IQVIA and Swedish Orphan Biovitrum’s PNH Study: A Potential Game-Changer for Pegcetacoplan

IQVIA Holdings ((IQV)), Swedish Orphan Biovitrum Ab ((GB:0MTD)) announced an update on their ongoing clinical study.

IQVIA Holdings and Swedish Orphan Biovitrum are collaborating on a clinical study titled ‘A Single Arm, Long-term, Multicentre Observational Study to Evaluate Effectiveness of Pegcetacoplan Under Real World Conditions in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH).’ The study aims to assess the real-world effectiveness of pegcetacoplan, a treatment for PNH, over a period of up to 72 months. This research is significant as it seeks to provide comprehensive data on the long-term impact of pegcetacoplan in a real-world setting.

The study focuses on the drug pegcetacoplan, which is intended to treat patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). Pegcetacoplan is administered as per its label to evaluate its effectiveness and safety in real-world conditions.

This observational study follows a cohort model, collecting both retrospective and prospective data. The primary purpose is to gather information on the effectiveness and safety of pegcetacoplan, alongside patient and clinician-reported outcomes and healthcare resource usage.

The study commenced on February 20, 2023, and is currently recruiting participants. The primary completion and estimated study completion dates are not specified, but the last update was submitted on May 9, 2025. These timelines are crucial for tracking the study’s progress and ensuring timely data collection.

The update on this study may influence investor sentiment positively, as successful outcomes could enhance the market position of Swedish Orphan Biovitrum and IQVIA Holdings. The study’s results could potentially impact stock performance, especially if pegcetacoplan proves to be effective, offering a competitive edge in the PNH treatment market.

The study is ongoing, and further details are available on the ClinicalTrials portal.