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By PCCA
In early May, the FDA made an announcement about oxitriptan, also known as 5-hydroxitryptophan or 5-HTP. It stated that it did “not intend to object to the compounding of oral oxitriptan for patients with BH4 deficiency who have a prescription identifying the disorder, as long as the drug is compounded in compliance with all other conditions of section 503A of the FD&C Act.”1 This was a pleasant surprise, since oxitriptan was one of the active ingredients that the FDA had decided not to include on the 503A bulk drug substances list (“the positive list”) according to its final rule that went into effect on March 21 of this year.2
This is an important statement for patients with BH4 deficiency as well as the practitioners and compounders who help them. Tetrahydrobiopterin (BH4) deficiency is a group of congenital metabolic disorders also known as primary tetrahydrobiopterin (BH4) deficiency, atypical phenylketonuria (PKU) or BH4-deficient hyperphenylalaninemia.1
In its May announcement about oxitriptan, the FDA stated that several compounding pharmacies and physicians had contacted the agency about compounding with it. One of them was Summit Health Pharmacy in Brookhaven, Pennsylvania, and specifically its owner, Vince Canzanese, RPh (pictured above). Vince had drafted and submitted to the FDA a detailed citizen petition requesting that oxitriptan be added to the positive list and that the agency allow it to be compounded for this patient population in the interim. Vince compounds medications for several patients with BH4 deficiency and works very closely with them as well as their prescribers. In addition to a review of the literature regarding the use of oxitriptan, he also included brief case histories of his patients in the petition.
While the agency has not added it to the positive list, the FDA announcing that it will not object to oxitriptan being compounded for patients with this condition is an example of the agency taking a patient-centered approach to regulation. Of course, this is also an outstanding example of a compounding pharmacist going out of his way to ensure that his patients have access to the medications they need.
We applaud the FDA for their decision, and we thank Vince Canzanese along with the other compounders and physicians who contacted the FDA to ensure that patients with BH4 deficiency can continue to receive the compounded medications they need.
A version of this article originally appeared in PCCA’s members-only magazine, the Apothagram.
References 1. U.S. Food and Drug Administration. (2019). Communications with stakeholders concerning access to compounded oral oxitriptan for patients with tetrahydrobiopterin deficiency. Retrieved from https://www.fda.gov/drugs/drug-safety-and-availability/communications-stakeholders-concerning-access-compounded-oral-oxitriptan-patients 2. U.S. Food and Drug Administration. (2019). List of bulk drug substances that can be used to compound drug products in accordance with section 503A of the Federal Food, Drug, and Cosmetic Act. Retrieved from https://www.federalregister.gov/documents/2019/02/19/2019-02367/list-of-bulk-drug-substances-that-can-be-used-to-compound-drug-products-in-accordance-with-section