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cipaglucosidase alfa + miglustat (ATB200 / ATB200 + AT2220 / Pombiliti)

✓ Approved

Amicus Therapeutics, Inc. · GAA · 小分子

什么是 cipaglucosidase alfa + miglustat？

cipaglucosidase alfa + miglustat 是一种小分子，由Amicus Therapeutics, Inc.研发。该药已获批，用于治疗相关适应症，给药途径：Injectable (Others)、Intravenous (IV)、Oral (PO)、Subcutaneous Injection。

药物档案

商品名ATB200, ATB200 + AT2220, Pombiliti
公司Amicus Therapeutics, Inc.
药物类别小分子, 重组蛋白
分子靶点GAA, UGCG
给药途径Injectable (Others), Intravenous (IV), Oral (PO), Subcutaneous Injection
状态Approved
来源： ClinicalTrials.gov, Amicus Therapeutics, Inc.

作用机制

分子靶点

cipaglucosidase alfa + miglustat 作用于 2 个分子靶点：

GAAglucosidase alpha, acid (LYAG)
UGCGUDP-glucose ceramide glucosyltransferase (GLCT1, GCS)
需要更深入的分析？Noah AI 可解释复杂机制并与同类药物比较。

治疗适应症

cipaglucosidase alfa + miglustat 针对 1 个适应症，涉及 1 个治疗领域。

治疗领域疾病/病症分期
Congenital, familial and genetic disordersGlycogen storage disease type II✓ Approved

相关研究文献

PubMedBMJ neurology open2026-10-25

Efficacy and safety of cipaglucosidase alfa plus miglustat in Japanese adults with late-onset Pompe disease: a case series from clinical trials.

Kobayashi Hiroshi H, Narita Aya A, Kawamoto Michi M, Saeki Junjiro J et al.

The safety and efficacy of cipaglucosidase alfa plus miglustat (cipa+mig) versus alglucosidase alfa plus placebo (alg+pbo) in late-onset Pompe disease (LOPD) were established in the randomised, double-blind, 52-week PROPEL study (NCT03729362) conducted across 24 countries, including Japan. An open-label extension (OLE; NCT04138277) is evaluating long-term cipa+mig outcomes. In Japan, cipa+mig was also available before marketing authorisation in an expanded access study (ATB200-19). This case series summarises the efficacy and safety of cipa+mig in Japanese patients in PROPEL, PROPEL OLE and ATB200-19. Endpoints included 6-minute walk distance (6MWD), forced vital capacity (FVC), biomarkers and safety. Cipa+mig (20 mg/kg intravenous cipa and 130-260 mg oral mig (weight-based dosing)) and alg+pbo (20 mg/kg intravenous alg and oral pbo) were given biweekly. Patients randomised to alg+pbo in PROPEL received cipa+mig in the OLE. Safety was assessed at every visit, and biomarkers at weeks 2, 4, 6, 12, 26, 38, 52 and every 26 weeks thereafter. Six patients from PROPEL/OLE (cipa+mig, n=2; alg+pbo, n=4; all enrolled in the OLE) and four from ATB200-19 were included. Two patients discontinued treatment: one in the OLE (anaphylactic reaction) and one in ATB200-19 (withdrawal of consent). For evaluable patients (n=8), 6MWD remained stable or improved from baseline to weeks 52 and 104. In PROPEL/OLE, FVC varied with most patients improving with cipa+mig treatment; in ATB200-19, FVC remained stable to week 104. In PROPEL/OLE and ATB200-19, biomarker levels improved or stabilised by week 104, including in the PROPEL switch group, where levels worsened at week 52 but improved after switching to cipa+mig. Seven patients experienced treatment-related adverse events. No deaths occurred. Although the number of patients was small, the efficacy and safety of cipa+mig in Japanese patients from PROPEL/OLE and ATB200-19 were generally sustained and durable responses consistent with positive results in the overall PROPEL population.

PMID 42921145
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PubMedReports (MDPI)2026-10-25

Liver Transplantation in Severe Hemophilia A: A Two-Patient Case Series with Divergent Long-Term Outcomes.

Shabunin Alexey Vasilyevich AV, Drozdov Pavel Alexeevich PA, Levina Oksana Nikolaevna ON, Astapovich Sergey Andreevich SA et al.

