDER, LYOPHILIZED, FOR SOLUTION in 1 VIAL, GLASS | 2023-10-11 | — | Active |
Pack size FAQ
What quantity is in this package?
What NDC number is used to bill for this package of Pombiliti Atga cipaglucosidase alfa-atga 105 mg Injection, Powder, Lyophilized, For Solution?
Therapeutic equivalents
| Product | Labeler | Pack | NADAC/unit | TE | Status | Price vs. this |
|---|---|---|---|---|---|---|
| Pombiliti Atga 105 mgthis 71904-0200-03 | AMICUS | 25 vials | — | — | FDA listed | — |
Where does this data come from?
- FDA openFDA NDC Directory · synced Oct 1, 2026
- FDA Purple Book · refreshed Oct 5, 2026
- CMS NADAC weekly file
Availability & biosimilar status
Biologics have no small-molecule generics; biosimilar competition is tracked in the FDA Purple Book.
Why the date isn’t exact: Biosimilar timing can change because patents may be challenged, settled, licensed, added or removed, and litigation can move the real date earlier or later.
🛈 What do these terms mean?
- Biologic patent
- A patent the reference product’s maker has publicly listed. A biosimilar generally can’t launch until these expire — unless they’re invalidated or resolved in a settlement.
- Reference-product exclusivity
- A flat 12 years of FDA market protection from the biologic’s first licensure (the BPCIA). No biosimilar can be licensed before it ends, regardless of patents.
- Interchangeable exclusivity
- The first interchangeable biosimilar can earn a period as the only interchangeable version (pharmacists can substitute it without the prescriber).
- Earliest biosimilar (LOE)
- The latest of all the dates above — the soonest a biosimilar can realistically reach the market. Litigation and settlements can move it earlier.
Biologics have no small-molecule “generics” — competition comes from FDA-licensed biosimilars, tracked in the FDA Purple Book.
| Code | What it grants | Expires |
|---|---|---|
| RefProduct | Reference-product exclusivity (12-year, BPCIA) — no biosimilar can be licensed before this date | Sep 28, 2035 |
Is there a biosimilar for POMBILITI 105 MG VIAL?
Why do different websites show different biosimilar dates?
Can a biosimilar launch before the last patent expires?
What does “current Purple Book estimate” mean?
What does “FDA listed” mean?
What does a patent or protection date mean here?
Where does this data come from?
- FDA Purple Book · refreshed Oct 5, 2026
What it looks like
Where does this data come from?
- FDA label on DailyMed · label index refreshed Oct 5, 2026
Inactive Ingredients / Excipients
Inactive ingredients, also called excipients, are components of the drug product other than the active ingredient. They may include fillers, dyes, coatings, preservatives, flavors, or other formulation ingredients.
🧪 Avoiding an ingredient? See Cipaglucosidase Alfa-Atga inactive ingredients by manufacturer: every current product's list side by side, so you can ask your pharmacy for the version that does not list it.
Where does this data come from?
IACT rows and label-wide narrative are kept separate; availability and product-level specificity depend on the submitted label.- FDA label on DailyMed · label index refreshed Oct 5, 2026
- FDA openFDA NDC Directory · synced Oct 1, 2026
Inactive ingredient FAQ
Are inactive ingredients the same for every manufacturer?
Why might an inactive ingredient be missing?
Can inactive ingredients matter?
Manufacturer & labeler
More NDCs from AMICUS THERAPEUTICS US, LLC labeler code 71904
Where does this data come from?
- FDA openFDA NDC Directory · synced Oct 1, 2026
- Drugs@FDA
Full prescribing information FDA SPL
🚨 Boxed Warning ▾
WARNING: SEVERE HYPERSENSITIVITY REACTIONS, INFUSION-ASSOCIATED REACTIONS, and RISK OF ACUTE CARDIORESPIRATORY FAILURE IN SUSCEPTIBLE PATIENTS Hypersensitivity Reactions Including Anaphylaxis Patients treated with POMBILITI have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Appropriate medical support measures, including cardiopulmonary resuscitation equipment, should be readily available during POMBILITI administration. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, POMBILITI should be discontinued immediately, and appropriate medical treatment should be initiated.
In patients with severe hypersensitivity reaction, desensitization measures to POMBILITI may be considered [see Warnings and Precautions (5.1) ]. Infusion-Associated Reactions (IARs) Patients treated with POMBILITI have experienced severe IARs. If severe IARs occur, immediately discontinue the POMBILITI infusion, initiate appropriate medical treatment, and assess the benefits and risks of readministering POMBILITI following severe IARs.
Patients with an acute underlying illness at the time of POMBILITI infusion may be at greater risk for IARs. Patients with advanced Pompe disease may have compromised cardiac and respiratory function, which may predispose them to a higher risk of severe complications from IARs [see Warnings and Precautions (5.2) ]. Risk of Acute Cardiorespiratory Failure in Susceptible Patients Patients susceptible to fluid volume overload, or those with acute underlying respiratory illness or compromised cardiac or respiratory function for whom fluid restriction is indicated may be at risk of serious exacerbation of their cardiac or respiratory status during POMBILITI infusion.
More frequent monitoring of vitals should be performed during POMBILITI infusion in such patients [see Warnings and Precautions (5.3) ]. WARNING: SEVERE HYPERSENSITIVITY REACTIONS, INFUSION-ASSOCIATED REACTIONS, and RISK OF ACUTE CARDIORESPIRATORY FAILURE IN SUSCEPTIBLE PATIENTS See full prescribing information for complete boxed warning Hypersensitivity Reactions Including Anaphylaxis Appropriate medical support measures, including cardiopulmonary resuscitation equipment, should be readily available. If a severe hypersensitivity reaction occurs, POMBILITI should be discontinued immediately and appropriate medical treatment should be initiated.
