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.


The Company has a number of assets that are potential near-term value drivers

  • Lojuxta®(lomitapide) – an approved treatment used as an adjunctive therapy for adult patients with Homozygous Familial Hypercholesterolaemia (HoFH).
    • Amryt Pharma has exclusive marketing rights for the sale of this drug in Europe (EEA & Switzerland), MENA, Israel, Turkey, Russia, the Commonwealth of Independent States, and the non-EU Balkan states.
    • It is a commercial stage asset that is delivering strong sales growth for the Company.
    • Sales of Lojuxta are generated both directly and through distribution agreements.
    • The market opportunity is estimated to be approximately €125m.
    • The infrastructure that supports Lojuxta sales can also be leveraged for other, future therapies.
    • See the Summary of Product Characteristics for further information.
  • AP101 (Oleogel S-10) – an innovative wound care treatment with a target indication for Epidermolysis Bullosa (EB). Currently there are no approved treatments for EB.
    • The Company is undertaking a pivotal, global Phase III trial in EB.
    • An interim readout halfway through the Phase III study is expected in the last quarter of 2018.
    • Top line results of the trial are expected in the second quarter of 2019.
    • The market opportunity is estimated to be over €1bn.
    • AP101 (Olegoel-S10) was approved in January 2016 by the EMA for the treatment of partial thickness wounds in adults.
    • Additional market opportunities for AP101 (Oleogel S-10) in partial thickness wound indications are under evaluation. These include AP101 as a treatment for:
      • Toxic Epidermal Necrolysis Syndrome (TENS)/Stevens-Johnson Syndrome (SJS);
      • Bullous Pemphigoid;
      • Pemphigus Vulgaris; and
      • Grade III/IV radiotherapy and chemotherapy-induced dermatitis.

  • Please refer to this page to learn more about the EASE study:
  • AP102 – an earlier stage therapy in pre-clinical development with the potential to treat resistant acromegaly and Cushing’s disease.
  • AP103 – Amryt Pharma has exclusively in-licensed a new non-viral vector platform technology for gene therapy with potential applicability across a range of genetic disorders, including EB. The technology has been in-licenced from University College Dublin and involves the delivery of gene therapy using Highly Branched Poly (β-Amino Ester) polymer technology. Preliminary data suggests that the treatment could be potentially disease-modifying for patients with Recessive Dystrophic EB (RDEB.)
    • Please visit this page to discover more details about AP103 [add hyperlink to new page when available]