QED Expanded Access Policy

QED Therapeutics, a subsidiary of BridgeBio Pharma, is a biotechnology company focused on precision medicine for FGFR-driven diseases. Our lead investigational candidate is infigratinib (BGJ398), an orally administered, FGFR1-3 selective tyrosine kinase inhibitor that has shown meaningful clinical activity in patients with chemotherapy-refractory cholangiocarcinoma with FGFR2 fusions and advanced urothelial carcinoma with FGFR3 genomic alterations.

QED is dedicated to bringing infigratinib, an oral, potent, selective inhibitor of FGFR1-3 to patients with FGFR-driven diseases. Our main priority is to complete the scientific research and clinical trials necessary to enable us to submit domestic and international marketing applications for infigratinib as soon as possible, so that we have the potential to help the greatest number of patients. Therefore, while clinical trials are ongoing, QED’s primary responsibility is to provide infigratinib to clinical trial participants, ensuring uninterrupted treatment and scientifically credible clinical trial results.

It is understood that there are some patients with cholangiocarcinoma, urothelial carcinoma, tumor-induced osteomalacia, or others with similar or related FGFR-driven tumors with similar high unmet medical need who will not qualify for treatment under clinical protocols. For these patients, QED is committed to a careful review of requests for Expanded Access on an individual, case-by-case basis.  Taken into consideration are factors such as a person’s current medical condition based upon information provided by their requesting physician, treatment options, the known safety and efficacy of the investigational medicine, available supply of medicine, applicable regulations, if any, among other pre-determined criteria for the patient and guidelines for the company.  In collaboration with regulatory authorities, a patient may be given access to infigratinib provided the criteria listed below are met.

At this time, infigratinib will not be provided via EA/CU for achondroplasia, other skeletal dysplasias, or any non-life-threatening conditions.

Under the 21st Century Cures Act, the manufacturer or distributor of one or more investigational drugs for the diagnosis, monitoring, or treatment of one or more serious diseases or conditions shall make available its policy on how it evaluates and responds to requests submitted under section 561(b) of the Federal Food, Drug, and Cosmetic Act for provision of such a drug. The following is QED’s expanded access policy for infigratinib:

Contact Information: Please submit any questions or requests regarding expanded access to the following: QEDExpandedAccess@qedtx.com.

Request Procedures: Requests should be submitted by licensed physicians and should include sufficient supporting detail to enable QED to evaluate the expanded access request. Please also include your contact information so QED may follow-up with you directly.

General Criteria

The individual patient’s situation must align with the following requirements:

  • The patient must first assess eligibility to applicable ongoing clinical trials utilizing QED product being requested for the disease in question
  • There must be documentation of not meeting eligibility criteria for all applicable ongoing trials, or inability to participate in a clinical trial
  • A record of prior utilization of all applicable approved on-label treatments
  • The patient has a serious or life-threatening condition with no satisfactory alternatives
  • The potential benefit must be considered to outweigh the collective potential risks for the patient

The qualified physician or healthcare professional (HCP) providing medical care to the individual must:

  • Initiate the request
  • Be experienced enough with the disease and drug class, with IND trial experience, and familiar/willing to work with the local regulatory authorities for single patient use

The QED Expanded Access Review Committee must confirm:

  • There is a regulatory mechanism in the country or region to support expanded access and must be compliant with local regulations and laws
  • There is a plan by QED Therapeutics to market the drug in the country or region from which expanded access was requested
  • There must be adequate supply of the investigational product to meet the needs of the expanded access program without impairing the QED clinical trials
  • The expanded access with not interfere with the company’s ability to complete the clinical trials in a timely fashion or which might otherwise delay marking approval and ultimately availability to all patients
  • There is a good understanding of the indication for which use is requested
  • The program will be discontinued as soon as feasible when the drug is approved for the relevant use in their country

Anticipated Timing: QED will endeavor to acknowledge receipt of any expanded access questions or requests within five business days of receipt.

Clinicaltrials.gov Hyperlink: This website and policy will be updated with a hyperlink or other reference to the expanded access record on clinicaltrials.gov after such record becomes active.

As authorized by the 21st Century Cures Act, QED may revise this expanded access policy at any time. Additionally, the posting of this policy by QED shall not serve as a guarantee of access to any specific investigational drug by any individual patient.