( 5.1 ) Infusion-Associated Reactions (IARs) If severe IARs occur, immediately discontinue POMBILITI and initiate appropriate medical treatment. ( 5.2 ) Risk of Acute Cardiorespiratory Failure in Susceptible Patients Patients susceptible to fluid volume overload, or those with acute underlying respiratory illness or compromised cardiac or respiratory function, may be at risk of serious exacerbation of their cardiac or respiratory status during POMBILITI infusion. ( 5.3 )
🎯 Indications and Usage ▾
1 INDICATIONS AND USAGE POMBILITI is indicated, in combination with Opfolda, 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). POMBILITI is a hydrolytic lysosomal glycogen-specific enzyme indicated, in combination with Opfolda, an enzyme stabilizer, 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).
( 1 )
⏱️ Dosage and Administration ▾
2 DOSAGE AND ADMINISTRATION Verify pregnancy status in females of reproductive potential prior to initiating treatment. ( 2.1 ) Administer POMBILITI in combination with Opfolda. ( 2.2 ) Consider administering antihistamines, antipyretics, and/or corticosteroids prior to POMBILITI administration.
( 2.2 ) Recommended POMBILITI dosage is 20 mg/kg (of actual body weight) administered every other week as an intravenous infusion over approximately 4 hours. ( 2.2 ) Start POMBILITI in combination with Opfolda 2 weeks after the last ERT dose. ( 2.2 ) Initiate the POMBILITI infusion approximately 1 hour after oral administration of Opfolda.
If the POMBILITI infusion cannot be started within 3 hours of oral administration of Opfolda, reschedule POMBILITI in combination with Opfolda at least 24 hours after Opfolda was last taken. If POMBILITI in combination with Opfolda are both missed, re-start treatment as soon as possible. ( 2.2 ) See the full prescribing information for dosage modifications due to hypersensitivity reactions or IARs.
( 2.3 ) Must be reconstituted and diluted prior to use. ( 2.4 ) See the full prescribing information for administration instructions. ( 2.6 )
2.1Pregnancy Evaluation Prior to Initiating Treatment Verify the pregnancy status of females of reproductive potential prior to initiating POMBILITI in combination with Opfolda [see Use in Specific Populations (8.1 , 8.3 )] .
2.2Recommended Dosage and Administration POMBILITI must be administered in combination with Opfolda (see Figure 1 for the dosing timeline). If the Opfolda dose is missed, POMBILITI should not be administered. Refer to the Opfolda Prescribing Information for Opfolda dosage and administration recommendations.
Prior to POMBILITI administration, consider pretreating with antihistamines, antipyretics, and/or corticosteroids [see Warnings and Precautions (5.1 , 5.2 )] . If premedication was used with previous enzyme replacement therapy (ERT), prior to POMBILITI administration, pretreat with antihistamines, antipyretics, and/or corticosteroids. The recommended dosage of POMBILITI is 20 mg/kg (of actual body weight) administered every other week as an intravenous infusion over approximately 4 hours (see Table 1 for the recommended total infusion volume based on the patient’s weight).
Start POMBILITI in combination with Opfolda 2 weeks after the last ERT dose. Initiate the POMBILITI infusion approximately 1 hour after oral administration of Opfolda. If the POMBILITI infusion cannot be started within 3 hours of oral administration of Opfolda, reschedule POMBILITI in combination with Opfolda at least 24 hours after Opfolda was last taken.
If POMBILITI in combination with Opfolda are both missed, re-start treatment as soon as possible. Figure 1. Dosing Timeline Figure 1
2.3Dosage and Administration Modifications Due to Hypersensitivity Reactions and/or Infusion-Associated Reactions In the event of a severe hypersensitivity reaction (including anaphylaxis) or a severe infusion-associated reaction (IAR), immediately discontinue the POMBILITI infusion, and initiate appropriate medical treatment. For additional recommendations in the event of a severe hypersensitivity reaction [see Warnings and Precautions (5.1 , 5.2 )] . In the event of a mild to moderate hypersensitivity reaction or moderate IAR, consider temporarily holding or slowing the infusion rate and initiating appropriate medical treatment [see Warnings and Precautions (5.1 , 5.2 )] .
If symptoms: Persist despite temporarily holding or slowing the infusion, stop the infusion for 30 to 60 minutes, monitor the patient, and consider resuming the infusion at a reduced rate if symptoms have improved. If symptoms continue to persist, discontinue the infusion, and consider re-initiating the infusion within 7 to 14 days with appropriate premedication. Subside following holding or slowing the infusion, increase the infusion rate to the rate at which the reaction occurred and consider continuing to increase… [Excerpted — this section continues on DailyMed.]
💊 Dosage Forms and Strengths ▾
3 DOSAGE FORMS AND STRENGTHS For injection: 105 mg of cipaglucosidase alfa-atga as a white to slightly yellowish lyophilized powder with a cake-like appearance in a single-dose vial for reconstitution For injection: 105 mg of cipaglucosidase alfa-atga as a lyophilized powder in a single-dose vial for reconstitution. ( 3 )
⛔ Contraindications ▾
4 CONTRAINDICATIONS POMBILITI in combination with Opfolda is contraindicated in pregnancy [see Warnings and Precautions (5.4) and Use in Specific Populations (8.1) ]. Pregnancy ( 4 , 5.4 , 8.1 )
⚠️ Warnings and Cautions ▾
5 WARNINGS AND PRECAUTIONS 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 , 5.4 , 8.1 , 8.3 ) Risks Associated with Opfolda: Refer to the Opfolda Prescribing Information for a description of additional risks for Opfolda. ( 5.5 )
5.1Hypersensitivity Reactions Including Anaphylaxis Life-threatening hypersensitivity reactions, including anaphylaxis, have been reported in POMBILITI-treated patients. In clinical trials, 43 (27%) POMBILITI-treated patients experienced hypersensitivity reactions, including 4 (3%) patients who reported severe hypersensitivity reactions and 4 (3%) additional patients who experienced anaphylaxis (fulfilling at least one of the Sampson criteria). Three of the 4 (2%) patients experiencing anaphylaxis discontinued from the trial [see Clinical Studies (14) ] .
