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 with a serious, life-threatening, or imminently debilitating condition when there is no therapeutic alternative, provided that the initial request meets the criteria listed below or otherwise set forth in our Expanded Access protocol (see www.ClinicalTrials.gov with identifier NCT04689360):

  • The patient (or patient’s legal guardian) and a licensed physician are both willing to participate and complete regulatory requirements
  • The patient’s physician determines that there is no comparable or satisfactory therapy available to treat the patient’s condition
  • The patient’s physician provides documentation showing genetic confirmation of a mitochondrial disease due to nuclear DNA (nDNA) mutations, such as Barth syndrome or primary mitochondrial myopathy due to a nDNA mutation, or determines that the patient exhibits serious or life-threatening clinical manifestations of mitochondrial dysfunction for which there is sufficient evidence of the safety and effectiveness of the investigational therapy to support its use for such condition
  • The patient meets inclusion criteria for our Expanded Access protocol participation based on their clinical status
  • In the opinion of the treating physician, the potential benefit to the patient outweighs the risk to the patient’s safety
  • We have adequate supply of the investigational therapy, and the logistical capacity to supply it for the anticipated duration of treatment
  • Providing the investigational therapy will not interfere with the initiation, conduct, or completion of clinical investigations to support regulatory approval
  • The patient is unable to obtain the investigational drug under another IND or to participate in a clinical trial
  • A regulatory agency/ ethics committee and/or Institutional Review Board reviews and approves the use of the investigational therapy for the patient when applicable

Procedure for Submitting a Request

For Healthcare Professionals: Patients who meet these criteria may be able to gain access through the Stealth BioTherapeutics’ Expanded Access Program.  Details of our Expanded Access protocol can be found here.

  1. Qualified physician submits initial request via e-mail to access@stealthbt.com
  2. We will acknowledge receipt of the request and provide a Patient Information Intake Form for the physician to provide additional data 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 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, 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 access@stealthbt.com or call 617.600.8888 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.