Access Policy

MyoKardia was founded on a commitment to improve the lives of those suffering from serious cardiovascular disease by discovering and developing targeted therapies that can change the course of their condition. Our initial focus is on the advancement of therapeutic candidates for the most common forms of heritable cardiomyopathies —hypertrophic cardiomyopathy, or HCM, and dilated cardiomyopathy, or DCM.

The most efficient and effective method of making new medicines available to patients is by conducting well-designed clinical trials. Companies like MyoKardia are required to evaluate the safety and efficacy of our novel therapeutic drug candidates in controlled clinical studies and submit results to regulatory agencies for consideration in order to obtain marketing approvals. We understand that for patients with HCM or DCM who do not qualify for participation in a clinical trial and their physicians, expanded access to investigational medicines may be of interest.

In order to ensure fair and equal access to our investigational therapies, and to safeguard the welfare of patients, MyoKardia has determined that the best course for accomplishing the broadest possible access and the long-term availability of our drugs is by first confirming their safety, tolerability and efficacy through controlled clinical trials. Offering access outside of these trials could jeopardize the conduct of these trials, delay or prevent future clinical development programs and potentially create a setback to all patients in need. Therefore, at this time, MyoKardia is not able to provide expanded access to its investigational therapies.

We are deeply grateful at MyoKardia for the participation by patients, their families and clinical researchers and their staffs in our clinical programs.  Without your commitment and efforts, we could not pursue our mission to innovate new medicines for the treatment of cardiovascular diseases.

For information on our current clinical studies, please go to, or

For additional information about this policy, please contact Responses will be given within 10 days.

MyoKardia may revise this expanded access policy at any time. This posting will be updated should there be any policy change.