Two of the 4 patients who experienced anaphylaxis developed high anti-cipaglucosidase alfa-atga antibody titers [see Clinical Pharmacology (12.6) ] . Anaphylaxis signs and symptoms included dyspnea, rash, hypotension, bronchospasm, edema, pharyngeal edema, and tongue swelling. Symptoms of severe hypersensitivity reactions included urticaria, pruritus, and flushing.
Prior to POMBILITI administration, consider pretreating with antihistamines, antipyretics, and/or corticosteroids. Appropriate medical support measures, including cardiopulmonary resuscitation equipment, should be readily available during POMBILITI administration. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, POMBILITI should be discontinued immediately, and appropriate medical treatment should be initiated.
The risks and benefits of readministering POMBILITI following severe hypersensitivity reaction (including anaphylaxis) should be considered. Patients may be rechallenged using slower infusion rates. In patients with severe hypersensitivity reaction, desensitization measures to POMBILITI may be considered.
If the decision is made to readminister POMBILITI, ensure the patient tolerates the infusion. If the patient tolerates the infusion, the dosage (dose and/or the rate) may be increased to reach the approved recommended dosage. If a mild or moderate hypersensitivity reaction occurs, the infusion rate may be slowed or temporarily stopped [see Dosage and Administration (2.3) ].
5.2Infusion-Associated Reactions In clinical trials, IARs were reported to occur at any time during and/or within a few hours after the POMBILITI infusion and were more likely to occur with higher infusion rates. IARs were reported in 48 (32%) POMBILITI-treated patients in clinical trials. In these trials, 4 (3%) POMBILITI-treated patients reported 11 severe IARs including symptoms of pharyngeal edema, anaphylactic reaction, urticaria, pruritus, chills, dyspnea, and flushing.
The majority of IARs were assessed as mild to moderate. IARs that led to treatment discontinuation were urticaria, anaphylactic reaction, chills, and hypotension. Antihistamines, antipyretics, and/or corticosteroids can be given prior to POMBILITI administration to reduce the risk of infusion-associated reactions (IARs).
However, IARs may still occur in patients after receiving pretreatment. If severe IARs occur, immediately discontinue the POMBILITI infusion, initiate appropriate medical treatment, and assess the benefits and risks of readministering POMBILITI following severe IARs. Patients may be rechallenged using slower infusion rates.
Once a patient tolerates the infusion, the infusion rate may be increased to reach the recommended infusion rate. If mild or moderate IARs occur regardless of pretreatment, decreasing the infusion rate or temporarily stopping the infusion may ameliorate the symptoms [see Dosage and Administration (2.3) ]. Patients with an acute underlying illness at the time of POMBILITI infusion may be at greater risk for IA… [Excerpted — this section continues on DailyMed.]
🤒 Adverse Reactions ▾
6 ADVERSE REACTIONS The following clinically significant adverse reactions are described elsewhere in the labeling: Hypersensitivity Reactions Including Anaphylaxis [see Warnings and Precautions (5.1) ] Infusion-Associated Reactions [see Warnings and Precautions (5.2) ] 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.
6.1Clinical 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 reflect the rates observed in practice. Adverse Reactions from the Pooled Clinical Trials Including Trial 1 The pooled safety analysis from 3 clinical trials included 151 adult patients with late-onset Pompe disease (LOPD) treated with POMBILITI in combination with Opfolda including: 85 patients in the randomized, double-blind, active-controlled trial in adults (Trial 1) [see Clinical Studies (14) ] , 37 patients in the open-label extension trial where patients switched from a non‑U.S.‑approved alglucosidase alfa product [see Clinical Studies (14) ] to POMBILITI in combination with Opfolda, 29 patients in an open-label trial.
The total median duration of exposure in these trials was 21 months, with 120 patients having at least 12 months exposure to POMBILITI in combination with Opfolda. In these trials, 78% (n=117) of the patients received previous ERT (ERT‑experienced) with a mean treatment duration of 7.7 years. In these trials, serious adverse reactions reported in 2 or more patients treated with POMBILITI in combination with Opfolda were anaphylaxis and urticaria.
A total of 5 patients treated with POMBILITI in combination with Opfolda in these trials permanently discontinued POMBILITI due to adverse reactions, including 4 of these patients who discontinued the treatment because of a serious adverse reaction. The most common adverse reactions (≥5%) reported in the pooled safety population of patients treated with POMBILITI in combination with Opfolda in the 3 clinical trials were headache, diarrhea, fatigue, nausea, abdominal pain and pyrexia. In these trials, IARs were reported in 48 (32%) patients treated with POMBILITI in combination with Opfolda.
IARs reported in more than 1 patient included headache, myalgia, diarrhea, nausea, fatigue, muscle spasms, pyrexia, dizziness, cough, chills, rash, vomiting, dyspnea, pain, abdominal distension, tachycardia, urticaria, flatulence, pruritus, abdominal pain, chest discomfort, flushing, hyperhidrosis, dysgeusia, hypotension, and hypertension [see Warnings and Precautions (5.2) ]. Adverse Reactions from Trial 1 Trial 1 (a randomized, double‑blind, active‑controlled trial) included 123 adult patients with LOPD who were randomized in a 2:1 ratio to receive treatment with POMBILITI in combination with Opfolda or a non-U.S.-approved alglucosidase alfa product with placebo [see Clinical Studies (14) ].
