Expanded Access Policy
Zevra aims to positively impact the lives of patients and their families by developing innovative therapies to address a variety of rare diseases where limited or no treatment options are available.
The process of seeking regulatory approval for our product candidates includes the need to recruit patients into clinical trials to evaluate and demonstrate the safety and effectiveness (benefits and risks) of our investigational products, and then to obtain regulatory approval and marketing authorization by authorities such as the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA).
Until any drug receives regulatory authority approval, it remains “investigational” and cannot be commercially available for the treatment of patients.
Expanded Access can only be offered:
- When substantial scientific evidence from clinical trials exists to support both safety and the efficacy of a therapy for an indication;
- When it neither compromises the development program nor the regulatory pathway and it is possible for an organized program to be established;
- When Zevra is able to maintain an adequate and sustainable supply of the investigational therapy, beyond the ongoing or planned clinical trial requirements;
- For serious or life-threatening medical conditions within the disease area and patient population that Zevra is investigating, there is scientific rationale for a possible therapeutic benefit and such benefit is likely to outweigh any known or anticipated risk;
- Where there are no satisfactory alternative therapies (either appropriate or effective) available to the patient;
- The patient is not eligible to participate in ongoing or planned clinical trials in a reasonably accessible geographical location;
- The patient seeking Expanded Access is a full-time resident of the country in which an EAP is approved and operating; in some countries the patient should have access to publicly funded healthcare services in that country;
- For protocol-driven programs the patient fulfills the EAP protocol eligibility criteria, as confirmed by the treating physician; and
- The request for Expanded Access is received from a qualified and licensed physician with expertise in the disease, who understands the potential risks and benefits of this investigational drug and agrees to comply with Zevra’s Expanded Access specifications and any applicable laws and regulations governing the use of the investigational drug.
Expanded Access under any program in any country will end:
- When the drug becomes commercially available and/or reimbursed in that country; or
- if Zevra determines to discontinue clinical development of the drug for the relevant disease area and/or indication.
Zevra currently offers a limited EAP for their investigational product candidate, arimoclomol, which is intended for use in the treatment of patients with Niemann-Pick type C (NPC).
Requests for expanded access to arimoclomol should only be made by qualified and licensed physicians. For more information, please contact firstname.lastname@example.org