Expanded Access Program/Policy
BioCryst Expanded Access Programs and Policy
BioCryst Pharmaceuticals, Inc. (BioCryst) is a biotech company that is committed to identifying, developing and delivering novel therapeutics for patients with rare and serious diseases. For information regarding BioCryst clinical trials, please visit clinicaltrials.gov and search for BioCryst in order to view clinical trials sponsored by BioCryst. 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.
ORLADEYO™ (berotralstat) Expanded Access Program
At this time, the only Expanded Access Program at BioCryst is for ORLADEYO for eligible patients in the United States.
If a treating physician believes Expanded Access to ORLADEYO may be an option for a patient, the physician should make a formal request on behalf of the patient. The request for access to a BioCryst investigational medicine can only be considered if the patient’s treating physician is committed to, and supportive of, the requested treatment.
Physicians seeking additional information regarding this Expanded Access Program for ORLADEYO should submit a request via firstname.lastname@example.org (telephone number 1-888-225-8677).
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 email@example.com . 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.