The duration of exposure was similar for both treatment groups (overall mean exposure of 12 months). Most patients (77%) were ERT‑experienced, and a majority of patients in both treatment groups had >5 years of prior treatment with ERT (69% and 63% of patients in the POMBILITI in combination with Opfolda group and the non-U.S.-approved alglucosidase alfa product with placebo group, respectively). The most common adverse reactions (≥5%) reported in the patients who received POMBILITI in combination with Opfolda in Trial 1 were headache and diarrhea.
Table 2 summarizes frequent adverse reactions that occurred in patients treated with POMBILITI in combination with Opfolda in Trial 1. Trial 1 was not designed to demonstrate a statistically significant difference in the incidence of adverse reactions in the POMBILITI in combination wit… [Excerpted — this section continues on DailyMed.]
👥 Use in Specific Populations ▾
8 USE IN SPECIFIC POPULATIONS Lactation: Breastfeeding not recommended. ( 8.2 )
8.1Pregnancy Risk Summary Based on findings from animal reproduction studies, POMBILITI in combination with Opfolda may cause embryo-fetal harm when administered to a pregnant female and is contraindicated during pregnancy. In a rabbit embryo-fetal development study, great vessel and cardiac malformations were increased in offspring of pregnant rabbits treated with cipaglucosidase alfa-atga in combination with miglustat at 16-fold and 3-fold, respectively, the MRHD of POMBILITI and Opfolda based on plasma AUC exposure.
A No Observed Adverse Effect Level (NOAEL) was not identified for the combination. In a pre- and post-natal development study in rats, increases in pup mortality were seen following maternal treatment with cipaglucosidase alfa-atga (400 mg/kg) in combination with miglustat, or with cipaglucosidase alfa-atga (400 mg/kg) alone. The NOAEL for cipaglucosidase alfa-atga alone is 150 mg/kg (5-fold the POMBILITI MRHD margin).
A NOAEL for the combination was not identified. Margins at the lowest observed adverse effect level (LOAEL), relative to exposures at the MRHD of POMBILITI and Opfolda were 20-fold and 4-fold, respectively, based on plasma AUC exposure (see Data ) . There are no available human data on POMBILITI in combination with Opfolda use in pregnant females to evaluate for a drug-associated risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes.
Data Animal Data Reproductive toxicity studies of cipaglucosidase alfa-atga in rats and rabbits included pretreatment with diphenhydramine (DPH) to prevent or minimize hypersensitivity reactions. In a rabbit embryo-fetal development study, cipaglucosidase alfa-atga (30, 70, or 175 mg/kg) was administered intravenously every other day to pregnant females during organogenesis (Gestation Day [GD] 7 through GD 19). Additional experimental groups received 25 mg/kg oral miglustat alone, or in combination with intravenous cipaglucosidase alfa-atga 175 mg/kg, with the same dosing frequency during organogenesis.
Clusters of great vessel and cardiac malformations were increased in offspring of pregnant rabbits treated with the combination of cipaglucosidase alfa-atga and miglustat at 16-fold and 3-fold the MRHD of POMBILITI and Opfolda, respectively, based on plasma AUC exposure. A NOAEL for the combination was not identified. One fetus treated with cipaglucosidase alfa-atga alone (175 mg/kg) and one fetus treated with miglustat alone (25 mg/kg), each showed a similar cluster of these great vessel and cardiac malformations.
In a rat embryo-fetal development study, cipaglucosidase alfa-atga (75, 150, or 400 mg/kg) was administered intravenously every other day to pregnant rats during organogenesis (GD 6 through GD 18). Additional experimental groups received 60 mg/kg oral miglustat alone, or in combination with intravenous cipaglucosidase alfa-atga 400 mg/kg, with the same dosing frequency during organogenesis. No evidence of adverse effects was noted in pregnant rats or their offspring in any experimental group.
The margin at the NOAEL for cipaglucosidase alfa‑atga (400 mg/kg) was 20-fold the POMBILITI MRHD based on plasma AUC exposure. The margin at the NOAEL for miglustat (60 mg/kg) was 4-fold the Opfolda MRHD based on plasma AUC exposure. In a pre-and post-natal development study in rats, cipaglucosidase alfa-atga (75, 150, or 400 mg/kg) was administered intravenously every other day to pregnant females from GD 6 through GD 18, and from Lactation Day (LD) 1 through LD 19.
Additional experimental groups received 60 mg/kg oral miglustat alone, or in combination with intravenous cipaglucosidase alfa‑atga 400 mg/kg, with the same dosing frequency during pregnancy and lactation. Maternal and pup mortality were increased with the combination, and pup mortality was also increased with cipaglucosidase alfa-atga 400 mg/kg alone. The NOAEL for cipaglucosidase alfa-atga alon… [Excerpted — this section continues on DailyMed.]
🤰 Pregnancy ▾
8.1Pregnancy Risk Summary Based on findings from animal reproduction studies, POMBILITI in combination with Opfolda may cause embryo-fetal harm when administered to a pregnant female and is contraindicated during pregnancy. In a rabbit embryo-fetal development study, great vessel and cardiac malformations were increased in offspring of pregnant rabbits treated with cipaglucosidase alfa-atga in combination with miglustat at 16-fold and 3-fold, respectively, the MRHD of POMBILITI and Opfolda based on plasma AUC exposure.
