Cytokinetics is committed to translating our advanced understanding of muscle biology into novel treatments that may help people with debilitating diseases in which muscle performance is compromised and/or declining. We recognize that people living with these diseases may be in desperate need of treatment options and may also be interested in the possibility of our making our investigational therapies available to them upon request and prior to potential regulatory approval.
Any access to our investigational therapies would have to be fair and equitable, safeguard the welfare of patients, and not put at risk the completion of our ongoing research and development. Cytokinetics has sought advice from patients, advocates, physicians and ethicists on these important considerations. Through these interactions, we have concluded that sustainable, long-term availability of our investigational therapies can best be achieved following demonstration of the investigational drug’s safety, tolerability, and effectiveness through controlled clinical trials, which may hopefully address the requirements of regulatory agencies.
Offering pre-approval access outside of a controlled clinical trial may jeopardize the conduct of our ongoing clinical trials and may also prevent or delay our broader clinical development programs, which would likely then result in a setback to all patients in need. Accordingly, we only provide access to some investigational therapies outside of our clinical trials through limited Managed Access Programs, and those programs have now concluded.
In many of our trials, we also provide the opportunity for participants to enroll in Open Label Extension (OLE) studies in which all participants, including those who previously received placebo, receive investigational therapy. In addition to providing trial participants access to the investigational therapy for an extended period time, an OLE study may provide useful data on the long-term efficacy, tolerability, and safety of the investigational drug.
More information on our clinical trials is available on www.clinicaltrials.gov and on www.cytokinetics.com/medicines-research/#clinical-trials
We are grateful to the people and families who have participated in our clinical trials. We may consider potential expanded access to our investigational therapies as we learn more about them in ongoing clinical trials.
For additional information about this policy, please contact [email protected]. Responses will be provided within approximately one business week.