The Company

Amryt Pharma focuses on the development and commercialisation of innovative medicines for the treatment of rare and orphan diseases with high unmet medical need.


The rare and orphan disease area presents a significant opportunity for commercialisation. While there is a growing number of rare diseases being identified globally (with over 7,000 identified to date), there are a relatively low number of approved medicines to treat them (in the region of 500), and many have no targeted treatment options at all.

As a result, the conditions for bringing new therapies to market are more favourable than for products aimed at treating common diseases:

  • Investment needs are typically lower as clinical trials require fewer patients.
  • Investment in marketing and distribution infrastructure is likely to be lower, given the limited number of doctors and centres specialising in a particular rare/orphan disease.
  • There are EMA and in-country incentives available for the development and commercialisation of these treatments.
  • The regulatory approval process offers mechanisms for accelerated review and/or conditional approval.


At present, Amryt Pharma has a number of assets that are potential near term value drivers for Amryt Pharma.

  • Lojuxta®(lomitapide) – an approved treatment used as an adjunctive therapy for adult patients with Homozygous Familial Hypercholesterolemia (HoFH).
    • Amryt has exclusive marketing rights for the sale of this drug in Europe (EEA & Switzerland), MENA, Israel and Turkey.
    • It is a commercial stage asset that is delivering strong sales growth and is cash generative for the Company.
    • The market opportunity is estimated to be approximately €100m.
    • Its commercial rollout has enabled Amryt Pharma to build infrastructure that can be leveraged for other, future therapies.
    • See the Summary of Product Characteristics for further information.
  • AP101 (Oleogel-S10) – an innovative wound care treatment with a target indication for Epidermolysis bullosa (EB), for which there are currently no approved treatments.
    • The Company is currently undertaking a pivotal, global Phase III trial in EB.
    • An interim readout is expected in the first half of 2018.
    • Top line results of the trial are expected by the end of 2018.
    • The estimated market opportunity is over €1bn.
    • AP101 (Olegoel-S10) was approved in January 2016 by the EMA for the treatment of partial thickness wounds in adults.
  • AP102 – an earlier stage therapy in pre-clinical development with the potential to treat resistant acromegaly and Cushing’s disease.