Drug approval means that a specific regulator has authorised a specific product, made by a specific manufacturer, for specific uses, in its own jurisdiction. It is not a global stamp on a molecule. The same compound can be approved in one country and not another, approved for one condition but not a different one, and approved in one product while the "same" substance sold elsewhere is not approved at all. This guide explains what approval means in several major jurisdictions and the terms that are often confused with it.
Key Takeaways
- Approval is product-specific, use-specific and jurisdiction-specific.
- In the United States, FDA approval means the agency has determined that a drug's benefits outweigh its known risks for its intended use [1].
- In the European Union, the European Commission grants centralised marketing authorisations based on the EMA's scientific recommendation [2].
- Registration, listing or compounding are not the same as approval in the US [3][4].
- WHO does not approve medicines for individual countries, but it recognises regulators that meet high performance standards [5].
What Approval Actually Covers
When a medicines regulator approves (or "authorises" or "licenses") a medicine, it is typically approving a package of things:
- the product (a specific formulation, strength and manufacturer),
- the indications (the conditions it is approved to treat or prevent),
- the dosing and population described in the official product information, and
- the manufacturing and quality controls used to make it.
Change any of these and the approval may no longer apply. That is why "Is compound X approved?" rarely has a simple yes-or-no answer. A better question is: which product, for what use, and where?
United States: The FDA
In the United States, a company must submit clinical data and other information to the Food and Drug Administration (FDA) showing that a drug is safe and effective for its intended uses. The FDA explains that "safe" does not mean the drug has no side effects; it means the FDA has determined that its benefits outweigh its potential risks for a particular use [1].
Several related situations are often mistaken for approval:
| Situation | What it means in the US |
|---|---|
| Off-label use | A healthcare provider prescribes an approved drug for an unapproved use, dose or form. The FDA has not determined it is safe and effective for that use [1]. |
| Compounded drugs | Medications prepared by licensed pharmacists or outsourcing facilities for individual patient needs. Compounded drugs are not FDA approved; the FDA does not review their safety, effectiveness or quality before marketing [4]. |
| "FDA registered" facility or listed drug | Registration and listing do not denote approval. Federal regulations state that implying approval because of registration or listing is misleading [3]. |
| Investigational drugs | Products still being studied. They have not been approved and the FDA has not found them safe and effective for their specific use [6]. |
| "Research use" products | The FDA has warned companies that illegally sold unapproved drugs falsely labelled "for research purposes" or "not for human consumption" [7]. |
The FDA has also noted that some compounders used salt forms of semaglutide (such as semaglutide sodium or acetate), which are different active ingredients from those in the approved drugs [7]. This shows how narrowly approval is defined: a closely related substance is not automatically covered.
European Union: EMA and the European Commission
The EU has two main routes to authorisation [2]:
- Centralised procedure. A company submits one application to the European Medicines Agency (EMA). The EMA's scientific committee assesses it and makes a recommendation, but the European Commission is the body that grants the legally binding marketing authorisation. That authorisation is valid in all EU Member States and in Iceland, Liechtenstein and Norway. It is compulsory for certain categories, such as medicines derived from biotechnology, orphan medicines, and new active substances for diseases including cancer and diabetes.
- National procedures. Many medicines, including most generics and non-prescription medicines, are authorised by individual Member States. Companies can extend national authorisations to other countries through the mutual-recognition or decentralised procedures.
An important nuance: authorisation does not guarantee a medicine is available everywhere in the EU. Pricing and reimbursement decisions are made at national or regional level [2].
United Kingdom: The MHRA
In the UK, medicines need a marketing authorisation from the Medicines and Healthcare products Regulatory Agency (MHRA). Authorised products carry a product licence number. From 1 January 2025, the MHRA no longer issues Great Britain-only (PLGB) numbers, and authorisations granted cover the whole of the UK [8].
Australia: The TGA
In Australia, medicines are included in the Australian Register of Therapeutic Goods (ARTG) and carry an AUST number on the label. The type of number matters [9]:
- AUST R (registered) and AUST L(A) (assessed listed) medicines have been assessed for efficacy.
- AUST L (listed) medicines, which are lower-risk products, have not been assessed for efficacy.
The TGA also notes that some legal access pathways allow supply of "unapproved" medicines without an AUST number, but that a medicine without one, supplied outside those pathways, may have been supplied illegally [9]. This is a good example of how "approved" can have layers within a single system.
Canada: Health Canada
In Canada, Health Canada reviews a drug's safety, efficacy and quality. If the benefits are judged to outweigh the risks, it issues a Notice of Compliance (NOC) and a Drug Identification Number (DIN), which authorise the drug to be marketed in Canada [10].
Where Does WHO Fit In?
The World Health Organization does not approve medicines for use in individual countries. Its role includes supporting and evaluating regulatory systems. Through its WHO Listed Authorities (WLA) framework, WHO publicly designates regulators that operate at an advanced level of performance. This framework replaces the older, procurement-oriented concept of "stringent regulatory authorities" and is intended to help other regulators and procurement agencies rely on trusted decisions [5].
Similarly, the ICH (International Council for Harmonisation) writes shared technical guidelines used by many regulators, but it does not approve products itself.