A No Observed Adverse Effect Level (NOAEL) was not identified for the combination. In a pre- and post-natal development study in rats, increases in pup mortality were seen following maternal treatment with cipaglucosidase alfa-atga (400 mg/kg) in combination with miglustat, or with cipaglucosidase alfa-atga (400 mg/kg) alone. The NOAEL for cipaglucosidase alfa-atga alone is 150 mg/kg (5-fold the POMBILITI MRHD margin).
A NOAEL for the combination was not identified. Margins at the lowest observed adverse effect level (LOAEL), relative to exposures at the MRHD of POMBILITI and Opfolda were 20-fold and 4-fold, respectively, based on plasma AUC exposure (see Data ) . There are no available human data on POMBILITI in combination with Opfolda use in pregnant females to evaluate for a drug-associated risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes.
Data Animal Data Reproductive toxicity studies of cipaglucosidase alfa-atga in rats and rabbits included pretreatment with diphenhydramine (DPH) to prevent or minimize hypersensitivity reactions. In a rabbit embryo-fetal development study, cipaglucosidase alfa-atga (30, 70, or 175 mg/kg) was administered intravenously every other day to pregnant females during organogenesis (Gestation Day [GD] 7 through GD 19). Additional experimental groups received 25 mg/kg oral miglustat alone, or in combination with intravenous cipaglucosidase alfa-atga 175 mg/kg, with the same dosing frequency during organogenesis.
Clusters of great vessel and cardiac malformations were increased in offspring of pregnant rabbits treated with the combination of cipaglucosidase alfa-atga and miglustat at 16-fold and 3-fold the MRHD of POMBILITI and Opfolda, respectively, based on plasma AUC exposure. A NOAEL for the combination was not identified. One fetus treated with cipaglucosidase alfa-atga alone (175 mg/kg) and one fetus treated with miglustat alone (25 mg/kg), each showed a similar cluster of these great vessel and cardiac malformations.
In a rat embryo-fetal development study, cipaglucosidase alfa-atga (75, 150, or 400 mg/kg) was administered intravenously every other day to pregnant rats during organogenesis (GD 6 through GD 18). Additional experimental groups received 60 mg/kg oral miglustat alone, or in combination with intravenous cipaglucosidase alfa-atga 400 mg/kg, with the same dosing frequency during organogenesis. No evidence of adverse effects was noted in pregnant rats or their offspring in any experimental group.
The margin at the NOAEL for cipaglucosidase alfa‑atga (400 mg/kg) was 20-fold the POMBILITI MRHD based on plasma AUC exposure. The margin at the NOAEL for miglustat (60 mg/kg) was 4-fold the Opfolda MRHD based on plasma AUC exposure. In a pre-and post-natal development study in rats, cipaglucosidase alfa-atga (75, 150, or 400 mg/kg) was administered intravenously every other day to pregnant females from GD 6 through GD 18, and from Lactation Day (LD) 1 through LD 19.
Additional experimental groups received 60 mg/kg oral miglustat alone, or in combination with intravenous cipaglucosidase alfa‑atga 400 mg/kg, with the same dosing frequency during pregnancy and lactation. Maternal and pup mortality were increased with the combination, and pup mortality was also increased with cipaglucosidase alfa-atga 400 mg/kg alone. The NOAEL for cipaglucosidase alfa-atga alone is 150 mg/kg (5-fold the POMBILITI MRHD margin).
A NOAEL was not identified fo… [Excerpted — this section continues on DailyMed.]
🧒 Pediatric Use ▾
8.4Pediatric Use Safety and effectiveness of POMBILITI in combination with Opfolda have not been established in pediatric patients with late-onset Pompe disease.
🧓 Geriatric Use ▾
8.5Geriatric Use Of the total number of patients treated with POMBILITI in combination with Opfolda in clinical trials for LOPD, 17 (11%) were 65 to 74 years of age, and none were 75 years of age and older [see Clinical Studies (14) ] . Clinical trials of POMBILITI in combination with Opfolda did not include sufficient numbers of patients 65 years of age and older to determine whether they respond differently from younger adult patients.
🧬 Clinical Pharmacology ▾
12 CLINICAL PHARMACOLOGY
12.1Mechanism of Action Pompe disease (also known as glycogen storage disease type II, acid maltase deficiency, and glycogenosis type II) is an inherited disorder of glycogen metabolism caused by a deficiency of lysosomal acid alpha‑glucosidase (GAA) that degrades glycogen to glucose in the lysosome. GAA deficiency results in intra-lysosomal accumulation of glycogen in various tissues. Cipaglucosidase alfa-atga provides an exogenous source of GAA.
The bis-M6P on cipaglucosidase alfa-atga mediates binding to M6P receptors on the cell surface with high affinity. After binding, it is internalized and transported into lysosomes where it undergoes proteolytic cleavage and N‑glycans trimming which are both required to yield the most mature and active form of GAA. Cipaglucosidase alfa-atga then exerts enzymatic activity in cleaving glycogen.
Miglustat binds with, stabilizes, and reduces inactivation of cipaglucosidase alfa-atga in the blood after infusion.
12.2Pharmacodynamics In patients with LOPD, excess of glycogen is degraded to hexose tetrasaccharide (Hex4) which is then excreted in urine. The urinary Hex4 assay measures its major component, glucose tetrasaccharide (Glc4). Treatment with POMBILITI in combination with Opfolda resulted in reductions of urinary Glc4 concentrations (normalized by urine creatinine and reported as mmol Glc4/mol creatinine) in adult patients with LOPD.
