Over 50 clinical trials with a single focus:Improve lives
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 physicians, ethicists, and patient advocates 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 have reached the difficult decision that it would not be appropriate to provide access to our investigational therapies outside of our clinical trials.*
We are grateful to the people and families living with debilitating diseases in which muscle performance is compromised and/or declining 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 firstname.lastname@example.org. Responses will be provided within approximately one business week.
* Due to the suspension of the development of tirasemtiv, patients previously enrolled in VIGOR-ALS, the open-label extension clinical trial following participation in VITALITY-ALS, who have chosen to continue to receive this investigational therapy, have been transitioned to a managed access program. This program is only available for these patients who had been receiving tirasemtiv in VIGOR-ALS.