Expanded Access

Stealth BioTherapeutics is committed to bringing therapies to patients for the treatment of rare and life-threatening diseases involving mitochondrial dysfunction. To do this, we conduct clinical trials to evaluate the safety and effectiveness of our investigational drugs in order to obtain regulatory approval to provide these investigational therapies to the broadest group of patients.

Enrolling in a clinical trial is the customary way for patients to access our investigational therapies. However, in some rare circumstances, when patients with serious, life-threatening or imminently debilitating conditions are not eligible and/or it is not feasible to enroll in a clinical trial, their physicians may seek special access to investigational therapies. These patients may be eligible to receive investigational therapies outside the clinical trial setting through an expanded access program (also known as compassionate use, named patient, or preapproval access). Our goal is to ensure access to our investigational therapies at the appropriate time, in the correct manner for patients, and in alignment with our active clinical development programs.

We will evaluate expanded access requests submitted by a treating physician on a case-by-case basis for patients who meet the criteria listed below. When an investigational drug is made available for the treatment of a particular patient it may not be made available in response to requests for other patients whose circumstances may differ.

  • The patient has a serious and life-threatening or severely debilitating condition that presents imminent risk of grave harm that, in the opinion of the treating physician, cannot be adequately addressed by any approved therapies.
  • The patient is not eligible to and/or it is not feasible for the patient to enroll in a clinical trial of the investigational therapy for which expanded access is requested.
  • The treating physician provides appropriate documentation of his or her determination that there is an unmet medical need and no comparable or satisfactory therapy available to treat the patient’s condition.
  • The potential benefit to the patient of treatment with the investigational therapy for the indication requested outweighs the risk to the patient’s safety.
  • The patient (or patient’s legally authorized representative) and a licensed physician are both willing to participate and complete regulatory requirements.
  • In our scientific judgement based on our knowledge of our investigational therapies, we believe that there is a sound scientific basis and rationale for use of the investigational therapy and we believe that the investigational therapy has been sufficiently tested in both animal models and early clinical trials to justify the potential risk of its provision in the proposed expanded access setting.
  • There is adequate supply of the investigational therapy, and the logistical capacity to supply it for the anticipated duration of treatment in the country where the request originates.
  • It is determined that providing the investigational therapy will not interfere with the initiation, conduct, or completion of clinical investigations to support regulatory approval for broader patient access.
  • A regulatory agency/ethics committee and/or Institutional Review Board reviews and approves the use of the investigational therapy for the patient when applicable.
  • The proposed use will occur in a country where we plan to seek approval of the investigational medicine.

Procedure for Submitting a Request

For Healthcare Professionals: Patients who meet these criteria may be able to gain access through Stealth BioTherapeutics’ Expanded Access Program.

  1. Qualified physician submits initial request via e-mail to EAP@stealthbt.com
  2. We will acknowledge receipt of the request and provide a Patient Information Intake Form for the physician complete to support our review of eligibility, including at least the following information:
    • Demographics of the proposed expanded access patient
    • Relevant medical history, including all therapies administered to date for the relevant indication
    • Recent laboratory assessments including genetic confirmation of mitochondrial disease due to nuclear DNA mutations when appropriate
    • Assessment of the patient’s prognosis
    • Rationale for requesting expanded access to the investigational drug
  1. Complete Patient Information Intake Form is returned to Stealth via provided e-mail
  2. We will notify requesting physician of our initial decision of patient eligibility based on the Patient Information Intake Form and any other supplemental information reviewed. We may request additional information
  3. If approved, we will provide the requesting physician with additional Expanded Access Program details
  4. All eligibility determinations for our Expanded Access Program are final; there is no appeal process
  5. Continued access to the investigational therapy is not guaranteed, and is contingent upon a determination by and documentation from the treating physician of ongoing benefit and clinical safety
  6. Access may end in other circumstances, including but not limited to the termination of the Expanded Access Program, an approval (or failure to approve) the investigational therapy in the patients’ country of residence for the indication under treatment, evolving clinical data, regional or country regulations, or supply limitations

Additionally, under certain circumstances emergency access may be considered. For emergency consideration the licensed treating physician should contact us at EAP@stealthbt.com or call 617.600.6888 and specify the emergency access need.

As provided by the 21st Century Cures Act, and with our regulatory obligations and the health and safety of patients in mind, we may revise or terminate our Expanded Access Program or this policy at any time.  An existing program does not guarantee availability in all countries. Various regulatory processes exist in different countries and country-specific variations for pre-approval access will occur. Any pre-approval access must always comply with the applicable country-specific laws and regulations. We reserve the right to not offer investigational drugs in situations when such legal, regulatory or compliance requirements may interfere with our ability to develop effective therapies for the diseases we seek to treat.

Information about our ongoing clinical trials can be found at https://clinicaltrials.gov or   www.StealthBt.com

Information about Stealth BioTherapeutics’ ongoing clinical trials can be found at https://clinicaltrials.gov or Programs & Pipeline.

This Program is not a guarantee of access to any Stealth BioTherapeutics investigational drug. Stealth BioTherapeutics reserves the right to revise or terminate this Program at any time.