In ERT‑experienced patients in Trial 1 [see Clinical Studies (14) ] , the baseline mean urinary Glc4 concentration was 4.6 mmol/mol and 7.2 mmol/mol in the POMBILITI in combination with Opfolda and the non-U.S.-approved alglucosidase alfa product with placebo groups, respectively. The mean percentage (SD) change in urinary Glc4 concentration from baseline to Week 52 was ‑29% (32) and +20% (32) in the POMBILITI in combination with Opfolda and the non-U.S.-approved alglucosidase alfa product with placebo groups, respectively.
12.3Pharmacokinetics The maximum plasma concentration (C max ) and area under the plasma concentration-time curve (AUC) of cipaglucosidase alfa-atga following administration with 20 mg/kg of cipaglucosidase alfa-atga in combination with a single oral dose of 260 mg of Opfolda in adult patients with LOPD are summarized in Table 3 . Table 3. Geometric Mean (CV%) Pharmacokinetic Parameters of Cipaglucosidase alfa‑atga in ERT‑experienced Adult Patients with LOPD CV: coefficient of variation; ERT: enzyme replacement therapy; LOPD: late-onset Pompe disease; PK: pharmacokinetic parameters; C max : maximum plasma concentration; AUC: plasma concentration-time curve † POMBILITI in combination with Opfolda is not approved for use without Opfolda [see Indications and Usage (1) ] .
PK Parameter Cipaglucosidase alfa-atga 20 mg/kg in Combination with Opfolda 260 mg Cipaglucosidase alfa-atga 20 mg/kg † C max (mcg/mL) 345 (18.5) 325 (13.5) AUC 0-inf (mcg*h/mL) 1812 (20.8) 1410 (15.9) Distribution The volume of distribution of cipaglucosidase alfa-atga ranged from 2.0 to
4.7L. Elimination When cipaglucosidase alfa-atga was administered with Opfolda, the mean total body clearance (CL) of cipaglucosidase alfa-atga was
0.8L/hour and the mean half-life of cipaglucosidase alfa-atga was 2.1 hours in ERT‑experienced patients. Metabolism The metabolic pathway of cipaglucosidase alfa-atga has not been characterized. Cipaglucosidase alfa-atga is expected to be metabolized into small peptides and amino acid via catabolic pathways. Specific Populations Age (18 to 74 years) and sex did not have clinically meaningful effects on the pharmacokinetics of cipaglucosidase alfa-atga.
12.6Immunogenicity The observed incidence of antidrug antibodies is highly dependent on the sensitivity and specificity of the assay. Differences in assay methods preclude meaningful comparisons of the incidence of antidrug antibodies (ADA) in the trials described below with the incidence of antidrug antibodies in other trials, including those of cipaglucosidase… [Excerpted — this section continues on DailyMed.]
🧬 Mechanism of Action ▾
12.1Mechanism of Action Pompe disease (also known as glycogen storage disease type II, acid maltase deficiency, and glycogenosis type II) is an inherited disorder of glycogen metabolism caused by a deficiency of lysosomal acid alpha‑glucosidase (GAA) that degrades glycogen to glucose in the lysosome. GAA deficiency results in intra-lysosomal accumulation of glycogen in various tissues. Cipaglucosidase alfa-atga provides an exogenous source of GAA.
The bis-M6P on cipaglucosidase alfa-atga mediates binding to M6P receptors on the cell surface with high affinity. After binding, it is internalized and transported into lysosomes where it undergoes proteolytic cleavage and N‑glycans trimming which are both required to yield the most mature and active form of GAA. Cipaglucosidase alfa-atga then exerts enzymatic activity in cleaving glycogen.
Miglustat binds with, stabilizes, and reduces inactivation of cipaglucosidase alfa-atga in the blood after infusion.
📦 How Supplied / Storage and Handling ▾
16 HOW SUPPLIED/STORAGE AND HANDLING How Supplied POMBILITI (cipaglucosidase alfa-atga) for injection is supplied as a sterile, white to slightly yellowish lyophilized powder with a cake-like appearance in a single‑dose vial. POMBILITI does not contain any preservatives. See Table 7 for the available POMBILITI packages.
Table 7. POMBILITI Cartons Carton NDC Contains one (1) 105 mg single-dose vial 71904‑200‑01 Contains ten (10) 105 mg single-dose vials 71904‑200‑02 Contains twenty-five (25) 105 mg single-dose vials 71904‑200‑03 Storage and Handling Store refrigerated at 2℃ to 8℃ (36℉ to 46℉) in the original carton to protect from light. Do not freeze.
For storage of the reconstituted solution and diluted solution [see Dosage and Administration (2.5) ] .
📋 Description ▾
11 DESCRIPTION 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.
💬 Information for Patients ▾
17 PATIENT COUNSELING INFORMATION POMBILITI must be administered in combination with Opfolda. Refer to the Opfolda Prescribing Information for Opfolda patient counseling information. Administration Advise the patient and caregiver to follow the timeline recommendations for taking Opfolda prior to the intravenous infusion with POMBILITI [see Dosage and Administration (2.2) ] .
Hypersensitivity Reactions (Including Anaphylaxis) and Infusion-Associated Reactions (IARs) Advise the patient and caregiver that reactions related to the infusion may occur during and after POMBILITI in combination with Opfolda treatment, including life-threatening hypersensitivity reactions, including anaphylaxis, and IARs. Inform the patient and caregiver of the signs and symptoms of hypersensitivity reactions and IARs and to seek medical care should signs and symptoms occur [see Warnings and Precaution (5.1 , 5.2) ] .
Risk of Acute Cardiorespiratory Failure Advise the patient and caregiver that a patient with underlying respiratory illness or compromised cardiac or respiratory function may be at risk of acute cardiorespiratory failure from volume overload during POMBILITI infusion and to seek medical care should signs and symptoms occur [see Warnings and Precaution (5.3) ] . Embryo-Fetal Toxicity POMBILITI in combination with Opfolda may cause embryo-fetal harm. Advise a female patient and caregiver to inform their healthcare provider of a known or suspected pregnancy [see Warnings and Precautions (5.4) and Use in Specific Populations (8.1) ] .