Comparing Drug Approval Terms Across Jurisdictions
| Jurisdiction | Regulator | Common term | Key identifier or outcome |
|---|---|---|---|
| United States | FDA | Approval | Approved labelling [1] |
| European Union | EMA (assessment) and European Commission (decision), or national authorities | Marketing authorisation | Valid EU/EEA-wide if centralised [2] |
| United Kingdom | MHRA | Marketing authorisation | Product licence (PL) number [8] |
| Australia | TGA | Registration or listing on the ARTG | AUST R, AUST L(A) or AUST L number [9] |
| Canada | Health Canada | Market authorisation | NOC and DIN [10] |
Terminology differs, but the core idea is similar: an independent authority has reviewed evidence about a specific product and allowed it to be marketed for defined uses.
Why a Compound Can Be Approved in One Place but Not Another
- The company never applied. Regulators generally assess products only when a company submits an application.
- Different evidence standards or timing. Regulators may reach decisions at different times or interpret evidence differently.
- Different indications. A product may be approved for one condition in one country and a broader set elsewhere.
- Different products. An approved branded medicine and an unregulated product containing a similar substance are not the same thing in regulatory terms [7].
This is why The Lab Rat avoids calling any compound simply "approved" or "unapproved." For research compounds still in development, see investigational compounds and clinical trial phases.
Approval Is Not the End of Oversight
Approval is followed by ongoing monitoring. In the EU, for example, the EMA and Member States continuously monitor suspected side effects and can restrict, suspend or change the information for a medicine if new evidence shows its benefit-risk balance has changed [2]. Manufacturing quality is also monitored; see research-grade vs. pharmaceutical-grade.
Regulatory Status
This article describes general frameworks, not the status of any particular compound. Regulatory status varies by jurisdiction and may change over time. Consult the relevant regulatory authority for current information. Useful official starting points include the FDA (United States), EMA and national competent authorities (European Union), MHRA (United Kingdom), TGA (Australia) and Health Canada. To weigh the underlying science, see how to judge peptide research.
Frequently Asked Questions
What does "FDA approved" mean?
It means the FDA has reviewed evidence and determined that a specific drug's benefits outweigh its known risks for its approved uses. It does not mean the drug has no side effects [1].
Is a drug approved in the US automatically approved in Europe, the UK or Australia?
No. Each jurisdiction has its own regulator and process. A company must seek authorisation separately, and outcomes can differ [2][8][9][10].
What is the difference between "approved" and "off-label"?
Approved uses are those listed in the official product information. Off-label use means an approved drug is prescribed for a different condition, dose or form, which the regulator has not evaluated for that use [1].
Are compounded medications approved?
In the United States, compounded drugs are not FDA approved, meaning the FDA does not review their safety, effectiveness or quality before marketing [4]. Rules differ in other countries.
Does WHO approve medicines?
Not for individual countries. WHO supports regulatory systems and designates high-performing regulators as WHO Listed Authorities [5].
References
- U.S. Food and Drug Administration. Understanding Unapproved Use of Approved Drugs "Off Label". https://www.fda.gov/patients/learn-about-expanded-access-and-other-treatment-options/understanding-unapproved-use-approved-drugs-label ↗
- European Medicines Agency. Authorisation of medicines. https://www.ema.europa.eu/en/about-us/what-we-do/authorisation-medicines ↗
- U.S. Code of Federal Regulations. 21 CFR 207.77: What legal status is conferred by registration and listing? https://www.ecfr.gov/current/title-21/chapter-I/subchapter-C/part-207/subpart-E/section-207.77 ↗
- U.S. Food and Drug Administration. Human Drug Compounding. https://www.fda.gov/drugs/guidance-compliance-regulatory-information/human-drug-compounding ↗
- World Health Organization. WHO-Listed Authority (WLA). https://www.who.int/initiatives/who-listed-authority-reg-authorities ↗
- U.S. Food and Drug Administration. Expanded Access. https://www.fda.gov/news-events/public-health-focus/expanded-access ↗
- U.S. Food and Drug Administration. FDA's Concerns with Unapproved GLP-1 Drugs Used for Weight Loss. https://www.fda.gov/drugs/postmarket-drug-safety-information-patients-and-providers/fdas-concerns-unapproved-glp-1-drugs-used-weight-loss ↗
- Medicines and Healthcare products Regulatory Agency (GOV.UK). Apply for a licence to market a medicine in the UK. https://www.gov.uk/guidance/apply-for-a-licence-to-market-a-medicine-in-the-uk ↗
- Therapeutic Goods Administration. AUST numbers on medicine labels. https://www.tga.gov.au/products/medicines/labelling-and-advertising/medicines-and-biologicals-labelling-and-packaging/aust-numbers-medicine-labels ↗
- Health Canada. How Drugs are Reviewed in Canada. https://www.canada.ca/en/health-canada/services/drugs-health-products/drug-products/fact-sheets/drugs-reviewed-canada.html ↗
This article is for educational purposes only and is not medical or legal advice. For health decisions, consult a qualified healthcare professional, and for regulatory questions, consult the medicines regulator in your jurisdiction.