Background and Clinical Significance: Before effective viral inactivation of plasma-derived factor concentrates, many patients with hemophilia acquired hepatitis C virus infection and later developed cirrhosis or hepatocellular carcinoma. Liver transplantation treats end-stage liver disease and may also restore factor VIII production; however, long-term oncologic outcomes in this setting are rarely reported. Case Presentation: Two men with severe hemophilia A (baseline factor VIII activity < 1%) and hepatitis C-related cirrhosis underwent deceased-donor orthotopic liver transplantation. Both received an extended-half-life recombinant factor VIII (efmoroctocog alfa; 33,000 and 35,000 IU in total) tapered until postoperative days 13-14, and intraoperative hemostasis was guided by conventional coagulation tests and thromboelastography. Neither patient had major bleeding or thrombotic events, and replacement therapy was not required thereafter. In Patient 1, graft function remained satisfactory 45 months after transplantation, with factor VIII activity of 62.5%. Patient 2 was transplanted for hepatocellular carcinoma outside the Milan criteria (two lesions of 6.8 and 2.7 cm; alpha-fetoprotein 110-150 ng/mL) under center-specific expanded criteria, without bridging therapy. Factor VIII activity remained clinically sufficient, but pulmonary nodules suspected 20 months after transplantation progressed; atypical pulmonary resections 45 months after transplantation confirmed metastatic hepatocellular carcinoma, and lenvatinib was initiated. Conclusions: Liver transplantation can provide durable correction of severe hemophilia A without long-term replacement therapy. However, correction of the coagulation disorder does not eliminate risks related to the original liver disease; in patients transplanted for hepatocellular carcinoma, particularly beyond conventional criteria, long-term outcome remains dependent on tumor burden, tumor biology, candidate selection, and sustained oncologic surveillance.

PMID 42924794
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PubMedCureus2026-10-25

Less Invasive Surfactant Administration Compared With Intubation-Surfactant-Extubation (INSURE), Nebulised Surfactant, and Continuous Positive Airway Pressure (CPAP) Alone in Preterm Infants With Respiratory Distress Syndrome: A Systematic Review.

Abdelgadir Nashwa Noureldeen NN, Babiker Mohamed Rasha Omer RO, Alla Bakhit Osman Saja Dafa SD, Mohieldin Sultan Safaa Bushra SB et al.

The optimal route of surfactant delivery in preterm infants, supported non-invasively, remains contested. Less invasive surfactant administration (LISA), or minimally invasive surfactant therapy (MIST), has been compared with intubation-surfactant-extubation (INSURE), with nebulised surfactant, and with continuous positive airway pressure (CPAP) alone, with inconsistent results. Five databases and registers were searched from January 2021 to July 2026 for randomised controlled trials (RCTs) in preterm infants (<37 weeks) with respiratory distress syndrome. The review was not prospectively registered. Two reviewers independently screened records, extracted data, and applied the revised Cochrane risk-of-bias tool (RoB 2). Reporting followed Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) 2020 and, because no meta-analysis was performed, the Synthesis Without Meta-analysis guideline; certainty was rated using the Grading of Recommendations Assessment, Development and Evaluation (GRADE). Eleven RCTs (1,455 infants) were included: eight compared LISA or MIST with INSURE (n = 792), one compared MIST with sham treatment and CPAP alone (n = 485), one compared nebulised poractant alfa with CPAP alone (n = 129), and one compared nebulised surfactant with LISA (n = 49). Compared with INSURE, three trials reported reduced invasive ventilation, but two of these were at high risk of bias, while all five trials showing no difference were at low risk or raised only some concerns; risk of bias and background ventilation mode are therefore confounded, and certainty was very low. Compared with CPAP alone, MIST halved intubation within 72 hours (36.5% compared with 72.1%; risk ratio: 0.50, 95% CI: 0.40-0.64; high certainty) and reduced pneumothorax. In that trial, bronchopulmonary dysplasia among survivors was reduced (0.83, 0.70-0.98), whereas the separate composite of death or BPD was not (0.87, 0.74-1.03). Nebulised surfactant did not reduce respiratory failure compared with CPAP alone. LISA reduces early invasive ventilation compared with CPAP alone. Evidence against INSURE is heterogeneous and of very low certainty, and any attenuation with nasal intermittent positive pressure ventilation is confounded by trial quality and requires prospective testing. Nebulised surfactant remains insufficiently supported, and long-term outcomes are uncertain.

PMID 42923349
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PubMedNature medicine2026-10-24

Bispecific dual T cell agonist invikafusp alfa in solid tumors: the phase 1/2 START-001 trial.

Sullivan Ryan J RJ, Tschernia Nicholas P NP, Friedman Claire F CF, Herrera Mercedes M et al.

