Our Product Pipeline

Innovative pre-clinical development and extensive experience from clinical development of pharmaceuticals.

This is essential in order to take orphan and specialty pharmaceuticals to the market, and to deliver valuable products and product support to our patients and customers.

Related Information


Cerys living with hereditary tyrosinemia type 1 (HT-1)
Cerys living with hereditary tyrosinemia type 1 (HT-1)

Our collaborative, patient-centric and sustainable way of developing new and effective therapies is the heart of our operations, aimed at improving quality of life for people who live with a rare disease all over the world.

We focus on medications for rare disease patients with a high unmet medical need. This category includes specialist and so-called orphan drugs, which are mainly prescribed by clinical specialists and aimed at small well-defined patient groups. These less common diseases are often neglected and inadequately treated which means that new medications can offer significant value for the patient and health care providers.

Influenced by the patient journey and based on the needs of patient representatives and caregivers, we continuously explore the potential to further develop authorised medications for new indications.

Responsible pricing

An effective treatment is one that not only gives a ­medical benefit but is also both available in the country where the patient lives and is affordable in the healthcare system. 

One of the most important factors to ensure patient access to treatment is responsible pricing. That means balancing the role of a sustainable company with being a sustainable part of the healthcare system. A number of multi-stakeholder initiatives to support dialogue and collaboration have been presented and are in place, with the objective of finding shared sustainable solutions or frameworks to include rare disease treatments in the healthcare systems.

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