Description
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) 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 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.
Key Topics
Terms and concepts identified from this document
Scope & Applicability
Stakeholders
2Typically submit individual patient expanded access requests; The individual responsible for treating the patient under expanded access.; Healthcare provider seeking information for patients; doctor or clinical staff whom the patient can contact
Individual compounding human drug products
Regulatory Context
Regulatory Activities
1Process of establishing the 503A Bulks List
Document Types
1Requirement for compounding under Section 503A
Technical Details
Substances
2Cofactor BH4
Bulk drug substance also known as 5-HTP; bulk drug substance used for compounding
Processes
1The act of creating a drug product from bulk drug substances
Clinical Concepts
2Serious and life-threatening drug interaction
Rare genetic disorder also known as BH4 deficiency; BH4 deficiency diagnosis for which oxitriptan is compounded
Identified Hazards
Hazards
1Ongoing scientific discussion or controversy about safety concerns
Standards & References
External Standards
2Official compendium for drug and device recognition
Official compendium for drug and device recognition
Related CFR Sections (2)
- 21CFR216.23§ 216.23 Bulk drug substances that can be used to compound drug products in accordance with section 503A of the Federal Food, Drug, and Cosmetic Act.
(a) The following bulk drug substances can be used in compounding under section 503A(b)(1)(A)(i)(III) of the Federal Food, Drug, and Cosmetic Act.Read full regulation →
- 21CFR10.115§ 10.115 Good guidance practices.
(a) What are good guidance practices? Good guidance practices (GGP's) are FDA's policies and procedures for developing, issuing, and using guidance documents.Read full regulation →
Enforcement Impact
Deficiencies cited in Warning Letters referencing the same regulations
Related Warning Letters (1)
See Also (8)
- Interim Policy on Compounding Using Bulk Drug Substances Under Section 503A of the Federal Food, Drug, and Cosmetic Act (Status: Final)
- IRB Review of Stand-Alone HIPAA Authorizations Under FDA Regulations: Guidance for Industry (Status: Final)
- Acceptability of Draft Labeling to Support Abbreviated New Drug Application Approval; Guidance for Industry (Status: Draft)
- DSCSA Implementation: Product Tracing Requirements for Dispensers — Compliance Policy (Revised) Guidance for Industry (Status: Final)
- Early Clinical Trials With Live Biotherapeutic Products: Chemistry, Manufacturing, and Control Information: Guidance for Industry (Status: Final)
- Determining Donor Eligibility for Autologous Donors of Blood and Blood Components Intended Solely for Autologous Use - Compliance Policy: Guidance for Industry (Status: Final)
- Guidance for Industry: Use of the Term “Healthy” in the Labeling of Human Food Products (Status: Final)
- Revised Recommendations for Determining Eligibility of Donors of Human Cells, Tissues, and Cellular and Tissue-Based Products Who Have Received Human-Derived Clotting Factor Concentrates: Guidance for Industry (Status: Final)