Advise a female of reproductive potential to use effective contraception during treatment with POMBILITI in combination with Opfolda and for at least 60 days after the last dose [see Use in Specific Populations (8.1 , 8.3) ] . Lactation Advise a lactating female not to breastfeed during treatment with POMBILITI in combination with Opfolda [see Use in Specific Populations (8.2) ] . Infertility Advise the male or female of reproductive potential that POMBILITI in combination with Opfolda may impair fertility [see Use in Specific Populations (8.3) ] .
Manufactured by: Amicus Therapeutics US, LLC 3675 Market Street Philadelphia, PA 19104 U.S. License No. 2224 POMBILITI and Opfolda are registered trademarks of Amicus Therapeutics, Inc.
For more information, go to pombilitiopfoldahcp.com.
🧬 Pharmacokinetics ▾
12.3Pharmacokinetics The maximum plasma concentration (C max ) and area under the plasma concentration-time curve (AUC) of cipaglucosidase alfa-atga following administration with 20 mg/kg of cipaglucosidase alfa-atga in combination with a single oral dose of 260 mg of Opfolda in adult patients with LOPD are summarized in Table 3 . Table 3. Geometric Mean (CV%) Pharmacokinetic Parameters of Cipaglucosidase alfa‑atga in ERT‑experienced Adult Patients with LOPD CV: coefficient of variation; ERT: enzyme replacement therapy; LOPD: late-onset Pompe disease; PK: pharmacokinetic parameters; C max : maximum plasma concentration; AUC: plasma concentration-time curve † POMBILITI in combination with Opfolda is not approved for use without Opfolda [see Indications and Usage (1) ] .
PK Parameter Cipaglucosidase alfa-atga 20 mg/kg in Combination with Opfolda 260 mg Cipaglucosidase alfa-atga 20 mg/kg † C max (mcg/mL) 345 (18.5) 325 (13.5) AUC 0-inf (mcg*h/mL) 1812 (20.8) 1410 (15.9) Distribution The volume of distribution of cipaglucosidase alfa-atga ranged from 2.0 to
4.7L. Elimination When cipaglucosidase alfa-atga was administered with Opfolda, the mean total body clearance (CL) of cipaglucosidase alfa-atga was
0.8L/hour and the mean half-life of cipaglucosidase alfa-atga was 2.1 hours in ERT‑experienced patients. Metabolism The metabolic pathway of cipaglucosidase alfa-atga has not been characterized. Cipaglucosidase alfa-atga is expected to be metabolized into small peptides and amino acid via catabolic pathways. Specific Populations Age (18 to 74 years) and sex did not have clinically meaningful effects on the pharmacokinetics of cipaglucosidase alfa-atga.
🧬 Pharmacodynamics ▾
12.2Pharmacodynamics In patients with LOPD, excess of glycogen is degraded to hexose tetrasaccharide (Hex4) which is then excreted in urine. The urinary Hex4 assay measures its major component, glucose tetrasaccharide (Glc4). Treatment with POMBILITI in combination with Opfolda resulted in reductions of urinary Glc4 concentrations (normalized by urine creatinine and reported as mmol Glc4/mol creatinine) in adult patients with LOPD.
In ERT‑experienced patients in Trial 1 [see Clinical Studies (14) ] , the baseline mean urinary Glc4 concentration was 4.6 mmol/mol and 7.2 mmol/mol in the POMBILITI in combination with Opfolda and the non-U.S.-approved alglucosidase alfa product with placebo groups, respectively. The mean percentage (SD) change in urinary Glc4 concentration from baseline to Week 52 was ‑29% (32) and +20% (32) in the POMBILITI in combination with Opfolda and the non-U.S.-approved alglucosidase alfa product with placebo groups, respectively.
🔬 Clinical Studies ▾
14 CLINICAL 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 POMBILITI (20 mg/kg by intravenous infusion) in combination with Opfolda (260 mg orally for those ≥50 kg or 195 mg orally for those ≥40 kg to <50 kg) 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 Table 5 for baseline sitting FVC [percent predicted] values). Of the 123 randomized patients, 56 were males, baseline mean age was 47 years old (range from 19 to 74 years old), and mean age at diagnosis was 39 years old (range from 1 to 66).
The racial groups for the patients consisted of 104 White (85%), 6 Japanese (5%), 6 Other racial group (5%), 4 Asian (3%), 1 Native Hawaiian or other Pacific Islander (1%), 1 American Indian or Alaska Native (1%), and 1 Black or African American (1%). Key efficacy endpoints included assessment of sitting FVC (% predicted) and 6MWD ( Table 5 and Table 6 ). Sitting FVC (Percent‑predicted) at 52 Weeks Patients treated with POMBILITI in combination with Opfolda 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 POMBILITI in combination with Opfolda showed a numerically favorable change in sitting FVC from baseline at Week 52 ( Table 5 and Figure 2 ). Table 5. Summary of Sitting FVC in Adults with LOPD by ERT Status at 52 Weeks in Trial 1 FVC: forced vital capacity; LOPD: late-onset Pompe disease; ERT: enzyme replacement therapy; SD: standard deviation; Diff.: difference; SE: standard error; CI: confidence interval ∗ POMBILITI in combination with Opfolda is not approved for use in ERT-naïve patients with LOPD [see Indications and Usage (1) ] .
