Our Policies

Expanded Access Policy

Under the recently enacted 21st Century Cures Act in the US, 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 the policy of the manufacturer or distributor on evaluating and responding to requests submitted for expanded access under section 561(b) of the US Federal Food, Drug, and Cosmetic Act.

As a company working toward scientific advancements in the treatment of diseases that have high unmet medical needs, the Company and its affiliates support the Cures Act and its provisions to speed the development of new treatments for patients. In accordance with the Cures Act, the following are the Company’s policies and procedures relating to how it will evaluate and respond to requests for expanded access to the Company’s investigational drugs to treat serious diseases:

Contact Information

The Company currently has no active clinical trials in the US. In the event that the Company begins a clinical trial involving a serious disease or condition in the US, this website and policy will be updated to notify the public whether the Company will consider accepting expanded access requests at that time. The following is the contact information at the Company for communications regarding expanded access requests as described above:

  • 1-888-292-0058 (please note that no expanded access program is available at this time)
Request Procedures

The Company currently has no active clinical trials in the US. In the event that the Company begins a clinical trial involving a serious disease or condition in the US, this website and policy will be updated to notify the public whether the Company will consider accepting expanded access requests at that time.

General Criteria

As noted above, the Company currently has no active clinical trials in the US. In the event that the Company begins a clinical trial involving a serious disease or condition in the US, this website and policy will be updated to notify the public whether the Company will consider accepting expanded access requests at that time. The following are the general criteria that the Company expects to use to evaluate any expanded access requests in the future:

  • The individual patient must suffer from a fatal or severely disabling condition for which no other effective treatment options exist.
  • There must be no other clinical trials underway, or the individual patient must be either categorically ineligible or disqualified from the clinical trials.
  • There must be clear and convincing evidence of safety of the investigational drug, demonstrated by a clinical trial.
  • There must be clear and convincing evidence that the investigational drug could be beneficial in the particular patient for whom the indication for which expanded access is sought.
  • There must be clear and convincing evidence that the risks associated with the use of the investigational drug are reasonable in light of the potential benefits.
  • There must be an open Investigational New Drug application (IND) in the US, under which clinical trials are to be conducted in the US.
  • The investigational drug must be used in clinical trials for the condition for which access is requested.
  • Any preapproval access to an investigational drug must always comply with the applicable local laws and regulations, including approvals from applicable regulatory bodies, and by an institutional review board or ethics committee for the treating hospital.
  • Qualified health professionals must be available to administer the investigational drug and monitor the patient under a study protocol.
  • There must be adequate supply of the investigational drug, beyond what is required for clinical trials.
  • In the event that the Company discontinues clinical development of the investigational drug, expanded access will be timely closed out as well for that investigational drug.
  • Once the US Food and Drug Administration (FDA) has approved the applicable drug for any indication, the expanded access program for that drug will be phased out, as the drug will then be commercially available.
  • The Company does not offer expanded access for an investigational drug which is in an early stage of testing since there are still lots of unknowns.
Anticipated Timing

The Company anticipates that it will acknowledge receipt of such request within five (5) business days of receipt of the applicable request.

Clinicaltrials.gov

The Company currently has no active clinical trials in the US. In the event the Company begins a clinical trial involving a serious disease or condition in the US that may qualify for expanded access under the US Federal Food, Drug, and Cosmetic Act, this website and policy will be updated to notify the public whether the Company will consider accepting expanded access requests at that time, and if so, with a hyperlink or other reference to the clinical trial record containing the required information about the expanded access for such drug.

Nothing herein shall prevent the Company from revising this policy at any time. The posting of this policy by the Company shall not serve as a guarantee of access to any specific investigational drug by any individual patient.