Respuestas rápidas
- ¿Para qué se usa Olipudase alfa-rpcp?
- La etiqueta aprobada por la FDA (Xenpozyme, Genzyme Corporation, 11 de diciembre de 2025) dice, en inglés: “XENPOZYME is indicated for treatment of non–central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients. XENPOZYME is a hydrolytic lysosomal sphingomyelin-specific enzyme indicated for treatment of non–central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients.” Esta página no es un consejo médico. Hable con su médico o farmacéutico antes de empezar, dejar o cambiar cualquier medicamento.
- ¿Cuáles son los efectos secundarios de Olipudase alfa-rpcp?
- La sección «Adverse Reactions» (reacciones adversas) de la etiqueta de la FDA (Xenpozyme, Genzyme Corporation, 11 de diciembre de 2025) comienza así, en inglés: «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 (IARs) [see Warnings and Precautions (5.2)] Elevated Transaminase Levels [see Warnings and Precautions (5.3)] Most common adverse reactions in adult patients (incidence ≥10%) are headache, cough, diarrhea, hypotension, and ocular hyperemia.» … Esta página no es un consejo médico. Hable con su médico o farmacéutico antes de empezar, dejar o cambiar cualquier medicamento.
- ¿Olipudase alfa-rpcp tiene una advertencia en recuadro?
- Sí. La etiqueta de la FDA de Xenpozyme tiene una advertencia en recuadro («WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS»), la advertencia más seria de la FDA. Léala en la etiqueta completa y pregunte a su farmacéutico.
- ¿Hay escasez de Olipudase alfa-rpcp?
- La FDA no indica una escasez actual para los productos de este medicamento (datos de la FDA del 30 de octubre de 2026).
- ¿Se ha retirado del mercado Olipudase alfa-rpcp?
- No aparece ningún retiro vinculado a los productos de este medicamento en los informes de la FDA que usamos.
- ¿Quién fabrica Olipudase alfa-rpcp?
- 1 empresa tiene productos con receta de Olipudase alfa-rpcp en el directorio NDC de la FDA, entre ellas Genzyme Corporation.
Para qué se usa Olipudase alfa-rpcp
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.
XENPOZYME is indicated for treatment of non–central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients. XENPOZYME is a hydrolytic lysosomal sphingomyelin-specific enzyme indicated for treatment of non–central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients.
— Etiqueta de la FDA: Xenpozyme, Genzyme Corporation, vigente desde el 11 de diciembre de 2025. Leer la etiqueta completa en DailyMed →
Etiqueta citada: la de la presentación más común (injection, powder, lyophilized, for solution, intravenous), Xenpozyme, Genzyme Corporation; abarca 2 productos del directorio NDC de la FDA. Las citas sobre efectos secundarios, interacciones, dosis y embarazo de abajo vienen de esta misma etiqueta.
Esta página no es un consejo médico. Hable con su médico o farmacéutico antes de empezar, dejar o cambiar cualquier medicamento.
Advertencia en recuadro
La advertencia en recuadro («caja negra») es la advertencia más seria de la FDA en la etiqueta de un medicamento, para riesgos graves o mortales.
Esta etiqueta tiene una advertencia en recuadro. Su título: WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS
. Leerla en la etiqueta completa →
Olipudase alfa-rpcp: 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: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS Patients treated with XENPOZYME have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Appropriate medical monitoring and support measures, including cardiopulmonary resuscitation equipment, should be readily available during XENPOZYME administration.
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If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue XENPOZYME immediately and initiate appropriate medical treatment. In patients with severe hypersensitivity reactions, a desensitization procedure to XENPOZYME may be considered [see Warnings and Precautions (5.1)]. WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS See full prescribing information for complete boxed warning. Appropriate medical monitoring and support measures, including cardiopulmonary resuscitation equipment, should be readily available. If a severe hypersensitivity reaction occurs, discontinue XENPOZYME immediately and initiate appropriate medical treatment. (5.1)
— Etiqueta de la FDA: Xenpozyme, Genzyme Corporation, vigente desde el 11 de diciembre de 2025. Leer la etiqueta completa en DailyMed →
Efectos secundarios de Olipudase alfa-rpcp
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 (IARs) [see Warnings and Precautions (5.2)] Elevated Transaminase Levels [see Warnings and Precautions (5.3)] Most common adverse reactions in adult patients (incidence ≥10%) are headache, cough, diarrhea, hypotension, and ocular hyperemia.
