Pombiliti (cipaglucosidase alfa-atga) / Amicus - LARVOL DELTA

Cipaglucosidase alfa plus miglustat in Pompe disease: two non-ambulatory patients switching from high‑dose, high-frequency alglucosidase alfa. (PubMed, Neuromuscul Disord) - "We analyzed outcomes in two non-ambulatory patients in study ATB200-02 who received alg for >13 years (including >2 years' HDHF) before switching to cipa+mig (20 mg/kg + 260 mg every 2 weeks). The two patients experienced 11 non-serious adverse events (no infusion-associated reactions). Data provide information for clinicians considering a transition from HDHF alg to cipa+mig."

Journal • Fatigue • Lysosomal Storage Diseases • Metabolic Disorders • Pompe Disease • Rare Diseases

Predicting PICOs for EU HTA: The Validated PICO Planner Approach Based on Retrospective Analysis (ISPOR-EU 2025) - "Exercises were conducted for four products across oncology (Pluvicto and non-small cell lung cancer [NSCLC]), rare disease (Pombiliti), and type 2 diabetes (T2DM). The findings demonstrate that a structured, data-driven approach to evidence collection and consolidation can predict PICOs. These predictions can be further supported through validation with country affiliates and systematic literature reviews. This approach fosters collaboration with HTA stakeholders, enhances evidence planning, accelerates reviews, and promotes equitable, value-based access—positioning secondary research as a strategic enabler of patient-centered innovation across Europe."

Retrospective data • Diabetes • Lung Cancer • Metabolic Disorders • Non Small Cell Lung Cancer • Rare Diseases • Solid Tumor • Type 2 Diabetes Mellitus

A Disease Progression Model Comparing Respiratory and Motor Decline in People With Late-Onset Pompe Disease Treated With Cipaglucosidase Alfa Plus Miglustat vs. Alglucosidase Alfa (ISPOR-EU 2025) - P1/2, P3 | "Given limited studies on lifetime trajectory of people with LOPD treated with ERT, we modelled long-term disease progression for people receiving alglucosidase alfa (alg) or cipaglucosidase alfa + miglustat (cipa+mig). A patient-level simulation model estimated lifetime mobility and respiratory disease progression outcomes among people with LOPD treated with cipa+mig versus alg based on 6-minute walk distance and % predicted forced vital capacity. In this model, cipa+mig delayed disease progression compared with alg in people with LOPD, increasing the time they did not depend on mobility and respiratory support. While data on progression to respiratory and mobility support are limited, this model, informed by clinical and real-world evidence and expert input, suggests potential for cipa+mig to provide quality-of-life benefits for people with LOPD compared with alg."

Pompe Disease • Rare Diseases • Respiratory Diseases

Effect size analysis of cipaglucosidase alfa + miglustat versus alglucosidase alfa in ERT-experienced adults with late-onset Pompe disease in PROPEL (SSIEM 2023) - No abstract available

Clinical • Pompe Disease

104-week efficacy and safety of cipaglucosidase alfa+miglustat in patients with late-onset Pompe disease previously treated with alglucosidase alfa (SSIEM 2023) - No abstract available

Clinical • Pompe Disease

Safety of home administration of cipaglucosidase alfa + miglustat in late-onset Pompe disease: results from multiple clinical trials (SSIEM 2023) - No abstract available

Clinical • Pompe Disease

Effect size analysis of cipaglucosidase alfa + miglustat versus alglucosidase alfa in ERT-experienced adults with late-onset Pompe disease in PROPEL (SSIEM 2023) - No abstract available

Clinical • Pompe Disease

104-week efficacy and safety of cipaglucosidase alfa+miglustat in patients with late-onset Pompe disease previously treated with alglucosidase alfa (SSIEM 2023) - No abstract available

Clinical • Pompe Disease

Safety of home administration of cipaglucosidase alfa + miglustat in late-onset Pompe disease: results from multiple clinical trials (SSIEM 2023) - No abstract available