Invikafusp alfa (invika), a first-in-class, bispecific, dual T cell agonistic antibody selectively targeting Vβ6/Vβ10 T cell subsets, is being evaluated in START-001, a multicenter phase 1/2 monotherapy trial in participants with mostly immune checkpoint blockade (ICB)-resistant, antigen-rich solid tumors. Primary outcomes were safety/tolerability and recommended phase 2 dose (RP2D) in phase 1 and anti-tumor activity in phase 2. As of 12 March 2025, phase 1 completed dose escalation (n = 34) through dose levels of 0.01, 0.02, 0.04, 0.08, 0.12 and 0.16 mg kg-1 without reaching maximum tolerated dose, and initial phase 2 enrolled 18 participants. Two participants experienced reversible dose-limiting toxicities, one at each of the 0.08 mg kg-1 and 0.16 mg kg-1 dose levels. The most common treatment-related adverse event was cytokine release syndrome, mainly grades 1 and 2. No treatment-related deaths or non-laboratory grade 4 adverse events were reported. At the optimal biological doses (OBDs; 0.08 mg kg-1 and 0.12 mg kg-1) in combined phase 1 and phase 2 cohorts, the overall objective response rate was 14.8% (4/27), including confirmed partial responses in immunologically cold tumors, such as microsatellite stable colorectal cancer and programmed death-ligand 1 (PD-L1)-negative non-small cell lung cancer. No objective responses were seen at doses lower than 0.08 mg kg-1. Invika selectively and potently expanded peripheral and intratumoral Vβ6/Vβ10 T cells, of a predominantly memory-like phenotype, dose-dependently, peaking at the OBDs. Pharmacodynamic increases in interferon-γ and soluble CD25 followed a similar pattern. Integrated analyses from safety, pharmacokinetic, pharmacodynamic and preliminary anti-tumor data supported the selection of 0.08 mg kg-1 as the RP2D. In conclusion, phase 1 of START-001 met its primary outcomes by demonstrating the acceptable safety profile of invika consistent with its mechanism of action and selecting an RP2D. Initial phase 2 results further corroborated the safety of invika and expanded its preliminary evidence of clinically meaningful anti-tumor activity in ICB-resistant solid tumors, supporting further evaluation in the ongoing phase 2. ClinicalTrials.gov identifier: NCT05592626 .

PMID 42919963
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PubMedClinical pharmacokinetics2026-10-23

The Variability in the Pharmacokinetics of Follitropin Alfa: A Systematic Review.

Kirubakaran Ranita R, Michael Toni J F TJF, Vollenhoven Beverley J BJ, Smith Vinayak V et al.

Despite extensive research on the pharmacodynamics of follitropin alfa, a comprehensive synthesis of its pharmacokinetic variability is lacking. Understanding the interindividual and intraindividual variability in the pharmacokinetics of follitropin alfa may help inform dosing strategies by reducing variability in systemic exposure. This systematic review aimed to characterise the variability in the pharmacokinetics of follitropin alfa. Embase, PubMed, Scopus and citation mining of included articles were searched from database inception to 10 November 2025. Studies performed in humans, published in English which reported the pharmacokinetics, route of administration and dosing regimen of follitropin alfa were eligible for inclusion. Fifteen eligible studies were evaluated. The pharmacokinetics of follitropin alfa were described by a two-compartment model following intravenous administration, and a one-compartment model with first-order absorption after intramuscular or subcutaneous administration. Follitropin alfa exhibited dose-proportional pharmacokinetics. Moderate interindividual variability (26-42%) in apparent clearance was reported following multiple subcutaneous administrations. Body weight influenced the pharmacokinetics of subcutaneously administered follitropin alfa. Comparable pharmacokinetic characteristics of follitropin alfa were observed across (i) originator and biosimilar preparations, (ii) healthy and infertile individuals, and (iii) subcutaneous and intramuscular routes of administration. Weight-based dosing of follitropin alfa may reduce pharmacokinetic interindividual variability. However, whether this reduction in variability translates into more consistent ovarian response remains unclear and requires further investigation.

PMID 42919001
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PubMedJournal of cardiothoracic and vascular anesthesia2026-10-23

Thromboelastography-Informed Andexanet Alfa Administration for Residual Apixaban Effect During Acute Aortic Dissection With Cardiopulmonary Bypass.

Noto Kohei K, Miura Nana N, Imamura Yuki Y, Daitoku Kazuyoshi K et al.

PMID 42919779
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