Healthcare Professionals

Medicine access programme

Amryt Pharma is focused on identifying, developing and commercialising innovative medicines that address unmet medical need, so people with serious diseases can lead improved lives.

To do this, we conduct clinical trials to assess the safety and efficacy of investigational medicines, and the data generated from these trials will help us obtain approvals from regulatory authorities around the world. These approvals are generally required before medicines can be made widely available to patients.

Amryt Pharma believes that the best way for patients to obtain access to investigational medicines before they are approved is by participating in clinical trials. We recognise however that in some extreme cases, when it is not possible for a patient to participate in a clinical trial and all other available medical options have been exhausted, the patient’s physician may seek special access to an Amryt Pharma investigational medicine outside of a clinical trial. These situations are referred to in various ways, including expanded access, early access, compassionate use, named patient use and emergency use.

How medicine access works

Making decisions about requests for medicine access to an investigational medicine is always complex. As a result, Amryt Pharma has engaged a group of clinicians, expert in managing the conditions for which our medicinal products are being studied, to participate in a Governance Review Board. This Review Board is responsible for assessing the requests that Amryt Pharma receive to ensure that the use of an Amryt Pharma product, in the absence of a regulatory approval in that specific condition, is not putting patients at risk of unnecessary harm.

To that end, Amryt Pharma may provide access to the Medicine Access Program, or a single request for expanded access use of an investigational medicine, only if the case has been reviewed and approved by the Amryt Pharma Governance Review Board and the benefit to the patients outweighs any potential risk.

The information that will be taken into consideration includes:

  • The patient has a serious or life-threatening illness.
  • All standard of care treatment therapies have been exhausted.
  • The patient is ineligible for, or otherwise unable to, participate in a clinical trial.
  • The patient has a disease or condition that is similar to the indication(s) for which the investigational medicine is currently being studied and for which there is sufficient evidence to expect that the patient may experience a clinically meaningful benefit.
  • Providing the investigational medicine in an expanded access framework will not interfere with the initiation, conduct, or completion of clinical trials or regulatory approval of the investigational medicine.
  • The expanded access will occur in accordance with the requirements of the national regulatory authority in which the patient resides.

How to request medicine access

If a treating physician believes medicine access may be the only option for a patient, the physician should contact Amryt Pharma to make a formal request on behalf of the patient. The requesting physician must agree to obtain appropriate regulatory and ethics committee approvals, if applicable, and to comply with all other safety, monitoring, and patient consent requirements.

Requests for medicine access may only be made by licensed physicians, in the country where the patient resides. Amryt Pharma is committed to evaluating all requests for medicine access in a timely, fair, and equitable manner. All requests will be evaluated based on available scientific evidence.

If you have any questions about Medicine Access please contact For any other medical information requests, please contact

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