Clinical • Pompe Disease

Effect size analysis of cipaglucosidase alfa + miglustat versus alglucosidase alfa in ERT-experienced adults with late-onset Pompe disease in PROPEL (SSIEM 2023) - No abstract available

Clinical • Pompe Disease

104-week efficacy and safety of cipaglucosidase alfa+miglustat in patients with late-onset Pompe disease previously treated with alglucosidase alfa (SSIEM 2023) - No abstract available

Clinical • Pompe Disease

Safety of home administration of cipaglucosidase alfa + miglustat in late-onset Pompe disease: results from multiple clinical trials (SSIEM 2023) - No abstract available

Clinical • Pompe Disease

Effect size analysis of cipaglucosidase alfa + miglustat versus alglucosidase alfa in ERT-experienced adults with late-onset Pompe disease in PROPEL (SSIEM 2023) - P3 | "Patients switching to cipa+mig did not demonstrate any significant within-group worsening and showed significant improvements for 6-minute walk distance (absolute and % predicted); upper, lower and overall manual muscle test; PROMIS fatigue; physician and subject global impression of change (5 of 8 subdomains); CK and Hex4 levels. Conclusions This analysis shows that ERT-experienced patients with LOPD who switched from alg to cipa+mig treatment achieved improvements in a number of outcomes, highlighting the potential of cipa+mig to become an important treatment option for these patients."

Clinical • Fatigue • Pompe Disease

104-week efficacy and safety of cipaglucosidase alfa+miglustat in patients with late-onset Pompe disease previously treated with alglucosidase alfa (SSIEM 2023) - P3 | "No new safety signals were identified. Conclusions These data show that switching treatment from alg to cipa+mig was associated with a durable effect up to 104 weeks and was well tolerated, supporting long-term benefits of cipa+mig treatment for patients with LOPD."

Clinical • Fatigue • Pompe Disease

Safety of home administration of cipaglucosidase alfa + miglustat in late-onset Pompe disease: results from multiple clinical trials (SSIEM 2023) - P1/2, P3 | "Overall, evidence across the clinical trials shows that cipa+mig can be safely administered in a home setting."

Clinical • Pompe Disease

Switching treatment from alglucosidase alfa to cipaglucosidase alfa plus miglustat positively affects motor function and quality of life in patients with late-onset Pompe disease (SSIEM 2023) - P3 | "The Phase III PROPEL trial (NCT03729362) assessed efficacy and safety of the enzyme- replacement therapy (ERT) cipaglucosidase alfa+miglustat (cipa+mig) versus alglucosidase alfa+placebo (alg+pbo) in adults with LOPD. Results Group-level analyses favored cipa+mig versus alg+pbo in the vast majority of motor function and PRO measures, with nominal significance for walking tests and SGIC’s ‘ability to move around’ and ‘energy level.’ Patient-level responder analyses showed a greater proportion of patients improved with cipa+mig versus alg+pbo for most PRO measures. Differences in proportions of responders between cipa+mig versus alg+pbo were nominally significant for SGIC’s ‘overall well-being,’ ‘ability to move around,’ ‘muscle function’ and ‘energy level.’ Conclusion These analyses highlight the patient perspective and provide evidence that switching from alg+pbo to cipa+mig benefits patients’ motor function and HRQoL."

Clinical • HEOR • Pompe Disease

ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD (clinicaltrials.gov) - P3 | N=21 | Active, not recruiting | Sponsor: Amicus Therapeutics | Recruiting ➔ Active, not recruiting

Enrollment closed • Pediatrics • Pompe Disease

An Indirect Treatment Comparison of Avalglucosidase Alfa versus Cipaglucosidase Alfa Plus Miglustat in Patients with Late-Onset Pompe Disease. (PubMed, Adv Ther) - P1, P1/2, P2, P3 | "ITCs suggest more favourable respiratory and mobility outcomes with AVA versus Cipa+mig in patients with LOPD, regardless of prior ERT-experience."