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(6.1) Most common adverse reactions in pediatric patients (incidence ≥20%) are pyrexia, cough, diarrhea, rhinitis, abdominal pain, vomiting, headache, urticaria, nausea, rash, arthralgia, pruritus, fatigue, and pharyngitis. (6.1) To report SUSPECTED ADVERSE REACTIONS, contact Genzyme Corporation at 1-800-745-4447 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. The pooled safety analysis from 3 clinical trials included a total of 38 XENPOZYME-treated patients (30 adult and 8 pediatric patients) with age range from 1.5 to 59 years old receiving intravenous doses up to 3 mg/kg every 2 weeks [see Clinical Studies (14)]. The median exposure duration was 2.5 years (range: 0.4 to 3.7 years) in adult patients and 2.7 years (range: 2.5 to 3.2 years) in pediatric patients. Serious adverse reactions of anaphylactic reaction were reported in 2 (25%) XENPOZYME-treated pediatric patients. Most frequently reported adverse drug reactions in adults (incidence ≥10%) were headache, cough, diarrhea, hypotension, and ocular hyperemia. Most frequently reported adverse drug reactions in pediatric patients (incidence ≥20%) were pyrexia, cough, diarrhea, rhinitis, abdominal pain, vomiting, headache, urticaria, nausea, rash, arthralgia, pruritus, fatigue, and pharyngitis. Adult patients with ASMD type B and type A/B (Trial 1) In Trial 1, 13 adult patients received XENPOZYME once every 2 weeks for 52 weeks (primary analysis period (PAP)) at dosages escalating from 0.1 mg/kg to a target dose of 3 mg/kg [see Clinical Studies (14.2)]. Adverse reactions that occurred in at least 7% of XENPOZYME-treated adult patients during the PAP are described in Table 7. Table 7: Adverse Reactions Occurring at >7% in Adult Patients with ASMD During the 52-Week Primary Analysis Period in Trial 1 Adverse Reaction XENPOZYME N=13 Placebo N=18 Headache 7 (54%) 8 (44%) Cough 4 (31%) 2 (11%) Diarrhea 2 (15%) 2 (11%) Hypotension 2 (15%) 2 (11%) Ocular hyperemia 2 (15%) 1 (6%) Erythema 1 (8%) 1 (6%) Asthenia 1 (8%) 1 (6%) Pharyngitis 1 (8%) 1 (6%) Dyspnea 1 (8%) 0 Urticaria 1 (8%) 0 Papule 1 (8%) 0 Myalgia 1 (8%) 0 Throat irritation 1 (8%) 0 C-reactive protein abnormal 1 (8%) 0 Pediatric Patients with ASMD type B and type A/B (Trial 2 and Trial 3) In Trial 2, 8 pediatric patients less than or equal to 17 years of age received XENPOZYME intravenously once every 2 weeks for 64 weeks [see Clinical Studies (14.3)]. After 64 weeks, all pediatric patients entered into Trial 3.
— Etiqueta de la FDA: Xenpozyme, Genzyme Corporation, vigente desde el 11 de diciembre de 2025. Esto es solo una parte de la sección. Leer la etiqueta completa en DailyMed →
Dosis de Olipudase alfa-rpcp
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.
See Full Prescribing Information for important recommendations prior to XENPOZYME treatment initiation. (2.1) Adults: Recommended starting dose is 0.1 mg/kg administered as an intravenous infusion. (2.2) Pediatrics: Recommended starting dose is 0.03 mg/kg administered as an intravenous infusion.
