Personal Importation of Pitolisant
The Food and Drug Administration (FDA) allows for the personal importation of medications (www.fda.gov/ForIndustry/ImportProgram/ImportBasics/ucm432661.htm). Patients who believe that pitolisant may be useful in relieving their disease burden may petition the FDA for approval for personal importation. There is now precedence for personal importation of Wakix™ (pitolisant) for the treatment of narcolepsy and cataplexy-like symptoms in a child with PWS; Chion Foundation has facilitated the personal importation of Wakix™ into the U.S. to fulfill a prescription for one child and two additional requests are pending FDA approval. Please contact Chion Foundation if you would like for us to help you through the process.
From the FDA Web Site
Personal importation may be requested for drugs that are available in foreign countries but not in the United States. These drugs are available in international pharmacies, and, in the case of pitolisant, now available in German pharmacies. The FDA will use its discretion to allow entry of drugs mailed from a foreign pharmacy in order to fill a prescription-like order written by a physician in the United States.
While packages that contain a drug, biologic or medical device are typically denied entry by Customs and Border Protection officers, FDA personnel may use their discretion to allow entry of shipments of currently unapproved FDA-regulated products. They will do this when the quantity and purpose of the products are clearly for personal use and the product does not present an unreasonable risk to the user.
To facilitate personal importation, patients and physicians may contact the FDA in advance of purchasing the medicine to request advance approval. FDA personnel may use their discretion to examine the background, risk and purpose of the product before making a final decision about personal importation.
What this Means for Us
Briefly, any physician in the United States can write a prescription for Wakix™ (pitolisant). Pitolisant is intended to be started at a low dose and the dosage increased until symptoms resolve. The tablets are available in 4.5 mg and 18 mg doses and thus the prescription should be written to accommodate a starting dose of 4.5 mg but allowing for the possibility of an increase in dosing up to 18 mg.
The prescription must be accompanied by a simple one page letter from the prescribing physician to the FDA indicating that PWS is a serious condition, that there is no known health risk from pitolisant, and that currently available treatments have not effectively normalized the sleep-wake cycle in the patient If you need assistance with this letter, please contact the Chion Foundation as we have developed a template for this letter that has successfully been used to secure FDA approval for personal importation of Wakix™. The patient’s family must also write a simple one page letter to the FDA requesting permission for personal importation. The letter must affirm that the pitolisant is for personal use, that you will not commercialize (sell) it, and that it does not represent an unreasonable risk to the patient. Chion Foundation has also developed a template for this letter that you may want to peruse.
These two letters and the prescription can then be emailed to a patient liaison at the FDA who will then “walk” the request through the FDA. The FDA will use its discretion to determine whether or not to approve the request. If the request is approved, it will be possible to purchase a three month supply of pitolisant. In order to do this, the email from the FDA must be sent/emailed to a pharmacy in Europe that has access to pitolisant.
In order to collect data outcomes on the use of pitolisant in our PWS population, we ask that all patients with PWS who try pitolisant to please document the patient’s response to pitolisant. One way to do this is to write to us at firstname.lastname@example.org and request an invitation to the patient experience platform.