At Prilenia, we are committed to developing safe and efficacious therapies for rare neurodegenerative diseases. We understand the huge impact these diseases can have on the patients and their families' lives. We are committed to providing these therapies to the broadest group of patients as quickly as possible.
As part of this commitment, we conduct clinical trials to determine if our investigational medicines are safe and effective for patients with a particular disease, disorder or condition which may allow us to seek the necessary regulatory approvals to provide wide access to these medicines. To date, pridopidine has not been approved in any country and is in late-stage development for Huntington's Disease (Phase 3) and ALS (phase 2/3).
We understand that, in some cases, patients with serious diseases or conditions may not be eligible for clinical trials and may not have other treatment options. Under limited circumstances, we will consider making investigational medicines available for individual patients to treat a serious disease or condition outside of a clinical trial under so called early access. Early access is generally referred to as “expanded access” or “compassionate use”. Under limited circumstances, we will consider potential implementation of early access based on substantial scientific evidence to justify the use of an investigational product for a serious disease or condition, for patients that are not eligible for clinical studies and when provision of the investigational drug is logistically feasible.
The following criteria must be met for us to consider making one of our investigational medicines available to individuals under early access:
The patient has a serious or life-threatening disease, and existing approved therapies are not appropriate or effective to an extent that consideration of using an unapproved therapy is justified;
There are no other viable therapeutic options, including participation in ongoing relevant clinical trials;
The investigational medicine is currently being studied, in clinical trials for the treatment of a disease or condition that is similar in type and stage to the indication for which early access is sought.
The patient’s underlying medical conditions do not pose undue safety risks;
The request must be made by a licensed physician who is qualified and knowledgeable in the treatment of the disease or condition. Patients who are interested in understanding access options should speak with the physician overseeing their care;
There is sufficient scientific evidence that the potential benefit to the patient, taking into account available safety and efficacy information, would likely outweigh the potential risks to the patient;
Adequate clinical data exist to support the appropriate dosing regimen of the investigational medicine and duration of treatment in the disease or condition;
The company has an adequate supply of the investigational product to meet the needs of the expanded access program without impairing ongoing development, including ongoing and planned clinical trials.
It should be logistically feasible for the company to provide the patient with the investigational drug;
The requested use complies with applicable laws and regulations.
Any requests for early access to experimental therapies must be made by a qualified and licensed physician and will be referred to an internal team for evaluation. Qualified and licensed physicians may make such requests at email@example.com.
Requests are assessed, on a case-by-case basis, by a multidisciplinary team at Prilenia. We target acknowledging receipt of requests within ten (10) business days. The requesting physician must agree to obtain appropriate regulatory and ethics committee approvals, if applicable, and to comply with all other safety, monitoring, and patient consent requirements. The request must not contain any patient personal identifiable information.
Early access may be discontinued when approval of the drug is achieved in the patient’s country, subject to any legal or regulatory requirements. Similarly, expanded access may be discontinued if the benefit risk is no longer positive or if any of the original required criteria are no longer met.
Additional information regarding our clinical studies can be found at https://www.prilenia.com/pipeline
We may revise this policy at any time consistent with applicable law and regulations.