Patients & Families

Real People.
Real Stories.

People who suffer from diseases of abnormal mineralization face many challenges. Some of these brave patients and families are dedicated to raising awareness and explaining the urgent need for an approved treatment. Find their stories below, along with other important patient resources.  

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Expanded Access

Inozyme is committed to developing therapies for patients with rare, life-threatening disorders of pathologic mineralization and intimal proliferation. At Inozyme, our decision making is grounded in our core values that ensure the highest ethical standards of our scientific and clinical research programs. This, coupled with our compliance with regulatory and industry standards, enables our mission to advance science and research for the treatment of diseases with high unmet need.

 Participation in a clinical trial provides the best opportunity to gain access to an investigational medication. Information about ongoing Inozyme sponsored clinical trials can be accessed either at the Inozyme website ( or at the National Institutes of Health (NIH) website for clinical trials being conducted in the US (  

In some circumstances, a patient with a serious or life-threatening disease may not be able to participate in a clinical trial or a clinical trial for that patient may not be available. Seeking use of an investigational medication under these circumstances (early access or compassionate use) is permitted by the US Food and Drug Administration (FDA) under Expanded Access Programs (EAP). Analogous programs are available outside the US; requirements are country specific.

 Our policy is intended to comply with applicable regulatory requirements for any such use or access.

The following factors are considered when reviewing requests for EAP:

  • The illness must be serious or immediately life-threatening with no available treatment options (such as approved products or enrolling clinical trials).  
  • The patient must have undergone appropriate standard treatments without success and no comparable alternative treatment is available or exists to treat the disease or condition.
  • The patient is ineligible for participation in any ongoing clinical study of the investigational product, which includes lack of access due to geographic limitations. If a clinical trial is pending (almost ready to begin) for which the patient would be eligible, then the patient’s condition must be such that he/she would suffer serious  morbidity or death if required to wait for the start of the clinical trial.  
  • The patient has a disease for which there is sufficient evidence of a projected benefit from the use of the investigational product and the benefit outweighs the known or anticipated risks.
  • There is an ability to provide the product in a fair and equitable manner and there is adequate manufacturing capacity for ongoing programs.
  • Granting expanded access would not potentially compromise the scientific validity of broader development programs or interfere with or delay current clinical trials or regulatory filings designed to make the therapy available to many more patients.

All requests must be submitted by the appropriately licensed treating physician caring for the patient to better understand the benefit-risk of the therapy for the patient’s condition. Physicians wishing to discuss an early access request should submit the request via email using the email address 

The treating physician should include in the email communication the following:

  • A brief history of the patient.
  • Medications administered to control the underlying disease condition.
  • List of available alternative treatments.
  • A description of why the treating physician believes the patient would benefit from receiving the investigational medicine.

Inozyme will be solely responsible for granting expanded access requests based on information provided by the treating physician, contingent upon the approval of applicable regulatory authorities.  In some cases, Inozyme will ask the requesting physician to contact regulatory authorities directly to request expanded access.  

 For Patients/Caregivers/Patient Advocates

Inozyme does not accept requests directly from patients or family members.  All requests should be made through the patient’s treating physician. Inozyme will respond to requests within 3 business days.

For more information on early access, visit the U.S. Food and Drug Administration (FDA) website.