An expanded access program designed to help those in need

We are a biotech company that is committed to identifying, developing and delivering first-in-class or best-in-class oral small-molecule and protein therapeutics for patients living with complement-mediated and other rare diseases. For information regarding BioCryst clinical trials, please visit We understand that there are seriously ill patients who will not be eligible for available clinical trials and don’t have options for alternative therapies. In these circumstances, BioCryst will consider providing a requesting physician with access to an investigational medicine for the treatment of an individual patient outside of a clinical trial, or through an early access program when certain conditions are met.

Policy regarding expanded access

BioCryst evaluates physician requests for individual patients to receive investigational medicines for serious or life-threatening diseases or conditions outside of a clinical trial on a case-by-case basis. BioCryst cannot guarantee that the investigational medicine will be available for a particular patient.

In evaluating these physician requests, BioCryst considers many factors, including the following:

  •  Whether the request is likely to interfere with BioCryst’s overall development plan for the medication
  • Whether there are other adequate alternative therapies or clinical trials available
  • Whether the scientific evidence supports both the safety and the efficacy of the medicine for the requested use at an appropriate dose
  • Whether the potential benefit to the patient justifies the potential risks
  • Whether an adequate and appropriate supply of the medicine is available
  • Whether the requesting physician is sufficiently qualified

A requesting physician should expect to receive a response acknowledging receipt of a request within 7 business days after a completed request has been received. For investigational medicines other than berotralstat, requesting physicians should submit a request via our Medical Information page. If granted, access to the investigational medicine will be provided for a designated period of time and will require additional submissions to continue.

BioCryst may revise this policy at any time.

Grants and sponsorships: supporting the communities we serve

We build genuine, enduring relationships with members of patient communities and all who support them. We offer a range of opportunities to provide financial support to these communities, from medical educational grants and sponsorships to charitable contributions and donations. Request a grant or sponsorship >>

Your right to privacy

Clinical research is critical for the development of medicines for complement-mediated and other rare diseases. Just as we recognize the importance of sharing learnings from clinical trial data to advance our research, we hold ourselves accountable to the highest standards when handling patient privacy. Learn about our commitment to clinical trial data transparency >>