Regional development status
EU
Filed
US
Planned submission · Dec 2026
Development Pipeline
Our commitment to advancing rare disease treatments through innovative research and development
Explore our pipeline of therapies in development, from early-stage research to regulatory filing.
Advancing Treatment Standards for Rare Diseases
Orphalan's pipeline is focused on unmet needs of patients with rare diseases.
Note: The Orphalan Pipeline data is updated quarterly.
Driven by Patients, Guided by Science
Rare diseases often have limited treatment options available. Orphalan focuses on developing medicines with the potential to improve outcomes for patients living with these conditions.
Our R&D pipeline spans clinical-stage projects, from early-phase studies through to regulatory submission, with a focus on difficult-to-treat conditions. Some of these are new molecular entities, while others are existing products being studied for potential new indications or different formulations.
Therapeutic Pipeline
The table below reflects our current clinical development portfolio, spanning regulatory submissions, late-stage and early-stage programmes.
Active ingredient
Pharmaceutical form
Dosing
Indication
Phase
Products that have completed development are listed on the Products page .
iWilson's Registry
Wilson's disease data is also collected via our iWilson's Disease Registry.
Diagnostic Innovation
Beyond treatments, we are developing new diagnostic tools to help doctors detect and monitor rare diseases more accurately.
Available in the US since April 2025
NCC Assay: Measuring Non-Ceruloplasmin Copper
Copper is an essential dietary element, usually bound tightly in blood to a protein called ceruloplasmin. In Wilson's disease, the inability to remove excess copper leads to accumulation of "free" copper (non-ceruloplasmin copper, or NCC) in the blood. This excess NCC is directly responsible for the organ damage seen in Wilson's disease.
Critically, there are no validated tests readily available to measure NCC, making it difficult for physicians to monitor copper levels and assess treatment response. As part of the pivotal Phase 3 CHELATE trial, Orphalan was asked by the FDA to develop a new assay for NCC. This reliable and reproducible test has been validated and is now being made widely available to healthcare professionals.
The NCC assay was made available to healthcare professionals in the United States in April 2025, providing a valuable new tool for monitoring Wilson's disease treatment effectiveness.
Read the published research: The Lancet Gastroenterology & Hepatology- Disease Area
- Wilson's Disease
- Innovation Type
- Diagnostic Assay
- Regulatory Context
- FDA-requested as part of CHELATE Phase 3 trial
- Availability
- United States, April 2025
Interested in collaborating on rare disease research?
We welcome partnerships with researchers, clinicians and institutions committed to advancing treatments for patients with rare diseases.
Visit our Partnering page