Pombiliti (cipaglucosidase)
Research Synopsis
- Pombiliti (cipaglucosidase alfa) is a new enzyme replacement therapy that has received approval in the EU for treating adults with late-onset Pompe disease, providing significant advancements in treatment options compared to standard therapies.
- Research has demonstrated that cipaglucosidase alfa combined with miglustat results in marked improvements in patients' walking capabilities and respiratory health over extended treatment durations, indicating its potential superiority over traditional therapies.
- A pivotal study, the PROPEL trial, showcased this new combination therapy's efficacy compared to standard treatment (alglucosidase alfa plus placebo) over a 52-week period, with participants reporting significant enhancements in mobility.
- Further long-term studies, including open-label extension trials, are assessing the sustained benefits and safety of cipaglucosidase alfa, particularly among patients who have not previously undergone enzyme replacement therapy.
- Recent research emphasizes the need for continuous monitoring of cardiac and respiratory conditions in Pompe disease patients, highlighting varying responses to treatments in different individuals.
- The analysis of health-related quality of life metrics from the PROPEL trial revealed a direct correlation between mobility (measured by walking ability) and overall health outcomes, further confirming the therapy's effectiveness.
- New studies are also utilizing network meta-analysis to compare cipaglucosidase alfa with other ERTs, revealing its potential advantages in improving functional abilities, such as walking distance and lung capacity.
- Despite promising results, challenges remain in ensuring broad access to this new therapy and addressing individual variances in treatment response among Pompe disease patients.
- The landscape of Pompe disease therapies is rapidly evolving, with ongoing clinical trials for next-generation ERTs, gene therapies, and other innovative treatment modalities.
- As cipaglucosidase alfa gains traction in the medical community, continuous assessment of its long-term impact and safety profile remains essential to optimize patient care.
Pombiliti (cipaglucosidase)
Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial.
London, UK
2 hours ago
1 Received
- Pompe disease is a rare genetic condition that causes muscle and respiratory issues due to a lack of a specific enzyme, and the standard treatment (alglucosidase alfa) is not effective for all patients.!
- This study tested a new combined therapy (cipaglucosidase alfa and miglustat) to see if it was safer and more effective for late-onset Pompe disease compared to the standard treatment, involving a randomized trial across multiple countries.!
- The primary goal was to measure improvements in patients' walking ability over 52 weeks, with 125 participants divided into two treatment groups in a double-blind setup to ensure unbiased results.!
$100 - $150
Hourly Rate
The new horizons for treatment of Late-Onset Pompe Disease (LOPD).
London, UK
2 hours ago
1 Received
- - Late-onset Pompe disease (LOPD) is a genetic disorder that leads to muscle weakness and respiratory problems due to a lack of the enzyme acid alpha-glucosidase (GAA), resulting in harmful glycogen buildup in cells.
- - The first enzyme replacement therapy (ERT), alglucosidase alfa, was approved in 2006 and provided some benefits, but its effectiveness diminishes over time, prompting the search for new treatments.
- - New next-generation ERTs, like avalglucosidase alfa and cipaglucosidase alfa, are being tested in clinical trials, and innovative therapies, including gene transfer and antisense oligonucleotides, are also in early development, but challenges remain
$100 - $150
Hourly Rate
Unusual Evolution of Hypertrophic Cardiomyopathy in Non-Compaction Myocardium in a Pompe Disease Patient.
London, UK
2 hours ago
1 Received
- Classic infantile Pompe disease typically causes severe heart issues like hypertrophic cardiomyopathy, which usually improves with enzyme replacement therapy (ERT).
- A unique case is reported where a newborn initially treated for hypertrophic cardiomyopathy developed non-compaction myocardium despite ERT, indicating a different cardiac adaptation.
- This transition highlights the need for careful monitoring in Pompe disease patients and offers insights into the underlying mechanisms of non-compaction myocardium.
$100 - $150
Hourly Rate
Cipaglucosidase Alfa: First Approval.
London, UK
2 hours ago
1 Received
- - Cipaglucosidase alfa (Pombiliti) is a recombinant human enzyme therapy developed by Amicus Therapeutics to treat Pompe disease, which is caused by a deficiency of the enzyme acid α-glucosidase (GAA).
- - The treatment involves combining cipaglucosidase alfa with miglustat, an enzyme stabilizer, to address the condition effectively.
- - On March 27, 2023, cipaglucosidase alfa received approval in the EU as a long-term enzyme replacement therapy for adults with late-onset Pompe disease, marking a significant milestone in its development.
$100 - $150
Hourly Rate
Improved Enzyme Replacement Therapy with Cipaglucosidase Alfa/Miglustat in Infantile Pompe Disease.
