Expanded Access Policy

Achillion Policy on Expanded Access to Investigational Drugs

The mission of Achillion is to provide better treatments for people with serious diseases. We are a science-driven company committed to putting patients first. In our endeavor to bring innovative medicines to patients as fast and safely as possible, we conduct clinical trials to establish the safety and efficacy of an ‘investigational drug’. An ‘investigational drug’ is a potential medicine that is in active clinical development, but has not yet received marketing approval by regulatory authorities. Clinical trials and the subsequent timely generation of safety and efficacy data are the most effective way of ensuring review and decision making by Regulatory Authorities. This ultimately has resulted in access to new, safe and effective approved medicines for patients.

Achillion supports the need for Early Access to investigational drugs for patients ineligible to enroll in clinical trials.

An Early Access program for a specific investigational drug may be opened if Achillion determines that all the following criteria are met:

  1. The investigational drug is the subject of an active clinical development program and is not approved in any indication in the country concerned
  2. An adequate supply of the investigational drug exists to perform necessary clinical studies as well as to provide Early Access to patients who do not have alternative treatment options
  3. Early Access does not impede or compromise the clinical development or regulatory approval of the drug under investigation
  4. There are sufficient clinical data available with respect to both the investigational drug and the disease condition for which the application is being sought, to anticipate that any potential benefits from treatment are likely to outweigh any potential risks to the patient

Achillion continues to assess the eligibility requirements and criteria for Early Access to the investigational drug danicopan (ACH-4471).  At this time, an Early Access Program (EAP) is not available.

We will re-evaluate this policy from time to time.