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(2.3) See Full Prescribing Information for the recommended dose escalation and maintenance dosage, dosage modifications to reduce the risk of adverse reactions, and preparation and administration instructions. (2.2, 2.3, 2.5, 2.6, 2.7) 2.1 Important Recommendations Prior to XENPOZYME Treatment Initiation Therapy with XENPOZYME should be directed in consultation with physicians knowledgeable in the management of ASMD. In order to avoid dosing errors including overdosage [see Overdosage (10)], follow all instructions for dosage and administration. Laboratory Testing Before initiating XENPOZYME: Obtain baseline transaminase (alanine aminotransferase [ALT] and aspartate aminotransferase [AST]) levels in all patients within 1 month prior to treatment initiation [see Warnings and Precautions (5.3)]. Verify pregnancy status in females of reproductive potential [see Use in Specific Populations (8.1, 8.3)]. Premedication Prior to XENPOZYME administration, consider premedicating with antihistamines, antipyretics, and/or corticosteroids [see Warnings and Precautions (5.1, 5.2)]. Medical Support Appropriate medical support measures including cardiopulmonary resuscitation equipment should be readily available during XENPOZYME administration [see Warnings and Precautions (5.1)]. Weight-Based Dosing Information The recommended adult and pediatric dosages of XENPOZYME for the dose escalation and maintenance phases [see Dosage and Administration (2.2, 2.3)] are based on body weight as follows for patients with a body mass index (BMI): Less than or equal to 30, the dosage is based on actual body weight (kg) Greater than 30, the dosage is based on adjusted body weight (kg). Calculate an adjusted body weight (kg) based on height in meters as described below: Adjusted body weight (kg) = (actual height in m) 2 × 30 2.2 Recommended Dosage in Adult Patients Dose Escalation Phase The recommended starting dose of XENPOZYME in adults is 0.1 mg/kg. In order to reduce the risk of infusion-associated reactions or elevated transaminase levels, follow the dose escalation regimen in Table 1 [see Warnings and Precautions (5.1, 5.2, 5.3)]. Administer XENPOZYME via intravenous infusion every 2 weeks. Table 1: XENPOZYME Dose Escalation Regimen for Adult Patients Use actual body weight for patients with a BMI less than or equal to 30. For patients with a BMI greater than 30, calculate adjusted body weight (kg) = (actual height in m) 2 × 30 [see Dosage and Administration (2.1)].
Tabla de la etiqueta
| Consecutive Missed Doses In: | Escalation Phase | Maintenance Phase |
|---|---|---|
| 1 missed dose | First dose after a missed dose: Administer last tolerated doseSecond and subsequent doses after missed dose: Resume dose escalation at next infusion according to Table 1 for adult patients or Table 2 for pediatric patients | First and subsequent doses after missed dose: Administer maintenance dose |
| 2 consecutive missed doses | First dose after missed dose: Administer 1 dose below last tolerated dose Second and subsequent doses after missed dose: Resume dose escalation according to Table 1 for adults or Table 2 for pediatric patients | First dose after missed dose: Administer 1 dose below the maintenance dose Second and subsequent doses after missed dose: Resume the maintenance dose |
| 3 or more consecutive missed doses | For adult patients who have not completed the dose escalation phase: Reinitiate dose escalation regimen starting at 0.1 mg/kg and follow Table 1.For pediatric patients who have not completed the dose escalation phase: Reinitiate dose escalation regimen starting at 0.03 mg/kg and follow Table 2. | First and subsequent doses after missed doses: Restart dosing at 0.3 mg/kg and follow Table 1 for adult patients or Table 2 for pediatric patients.For adult patients who have missed 3 or more consecutive doses in the mainenance phase during which sphingomyelin could have reaccumulated: The treating physician may consider resuming dosing at 0.1 mg/kg and dose escalate according to Table 1.For pediatric patients who have missed 3 or more consecutive doses in the maintenance phase during which sphingomyelin could have reaccumulated: The treating physician may consider resuming dosing at 0.03 mg/kg and dose escalate according to Table 2. |
— Etiqueta de la FDA: Xenpozyme, Genzyme Corporation, vigente desde el 11 de diciembre de 2025. Esto es solo una parte de la sección. Leer la etiqueta completa en DailyMed →
Olipudase alfa-rpcp 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, XENPOZYME may cause embryo-fetal harm when administered to a pregnant female. XENPOZYME dosage initiation or escalation, at any time during pregnancy, is not recommended as it may lead to elevated sphingomyelin metabolite levels that may increase the risk of fetal malformations (see Data), [see Clinical Pharmacology (12.2)].
