International Niemann–Pick Disease Alliance
Clinical trails in NPC completed with results moving on to next steps with regulators/HTA
| Study Drug | Route | Sponsor | Trial Name | Trial sites with enrolled patients | ClinicalTrials.gov Reference # | Current status |
|---|---|---|---|---|---|---|
| Adrabetadex (2-hydroxypropyl-beta cyclodextrin) |
Intrathecal | Beren Therapeutics | Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease | Australia, France, Germany, New Zealand, Singapore, Switzerland, Türkiye, United Kingdom, USA | NCT04958642 | Updated NDA submitted to FDA PDUFA date: 17 Nov 2026 |
| Adrabetadex (2-hydroxypropyl-beta cyclodextrin) |
IV | Washington University | Study of IV VTS-270 for infantile liver disease associated with NPC | St. Louis, Missouri, USA | NCT03471143 | Study completed |
| Arimoclomol / Miplyffa™ | Oral | Zevra Therapeutics | Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C | Denmark, France, Italy, Poland, Germany, Spain, Switzerland, United Kingdom, USA | NCT02612129 | Approved by FDA September 2024 Submission to EMA under way (June 2026) |
| Hydroxypropyl-beta-cyclodextrin / Trappsol® Cyclo™ | IV | CycloTherapeutics | Transport NPC – Phase 3 Study to Evaluate Intravenous Trappsol® Cyclo™ in Pediatric and Adult Patients With Niemann-Pick Disease Type C1 | Australia, Brazil, Germany, Israel, Italy, Poland, Spain, Taiwan, Türkiye, United Kingdom, USA | NCT04860960 | Study completed and data being evaluated Patients rolling over into extension arm |
| N-acetyl-L-leucine / Aqneursa™ | Oral | IntraBio | A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C | Denmark, France, Italy, Poland, Germany, Spain, Switzerland, United Kingdom, USA | NCT05163288 | Approved by FDA September 2024 Approved by EMA January 2026 |
Clinical Trials in ASMD completed with results
| Study Drug | Route | Sponsor | Trial Name | Trial sites with enrolled patients | ClinicalTrials.gov Reference # | Current status |
|---|---|---|---|---|---|---|
| Olipudase Alfa / Xenpozyme™ | IV | Sanofi | Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency (ASCEND) | Argentina, Australia, Belgium, Brazil, Bulgaria, Chile, France, Germany, Italy, Japan, Netherlands, Portugal, Spain, Tunisia, Türkiye, UK, USA | NCT02004691 | Regulatory approval received from FDA, EMA and others Global submissions ongoing Not all regulatory approvals are accompanied by HTA approvals for funding |
| Olipudase Alfa / Xenpozyme™ | IV | Sanofi | Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency (ASCEND-Peds) | Brazil, France, Germany, Italy, United Kingdom, USA | NCT02292654 | As above |
| Olipudase Alfa / Xenpozyme™ | IV | Sanofi | Data Analysis of Adult and Pediatric Participants With Acid Sphingomyelinase Deficiency (ASMD) on Early Access to Olipudase Alfa in France (OPERA) | France | NCT05359276 | Study complete |
Further resources to understand clinical trials and drug development:
What patients need to know about clinical trials – resource from the FDA:
https://www.fda.gov/patients/clinical-trials-what-patients-need-know/basics-about-clinical-trials
The drug development process – resource from the FDA:
https://www.fda.gov/patients/learn-about-drug-and-device-approvals/drug-development-process