Expanded Access Policy
CYPRIUM THERAPEUTICS EXPANDED ACCESS POLICY FOR INVESTIGATIONAL DRUG CUTX-101 (COPPER HISTIDINE) FOR TREATMENT OF MENKES DISEASE
Cyprium Therapeutics, Inc. (Cyprium) is a biopharmaceutical company focused on the development of novel therapies for the treatment of Menkes disease and related copper metabolism disorders. Under the 21st Century Cures Act, the manufacturer or distributor of one or more investigational drugs for the treatment of one or more serious diseases or conditions is required to 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. Described below is Cyprium Therapeutics’ policy for reviewing and responding to requests for individual patient access to our investigational drug CUTX-101 (Copper Histidinate) for the treatment of Menkes disease.
CUTX-101 is under investigation for the treatment of Menkes disease and related copper metabolism disorders. It has not yet been established as safe and effective for any such treatment. Cyprium Therapeutics believes that CUTX-101 should be available and studied in patients as part of an Intermediate-Size Patient Population Expanded Access Protocol to obtain further data on the safety and efficacy of CUTX-101 that may be used to support approval of the product and subsequent wider accessibility to Menkes disease patients. We encourage parents/legal guardians who have children with Menkes disease to speak with their physicians and to consider having their child participate in clinical trials. Cyprium Therapeutics may consider providing CUTX-101, as an investigational drug, under an Intermediate-Size Patient Population Expanded Access Protocol.
Cyprium Therapeutics will review and respond to a request that it receives on a case-by-case basis, applying the following criteria:
- There is no guarantee that Cyprium Therapeutics will provide CUTX-101 for any request.
- The patient must have a diagnosis of Menkes disease, verified by the treating physician.
- The enrollment of the patient into the Intermediate-Size Patient Population Expanded Access Protocol must be in full compliance with ICH guidelines and FDA and local regulations.
- The Intermediate-Size Patient Population Expanded Access Protocol may be discontinued by the company at any time or upon approval of CUTX-101 by the FDA for Menkes disease.
- The treating physician, if eligible as an Investigator, must be willing to comply with the assessments in the Intermediate-Size Patient Population Expanded Access Protocol.
- Only patients residing in the U.S. will be considered for enrollment into the study.
A treating physician may submit questions or requests regarding expanded access to the following: StudyInfo@CypriumTx.com
Additional information may be obtained from the U.S. Food and Drug Administration at: https://www.fda.gov/downloads/newsevents/publichealthfocus/expandedaccesscompassionateuse/ucm504494.pdf
Requests for participation in the Intermediate-Size Patient Population Expanded Access trial should be submitted to Cyprium Therapeutics by the treating licensed physician in the U.S. and should include sufficient supporting de-identified patient information to enable Cyprium Therapeutics and the Principal Investigator of the study to evaluate the expanded access request.
Please include contact information so Cyprium Therapeutics can follow-up with the physician directly (i.e., physician’s name/institution, address, phone number, e-mail, and IRB information). Requests for expanded access need to be made by the physician responsible for treating the patient, and each request needs to relate to a single patient.
If the physician’s request to enroll the patient is approved, the requesting physician would be expected to become an Investigator to the Intermediate-Size Patient Population Expanded Access Protocol.
Cyprium Therapeutics will attempt to acknowledge receipt of a request for expanded access within 5-10 business days of receipt, if possible.
The clinical trial record containing information on the Intermediate-Size Patient Population Expanded Access Protocol (CYP-001) is available on clinicaltrials.gov: https://clinicaltrials.gov/ct2/show/NCT04074512
As authorized by the 21st Century Cures Act, Cyprium Therapeutics may revise this expanded access policy at any time. Additionally, the posting of the policy by Cyprium Therapeutics shall not serve as a guarantee of access to CUTX-101 and/or enrollment into the Intermediate-Size Patient Population Expanded Access Protocol for any patient.