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However, the decision to continue or discontinue XENPOZYME maintenance dosing in pregnancy should consider the female's need for XENPOZYME, the potential drug-related risks to the fetus, and the potential adverse outcomes from untreated maternal ASMD disease. In an embryo-fetal toxicity study in pregnant mice, a rare malformation (exencephaly) was observed in offspring at an exposure less than the exposure at the maximum recommended human dose (MRHD) of olipudase alfa-rpcp (see Data). There are no available data on XENPOZYME use in pregnant females to evaluate for a drug associated risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes. Advise the pregnant female of the potential risk to the fetus. The background risk of major birth defects and miscarriage for the indicated population is unknown. All pregnancies have a background risk of birth defect, miscarriage, or other adverse outcomes. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively. Data Animal Data In an embryo-fetal development study in pregnant mice, olipudase alfa-rpcp was administered intravenously at doses of 3, 10, or 30 mg/kg daily from gestation day (GD) 6 through GD 15. Exencephaly was observed in 1 litter at each of the 10 and 30 mg/kg dose groups (2 and 3 fetuses, respectively). These data are consistent with published literature reports that brief embryonic exposures to sphingomyelin metabolites or a sphingosine-1-phosphate (S1P) receptor modulator produced neural tube defects, including exencephaly, in chicks and mice. The developmental No Observed Adverse Effect Level (NOAEL) is 3 mg/kg. The AUC associated with this dose is 0.14-fold the clinical exposure at the MRHD. The developmental Lowest-Observed-Adverse-Effect Level (LOAEL), 10 mg/kg, is also associated with an exposure that is less than the clinical exposure at the MRHD. In an embryo-fetal development study in pregnant rabbits, olipudase alfa-rpcp was administered intravenously at doses of 3, 10, or 30 mg/kg daily from GD 6 through GD 19. There was no maternal or developmental toxicity. The developmental NOAEL was 30 mg/kg; the AUC 0–24 at this dose is approximately 10.5-fold the exposure at the MRHD. In a study of pre- and postnatal development in mice, olipudase alfa-rpcp was administered intravenously every other day from GD 6 through GD 18; then resumed every other day after parturition, from Lactation Day (LD) 1 through LD 19. Olipudase alfa-rpcp did not induce any effect on maternal reproductive function or on developmental and reproductive parameters of male and female offspring.
— Etiqueta de la FDA: Xenpozyme, Genzyme Corporation, vigente desde el 11 de diciembre de 2025. Esto es solo una parte de la sección. Leer la etiqueta completa en DailyMed →
Sobredosis de Olipudase alfa-rpcp
Cita de la sección «Overdosage» (sobredosis) de la etiqueta aprobada por la FDA. Se muestra en inglés. Si alguien pudo haber tomado demasiado, llame a Poison Help al 1-800-222-1222 (o al 911 en una emergencia).
Cases of overdosage with XENPOZYME have been reported in pediatric patients during dose escalation. Some patients experienced serious adverse reactions including death within 24 hours of initial dose [see Warnings and Precautions (5.2)]. The clinical findings included fever, hypotension, gastrointestinal bleeding, marked elevation in liver tests, metabolic acidosis, respiratory failure, and vomiting. There is no known specific antidote for XENPOZYME overdosage.
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In the event of overdosage, immediately stop the infusion, and monitor the patient closely in a hospital setting for the development of hypersensitivity reactions and IARs including acute phase reactions. For the management of adverse reactions, see Warnings and Precautions (5.1, 5.2, 5.3) and Adverse Reactions (6.1).
— Etiqueta de la FDA: Xenpozyme, Genzyme Corporation, vigente desde el 11 de diciembre de 2025. Leer la etiqueta completa en DailyMed →
Presentaciones y vía de administración
| Formas farmacéuticas | Inyección, polvo, liofilizado, para solución |
|---|---|
| Vía | Intravenosa |
| Productos con receta | 2 en el directorio NDC de la FDA |
| Nombres de marca | Xenpozyme |
Empresas que tienen este medicamento
Empresas en el directorio NDC de la FDA: la empresa que aparece en el envase (fabricante o distribuidor), con su número de productos con receta.
- Genzyme Corporation2 productos
Historial de aprobación de la FDA
- BLA: Solicitud de licencia de productos biológicos (BLA): así se aprueba un medicamento biológico (hecho a partir de células vivas).
| Solicitud | Patrocinador | Aprobada | Cambios aprobados después |
|---|---|---|---|
| BLA761261 XENPOZYME | Genzyme Corp | 31 de agosto de 2022 | 2, el último 20 de diciembre de 2024 2024: Etiquetado · 2023: Etiquetado |
Escasez
La lista de escasez de la FDA no incluye estos productos.
Retiros del mercado
No aparece ningún retiro vinculado a estos productos (por su código NDC) en los informes de la FDA.
Identificadores oficiales
| RxCUI (RxNorm) | 2610411, 2610416, 2634357, 2634359 |
|---|---|
| ID (SPL set ID) de la etiqueta citada | 01a910ee-a33e-4be3-ac41-322d64c34311 |
| Etiquetas de la FDA (SPL) | 1 |
| Solicitudes de la FDA | BLA761261 |
| Principios activos (FDA) | OLIPUDASE ALFA |
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Fuentes
- Etiqueta aprobada por la FDA en DailyMed (Biblioteca Nacional de Medicina) — Xenpozyme, Genzyme Corporation, vigente desde el 11 de diciembre de 2025; vía openFDA, datos de la FDA exportados el 30 de octubre de 2026, consultados el 31 de octubre de 2026
- 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
- 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
- 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
- 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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