Discovering new medicines for patients

Making new medicines available for patients is a complex process taking many years, in which our company investigates thousands of potential new medicines for every one that proves to be safe, effective, and can be prescribed by doctors.

This process can be divided into the following steps:

  1. Target discovery: Detailed knowledge of disease processes at the molecular level allows us to determine how and where a candidate medicine may act.
  2. Compound optimisation: Synthesis of compounds that may be effective at the target, and selection and testing of the most promising candidate medicines to establish their safety and likely efficacy, prior to testing in humans.

  3. Clinical studies: This entails testing candidate medicines in humans. These clinical studies are strictly controlled and monitored by government agencies and Ethics Committees to ensure the very highest standards of safety and ethics. Our studies follow the guidelines laid down by the World Medical Association on the Ethical Principles for Medical Research Involving Human Subjects (Declaration of Helsinki 2013) and the International Conference on Harmonisation (ICH).

    There are two main types of clinical studies:

    Healthy volunteer studies: Studies in healthy volunteers, who may be paid for their time.

    Patient studies: Studies in patients who have one of the diseases for which we are developing promising new candidate medicines.

    If you would like further information, please click on the appropriate disease area below. Also you can contact us on

    Amyotrophic Lateral Sclerosis

    Diffuse Cutaneous Systemic Sclerosis

    Erythropoietic Protoporphyria

    X-Linked Protoporphyria

  4. Regulatory Approval: Once a drug has successfully completed all three phases of clinical studies in humans, all of the information showing that the drug works, is safe and of an acceptable quality, must be submitted to the government regulators in each country where we plan to market the medicine. The details of the information that must be provided to the regulators and the way in which the information must be provided, is detailed in guidelines that are available on the internet (EU Legislation - Eudralex and Committee for Medicinal Products for Human Use). The government regulators will formally review all available information. If they are satisfied that it meets all of the relevant guidelines and requirements, and the benefits of the medicine outweigh any possible risks, they will grant a marketing authorisation or licence.

    For further information on research and development, please contact us.