London, UK
2 hours ago
1 Received
- - Pompe disease is a serious genetic disorder caused by a deficiency in the enzyme acid α-glucosidase (GAA), leading to severe health issues in infants without treatment and typically resulting in death within the first year.
- - Enzyme replacement therapy (ERT) has been used since 2006 with Myozyme (recombinant human GAA), which prolongs life but has limited effectiveness against disease progression.
- - A new treatment, Cipaglucosidase alfa combined with Miglustat, has shown significant improvements in a patient’s symptoms such as respiratory failure and motor skills, indicating its potential superiority over previous therapies.
$100 - $150
Hourly Rate
Enzyme replacement therapy for late-onset Pompe disease.
London, UK
2 hours ago
1 Received
- Pompe disease is caused by a lack of the enzyme acid alpha-glucosidase (GAA), with treatments including enzyme replacement therapies like alglucosidase alfa and avalglucosidase alfa, though not all treatments are approved yet.
- A study was conducted to evaluate the effects of these therapies on individuals with late-onset Pompe disease (LOPD) by analyzing randomised controlled trials (RCTs).
- Six trials involving 358 participants were reviewed, showing that after 78 weeks, alglucosidase alfa likely improves walking distance but lacks data on some important outcomes like respiratory support needs.*
$100 - $150
Hourly Rate
Long-term safety and efficacy of cipaglucosidase alfa plus miglustat in individuals living with Pompe disease: an open-label phase I/II study (ATB200-02).
London, UK
2 hours ago
1 Received
- Cipaglucosidase alfa plus miglustat (cipa + mig) is a new two-part treatment for Pompe disease, assessed in the Phase I/II ATB200-02 study over 48 months.
- The study involved four adult groups, including both ambulating and non-ambulating patients, who received specific doses of cipa and mig biweekly.
- Results showed improvements in walking distances and respiratory capacity, especially in ERT-naïve patients, with the treatment generally well tolerated and a safety profile similar to existing therapies.
$100 - $150
Hourly Rate
104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07).
London, UK
2 hours ago
1 Received
- The PROPEL study compared a new therapy, cipaglucosidase alfa + miglustat (cipa + mig), to the standard treatment, alglucosidase alfa + placebo (alg + pbo), for adults with late-onset Pompe disease (LOPD).
- An ongoing open-label extension study is assessing the long-term safety and effectiveness of cipa + mig, focusing on various outcome measures like walking distance and lung function.
- After 104 weeks, patients on cipa + mig showed maintained improvements in walking distance and biomarkers with minimal safety concerns, indicating potential long-term benefits for those with LOPD.
$100 - $150
Hourly Rate
Regulatory news: Cipaglucosidase alfa-atga (Pombiliti) coadministered with Miglustat (Opfolda) for adults with late-onset Pompe disease.
London, UK
2 hours ago
1 Received
$100 - $150
Hourly Rate
Health-Related Quality-of-Life Utility Values in Adults With Late-Onset Pompe Disease: Analyses of EQ-5D Data From the PROPEL Clinical Trial.
London, UK
2 hours ago
1 Received
- Pompe disease is a rare condition causing glycogen buildup, leading to severe muscle weakness and early mortality if untreated, but enzyme replacement therapy shows promise in improving health for those with late-onset forms.
- The PROPEL trial tested two different treatments (cipaglucosidase alfa with miglustat vs. standard therapy) in 123 adults with late-onset Pompe disease, using various health metrics, including quality of life assessments over a year.
- Analysis of the trial's health-related quality of life data indicated that patients walking more than 75 meters had utility values between 0.55 and 0.67, while those needing wheelchairs had lower predicted values, signifying a potential correlation between walking ability and overall health
$100 - $150
Hourly Rate
Comparing the efficacy of cipaglucosidase alfa plus miglustat with other enzyme replacement therapies for late-onset Pompe disease: a network meta-analysis utilizing patient-level and aggregate data.
London, UK
2 hours ago
1 Received
- Late-onset Pompe disease leads to worsening muscle and respiratory function, traditionally treated with enzyme replacement therapy (ERT) using alglucosidase alfa, but newer options like avalglucosidase alfa and cipaglucosidase alfa with miglustat are now available.
- Researchers conducted a systematic review and multi-level meta-regression to compare the effectiveness of these treatments on 6-minute walk distance (6MWD) and forced vital capacity (FVC).
- Results indicated that cipaglucoside alfa with miglustat improved both 6MWD and FVC relative to alglucosidase alfa when assessing all available evidence, especially for patients with longer prior ERT duration.
$100 - $150
Hourly Rate
Pombiliti and Opfolda: shaping the future of adult late-onset pompe disease: an editorial.
London, UK
2 hours ago
1 Received
$100 - $150
Hourly Rate