Medical Affairs
Medical Information and
Investigator-Initiated Studies (IIS)
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Medical Information
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T: 1-844-600-2237
Email: [email protected]
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Adverse events may also be reported to the FDA’s MedWatch Program at www.fda.gov/medwatch or by calling (800) FDA-1088.
Investigator-Initiated Studies
Overview
The Zevra Therapeutics Investigator-Initiated Studies (IIS) Program will accept unsolicited submissions within our current areas of interest (AOI) as noted below. Submissions from qualified Investigators (i.e., health care professionals, scientists, and researchers) will be reviewed by the Zevra Investigator and Collaborative Research Review Committee for alignment with the AOIs. Research submissions that are not complete, that are not within the scope of these AOIs, or have low relevance to the therapeutic area, may be rejected without further review.
Proposals will be evaluated on their scientific merit to advance science, clinical medicine, and patient care according to availability of resources. Support can include, but is not limited to, drug material, expert advice and/or funding to pay expenses directly related to the research based on fair market value.
The Investigator, or the institution the investigator is affiliated with, will serve as the study sponsor. As such, the sponsor assumes responsibility for initiating and conducting the study, directing the administration of the study drug, ensuring compliance with all local laws and regulatory requirements, and analyzing and communicating all study results.
The goal of the IIS is to advance science and contribute to the development of better medicines for patients consistent with the company’s overall research and development strategies.
Areas of Interest (AOIs)
MIPLYFFA
- Further understand the mechanism of action of arimoclomol
- Further understand the benefit risk of arimoclomol for NPC
- Clinical or pre-clinical evidence of the effectiveness and safety of arimoclomol in neurological or other lysosomal storage disorders
OLPRUVA
- Management of neurological disorders with OLPRUVA
- OLPRUVA adherence, palatability, and treatment satisfaction in pediatric and adult populations
- Neurocognitive outcomes, including longitudinal assessment, in children and adult UCD patients that may or may not be treated with OLPRUVA
IIS Submission Process
The submission process consists of 2 key steps:
- Concept review
- Review of a 1-2 page summary of the study rationale, objectives, and design
- A description of the type of support requested (e.g., funding, study drug)
- An estimated budget (if funding is requested)
- Full proposal review
If the concept is approved for further review, you will be invited to submit a full proposal application including:
- Detailed study protocol
- Comprehensive budget
- Additional supporting documents as needed
To inquire about submitting a proposal, please send an email to [email protected]