Journal • Pompe Disease

Cipaglucosidase alfa and miglustat for treatment of late-onset Pompe disease (LOPD): A therapeutics bulletin of the American College of Medical Genetics and Genomics (ACMG). (PubMed, Genet Med Open) - No abstract available

Journal • Pompe Disease

Evaluating PICOs Requested by Member States During Scoping for EU HTA Versus Those Covered in Actual HTAs (ISPOR 2025) - "We aimed to assess the alignment between PICOs identified in EUnetHTA 21 scoping surveys for PLUVICTO®, EBVALLO® and POMBILITI™ published in 2023, versus PICOs considered in actual HTAs of these products. PICOs included in actual HTAs were predicted by the EUnetHTA 21 scoping exercise; however, there may be over-scoping of PICOs due to the scoping process and requirement to meet all member states' needs. This may place an unnecessary burden on manufacturers responding to irrelevant PICOs and manufacturer input into the scoping of EU JCAs could be beneficial"

A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPD (clinicaltrials.gov) - P3 | N=119 | Completed | Sponsor: Amicus Therapeutics | Active, not recruiting ➔ Completed | Trial completion date: Dec 2026 ➔ Dec 2024

Trial completion • Trial completion date • Pompe Disease

Amicus Therapeutics Announces Full-Year 2024 Financial Results and Corporate Updates (GlobeNewswire) - "Galafold (migalastat) net product sales for the full-year 2024 were $458.1 million, representing a year-over-year increase of 18%, or 19% at CER. Fourth quarter net product sales were $127.5 million...Pombiliti (cipaglucosidase alfa-atga) + Opfolda (miglustat) net product sales for the full-year 2024 were $70.2 million. Fourth quarter net product sales were $22.2 million...For the full year 2025, the Company provides revenue growth guidance for Pombiliti + Opfolda of +65% to +85% on a constant currency basis (CER)...First commercial patients from these countries are anticipated to begin treatment over the first half of 2025. Additionally, Pombiliti + Opfolda received regulatory approval in Australia and the Company anticipates new regulatory decisions in Canada and Japan in 2025 as well as additional reimbursement agreements throughout the year."

Canada approval • Japan approval • Reimbursement • Sales • Fabry Disease • Pompe Disease

Effect Size Analysis of Cipaglucosidase Alfa Plus Miglustat Versus Alglucosidase Alfa in ERT-experienced Adults with Late-onset Pompe Disease in PROPEL (S21.003). (PubMed, Neurology) - P3 | "The randomized, double-blind PROPEL study (ATB200-03; NCT03729362) compared the efficacy and safety of the investigational two-component enzyme replacement therapy (ERT) cipa+mig with alg plus placebo in adults with late-onset Pompe disease (LOPD); 77% of patients had received ERT with alg before study entry (median ERT duration 7.4 years)...Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cabaletta Bio...Dr. Mozaffar has received personal compensation in the range of $500-$4,999 for serving as a Study Section Member with NIH."

Clinical • Journal • CNS Disorders • Fatigue • Muscular Dystrophy • Pompe Disease • Rare Diseases

A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPD (clinicaltrials.gov) - P3 | N=110 | Active, not recruiting | Sponsor: Amicus Therapeutics | Trial completion date: Dec 2024 ➔ Dec 2026

Trial completion date • Pompe Disease

Challenges in multinational rare disease clinical studies during COVID-19: regulatory assessment of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease. (PubMed, J Neurol) - P3 | "PROPEL (ATB200-03; NCT03729362) compared the efficacy and safety of cipaglucosidase alfa plus miglustat (cipa + mig), a two-component therapy for late-onset Pompe disease (LOPD), versus alglucosidase alfa plus placebo (alg + pbo). Both statistical analysis approaches led to similar results and consistent conclusions, confirming the efficacy of cipa + mig for adults with LOPD. NCT03729362; trial start date: December 4, 2018.Trial registration number."

Journal • Infectious Disease • Lysosomal Storage Diseases • Metabolic Disorders • Novel Coronavirus Disease • Pompe Disease • Rare Diseases