The ERT-naïve patient subgroup enrolled too few patients to conclusively interpret the data. For the ERT-naïve group, the treatment difference was estimated using a 2-sample t-test. † A U.S.-approved alglucosidase alfa product was not used in this clinical trial. Conclusions cannot be drawn from this clinical trial regarding comparative effectiveness between a U.S.-approved alglucosidase alfa product and POMBILITI in combination with Opfolda for the treatment of adult patients with LOPD weighing ≥40 kg and who are not improving on their current ERT. ‡ For the ERT‑experienced group, the treatment difference of the mean was estimated by analysis of covariance which included treatment, gender, baseline FVC, age, weight, and height in the model.
Nominal p=0.006. Missing data at Week 52 was imputed using last observed values. Efficacy Endpoint ERT-experienced ERT-naïve* Sitting FVC (% predicted) POMBILITI in Combination with Opfolda A Non-U.S.-Approved Alglucosidase alfa Product † with Placebo POMBILITI in Combination with Opfolda A Non-U.S.-Approved Alglucosidase alfa Product † with Placebo Baseline n Mean (SD) Median n=65 67.9 (19.1) 68.0 n=30 67.5 (21.0) 69.0 n=20 80.2 (18.7) 82.3 n=8 79.6 (21.0)
88.5Change from baseline at Week 52 n Mean (SD) Median n=55 0.1 (5.9) 0.5 n=26 -3.5 (4.7) -2.5 n=19 -4.7 (6.2) -4.5 n=7 -2.4 (6.3) -3.0 Change to Week 52 Diff. of means (SE) (95% CI) 3.5 (1.3) (1.0, 6.0) ‡ -1.9 (2.7) (-7.3,… [Excerpted — this section continues on DailyMed.]
🧪 Nonclinical Toxicology ▾
13 NONCLINICAL TOXICOLOGY
13.1Carcinogenesis, Mutagenesis, Impairment of Fertility Carcinogenesis Studies in animals to evaluate the carcinogenic potential of cipaglucosidase alfa-atga have not been conducted. For information on an evaluation of carcinogenicity of miglustat in animals, see the Opfolda Prescribing Information. Mutagenesis Studies to evaluate the mutagenic or genotoxic potential of cipaglucosidase alfa-atga have not been conducted.
For information regarding the mutagenesis of miglustat, see the Opfolda Prescribing Information. Impairment of Fertility In a fertility study in rats, cipaglucosidase alfa-atga (75, 150, or 400 mg/kg) was administered intravenously to male and female rats every other day. Dosing in males was initiated 28 days prior to cohabitation with untreated females.
Dosing in females was initiated 14 days prior to cohabitation with untreated males and continued through GD 7. Additional experimental groups received 60 mg/kg oral miglustat alone, or in combination with intravenous cipaglucosidase alfa‑atga 400 mg/kg, with the same frequency over the same pre‑mating interval (males) or pre-mating and pregnancy interval (females). There was no effect on male or female rat fertility in any experimental group.
Treatment of male rats with the combination was associated with increased preimplantation loss that was reversible. Treatment of female rats with the combination, or with miglustat alone, resulted in preimplantation loss; whether this would be reversible if treatment were discontinued prior to cohabitation is unknown. NOAELs were not identified for the combination in either male or female rats.
The LOAEL margins for these doses represent 27-fold and 4-fold the MRHD of POMBILITI and Opfolda, respectively, based on plasma AUC exposure.
13.2Animal Toxicology and/or Pharmacology For additional animal toxicology findings for miglustat, see the Opfolda Prescribing Information.
📄 Carcinogenesis, Mutagenesis, Impairment of Fertility ▾
13.1Carcinogenesis, Mutagenesis, Impairment of Fertility Carcinogenesis Studies in animals to evaluate the carcinogenic potential of cipaglucosidase alfa-atga have not been conducted. For information on an evaluation of carcinogenicity of miglustat in animals, see the Opfolda Prescribing Information. Mutagenesis Studies to evaluate the mutagenic or genotoxic potential of cipaglucosidase alfa-atga have not been conducted.
For information regarding the mutagenesis of miglustat, see the Opfolda Prescribing Information. Impairment of Fertility In a fertility study in rats, cipaglucosidase alfa-atga (75, 150, or 400 mg/kg) was administered intravenously to male and female rats every other day. Dosing in males was initiated 28 days prior to cohabitation with untreated females.
Dosing in females was initiated 14 days prior to cohabitation with untreated males and continued through GD 7. Additional experimental groups received 60 mg/kg oral miglustat alone, or in combination with intravenous cipaglucosidase alfa‑atga 400 mg/kg, with the same frequency over the same pre‑mating interval (males) or pre-mating and pregnancy interval (females). There was no effect on male or female rat fertility in any experimental group.
Treatment of male rats with the combination was associated with increased preimplantation loss that was reversible. Treatment of female rats with the combination, or with miglustat alone, resulted in preimplantation loss; whether this would be reversible if treatment were discontinued prior to cohabitation is unknown. NOAELs were not identified for the combination in either male or female rats.
The LOAEL margins for these doses represent 27-fold and 4-fold the MRHD of POMBILITI and Opfolda, respectively, based on plasma AUC exposure.
📄 Package Label / Principal Display Panel ▾
PRINCIPAL DISPLAY PANEL - NDC: 71904-200-01 - 105 mg/vial 1 Count Vial Label 1 Count Carton Label
PRINCIPAL DISPLAY PANEL - NDC: 71904-200-01 - 105 mg/vial 1 Count Carton Label 1 Count Vial Label
PRINCIPAL DISPLAY PANEL - NDC: 71904-200-02 - 105 mg/vial 10 Count Multipack Label 10 Count Multipack Label
PRINCIPAL DISPLAY PANEL - NDC: 71904-200-03 - 105 mg/vial 25 Count Multipack Label 25 Count Multipack Label
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