FDA Compliance Policy for Certain Compounding of Oral Oxitriptan (5-HTP) Drug Products for Patients With Tetrahydrobiopterin (BH4) Deficiency Immediately in Effect

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Compliance Policy for Certain Compounding of Oral Oxitriptan (5-HTP) Drug Products for Patients With Tetrahydrobiopterin (BH4) Deficiency Immediately in Effect[附网盘链接]是于不久之前发布的FDA标准，适用于US。

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Compliance Policy for Certain Compounding of Oral Oxitriptan (5-

HTP) Drug Products for Patients With Tetrahydrobiopterin (BH4) Deficiency 1

Immediately in Effect Guidance for Industry This guidance represents the current thinking of the Food and Drug Administration (FDA, the Agency, or we) on this topic. It does not establish any rights for any person and is not binding on FDA or the public. You can use an alternative approach if it satisfies the requirements of the applicable statutes and regulations. To discuss an alternative approach, contact the FDA staff responsible for this guidance as listed on the title page. I. INTRODUCTION This guidance describes the Food and Drug Administration’s (FDA, we, or the Agency) policy concerning the conditions under which the Agency does not generally intend to take regulatory action against a licensed pharmacist in a State-licensed pharmacy or Federal facility or a licensed physician using the bulk drug substance oxitriptan (also known as 5-hydroxytryptophan or 5-HTP) 2,3

to compound oral drug products for patients with tetrahydrobiopterin (BH4) deficiency. On February 19, 2019, FDA issued a final rule (84 FR 4696) (“final rule”) that established the list of bulk drug substances that can be used to compound drug products under section 503A of the Federal Food, Drug, and Cosmetic Act (FD&C Act), even though they are not the subject of an applicable United States Pharmacopoeia (USP) or National Formulary (NF) monograph or a 4

component of an FDA approved drug product (503A Bulks List). The final rule, codified at 21 CFR 216.23, placed six bulk drug substances on the 503A Bulks List (21 CFR 216.23(a)), and identified four others, including oxitriptan, that cannot be used to compound drug products under section 503A of the FD&C Act (21 CFR 216.23(b)). Additional bulk drug substances nominated by the public for inclusion on this list are currently under consideration and will be the subject of future rulemaking. FDA developed this guidance in response to communications from pharmacists and caregivers regarding the use of oxitriptan to treat patients with BH4 deficiency following issuance of the final rule. According to those communications and other information available to the Agency, 1

This guidance has been prepared by multiple offices in the Center for Drug Evaluation and Research, in consultation with the Office of Regulatory Affairs at the Food and Drug Administration. 2

Tetrahydrobiopterin (BH4) deficiency is also known as: primary tetrahydrobiopterin deficiency, atypical phenylketonuria (PKU), GTP cyclohydrolase (GTPCH) deficiency, 6-pyruvoyl-tetrahydropterin synthase (6-PTPS) deficiency, and dihydropteridine reductase (DHPR) deficiency. 3

This guidance does not apply to drugs compounded for use in animals. All FDA guidances are available on the FDA guidance web page. FDA updates guidances regularly. To make sure you have the most recent version of a guidance, always consult the guidance web page at http://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm. 4

See section 503A(b)(1)(A) of the FD&C Act [21 U.S.C. 353a(b)(1)(A)].

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