Cipaglucosidase alfa-atga

Para qué se usa Cipaglucosidase alfa-atga

Cita de la sección «Indications and Usage» (indicaciones y uso) de la etiqueta aprobada por la FDA. Se muestra en inglés, tal como la publica la FDA.

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). …

— Etiqueta de la FDA: Pombiliti Atga, Amicus Therapeutics Us, LLC, vigente desde el 12 de agosto de 2026. Leer la etiqueta completa en DailyMed →

Etiqueta citada: la de la presentación más común (injection, powder, lyophilized, for solution, intravenous), Pombiliti Atga, Amicus Therapeutics Us, LLC; abarca 1 producto del directorio NDC de la FDA. Las citas sobre efectos secundarios, interacciones, dosis y embarazo de abajo vienen de esta misma etiqueta.

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Cipaglucosidase alfa-atga: lo que dice la advertencia en recuadro

Cita de la advertencia en recuadro (Boxed Warning) de la etiqueta aprobada por la FDA. Se muestra en inglés.

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.

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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)

— Etiqueta de la FDA: Pombiliti Atga, Amicus Therapeutics Us, LLC, vigente desde el 12 de agosto de 2026. Leer la etiqueta completa en DailyMed →

Efectos secundarios de Cipaglucosidase alfa-atga

Cita de la sección «Adverse Reactions» (reacciones adversas) de la etiqueta aprobada por la FDA. Se muestra en inglés. Avise a su médico de cualquier efecto secundario que le preocupe.

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.

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(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 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)].

— Etiqueta de la FDA: Pombiliti Atga, Amicus Therapeutics Us, LLC, vigente desde el 12 de agosto de 2026. Esto es solo una parte de la sección. Leer la etiqueta completa en DailyMed →

Dosis de Cipaglucosidase alfa-atga

Cita de la sección «Dosage and Administration» (dosis y administración) de la etiqueta aprobada por la FDA. Se muestra en inglés. Su dosis es la que le receta su médico: no la cambie por su cuenta.

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.

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(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) 2.1 Pregnancy 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)]. 2.2 Recommended 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 2.3 Dosage 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)].

— Etiqueta de la FDA: Pombiliti Atga, Amicus Therapeutics Us, LLC, vigente desde el 12 de agosto de 2026. Esto es solo una parte de la sección. Leer la etiqueta completa en DailyMed →

Cipaglucosidase alfa-atga en el embarazo

Cita de la parte sobre embarazo (Pregnancy) de la etiqueta aprobada por la FDA. Se muestra en inglés. Pregunte a su médico antes de tomar cualquier medicamento si está embarazada o planea estarlo.

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.

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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.

— Etiqueta de la FDA: Pombiliti Atga, Amicus Therapeutics Us, LLC, vigente desde el 12 de agosto de 2026. Esto es solo una parte de la sección. Leer la etiqueta completa en DailyMed →

Cuándo no se debe usar Cipaglucosidase alfa-atga

Cita de la sección «Contraindications» (contraindicaciones) de la etiqueta aprobada por la FDA. Se muestra en inglés.

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)

— Etiqueta de la FDA: Pombiliti Atga, Amicus Therapeutics Us, LLC, vigente desde el 12 de agosto de 2026. Leer la etiqueta completa en DailyMed →

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• Amicus Therapeutics US, LLC1 producto

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Fuentes

1. Etiqueta aprobada por la FDA en DailyMed (Biblioteca Nacional de Medicina) — Pombiliti Atga, Amicus Therapeutics Us, LLC, vigente desde el 12 de agosto de 2026; vía openFDA, datos de la FDA exportados el 30 de octubre de 2026, consultados el 31 de octubre de 2026
2. NDC Directory (openFDA) — Administración de Alimentos y Medicamentos de los EE. UU. (FDA), datos de la FDA exportados el 30 de octubre de 2026, consultados el 31 de octubre de 2026
3. Drugs@FDA (openFDA) — Administración de Alimentos y Medicamentos de los EE. UU. (FDA), datos de la FDA exportados el 30 de octubre de 2026, consultados el 31 de octubre de 2026
4. Drug recall enforcement reports (openFDA) — Administración de Alimentos y Medicamentos de los EE. UU. (FDA), datos de la FDA exportados el 27 de octubre de 2026, consultados el 31 de octubre de 2026
5. Drug shortages (openFDA) — Administración de Alimentos y Medicamentos de los EE. UU. (FDA), datos de la FDA exportados el 30 de octubre de 2026, consultados el 31 de octubre de 2026

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