Harriet Davies, PharmD
Stiripentol is not an FDA approved drug. For the last several years, US citizens have been able to obtain stiripentol by ordering directly from Biocodex, France OR by ordering through Caligor Pharmacy, NY via the FDA’s Personal Importation Policy (PIP):
FDA’s PIP is discretionary, meaning the FDA has the right to control the importation of a non-approved drug for personal use. This policy is meant to help people with life threatening illnesses obtain drugs when FDA approved drugs have failed. The FDA does not consider the PIP a “license” to import drugs for personal use (even for people with life threatening illnesses). However, the FDA will allow importation of investigational drugs for life threatening conditions if the criteria in the import policy are met in most cases.
There are now many patients with Dravet Syndrome on stiripentol in the United States, and the drug remains unapproved (investigational). Because stiripentol has been imported for most patients through the PIP, the FDA does not know exactly how many US citizens are on the drug and also does not have safety data on the use of an unapproved product in a fairly large group of patients. The FDA preferred way of obtaining a drug for investigational use is by the FDA Expanded Access IND process. The IND Expanded Access program allows physicians to request permission from the FDA to use an investigational drug in a patient with a severe or life threatening condition in which FDA approved drugs have failed. Safety data is collected at regular intervals on all patients receiving an investigational drug via and IND and the FDA is alerted to any serious or life threatening adverse events. Also, the FDA is able to ascertain how many US citizens are using an investigational drug at any given time. The FDA’s ultimate goal for investigational drugs for life threatening conditions is for the sponsor of the drug to submit the necessary data for FDA approval so the drug will be available to all patients with the disease. When growing numbers of patients with a rare disease such as Dravet Syndrome use an investigational drug for treatment, there is concern that once the clinical studies that are necessary for FDA approval begin, there will not be enough patients that haven’t used the drug to enroll in the studies. This may delay or prevent FDA approval. This website gives consumer information on Expanded Access INDs:
Considering all of the above, in 2013 the FDA mandated that all new patients who obtain stiripentol in the United States through Caligor Pharmacy must have an IND submitted to the FDA by the prescribing physician. New patients are considered those who had not been receiving a continued supply of stiripentol prior to December 2012. Patients who were prescribed stiripentol prior to December 2012 and who had received continual shipments of stiripentol from Caligor Pharmacy are “grandfathered” and will continue to be able to receive stiripentol from Caligor Pharmacy without an IND in place.
The following websites contain instructions on how the physician should submit the IND:
If the physician has questions about the IND process, he or she should call FDA’s Center for Drug Evaluation and Research (CDER) Neurology division at 301-796-2250 and ask for the project manager that is handling stiripentol INDs. It is not appropriate for parents or caregivers to call this number to get information about the IND on the doctor’s behalf, the doctor or the doctor’s agent must communicate with the FDA. It is recommended that the doctor call prior to submitting the IND so that he or she has all the information necessary for the IND to go forward.
New patients whose parents or caregivers wish to import stiripentol directly from the manufacturer, Biocodex (France) through FDA’s Personal Importation Policy may order the drug without an IND in place.