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Accelerated approval

Also known as: accelerated approval pathway, conditional approval, surrogate endpoint approval

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Accelerated approval is an FDA pathway that lets drugs for serious conditions with unmet medical need be approved based on a surrogate endpoint, a marker such as tumor shrinkage or a lab value that is reasonably likely to predict clinical benefit, rather than waiting for outcomes like survival. Sponsors must then run confirmatory trials.

How it works

The pathway was established in 1992, largely in response to the HIV epidemic, and is now used most heavily in oncology. The sponsor must show an effect on a surrogate or intermediate clinical endpoint. After approval, the company has to conduct post-marketing confirmatory trials. If they fail to verify benefit, the FDA can withdraw the indication.

The Food and Drug Omnibus Reform Act of 2022 (FDORA) strengthened the program: the FDA can require that confirmatory trials be underway before granting approval, sponsors must report progress regularly, and the agency has an expedited procedure to withdraw approvals.

A well-known example: the FDA granted accelerated approval to Biogen's Alzheimer's drug aducanumab (Aduhelm) in June 2021 based on amyloid plaque reduction, despite an advisory committee voting against it in November 2020.

Why it matters for traders

Accelerated approval can bring a drug to market years earlier, which is a big deal for a cash-burning biotech. Risks traders track:

  • Confirmatory trial results. A failure can lead to label withdrawal.
  • Payer pushback. Insurers may restrict coverage for drugs with unconfirmed benefit.
  • Endpoint debate. Whether the FDA accepts a given surrogate can make or break a filing.

Example

Northwind Therapeutics (NWTX) shows a 40% tumor response rate in a single-arm trial in a cancer with no good options. The FDA grants accelerated approval based on response rate, conditioned on a randomized trial measuring progression-free survival that is already enrolling.

FAQ

Is accelerated approval a full approval?

It is a real approval that allows marketing, but it is conditional on confirmatory trials, and the indication can be withdrawn if they fail.

What is a surrogate endpoint?

A measurable marker, such as tumor shrinkage or a blood test value, used in place of a direct measure of how patients feel, function or survive.

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Related terms

Primary endpointThe main outcome a clinical trial is designed and statistically powered to measure, used to judge whether the drug worked.Breakthrough therapy designationAn FDA designation that speeds development of a drug for a serious condition when early clinical data show substantial improvement over existing therapy.Fast track designationAn FDA designation that speeds development of drugs for serious conditions with unmet need, adding FDA contact and rolling review of the application.Priority reviewAn FDA designation that shortens the review goal for a drug application to 6 months instead of the standard 10 months.Phase 3 clinical trialThe large, late-stage trial that tests whether a drug works and is safe in a broad patient population, usually forming the basis for an FDA application.Biologics License Application (BLA)The application a company submits to the FDA to market a biologic, such as an antibody, vaccine or gene therapy, in the United States.

Part of the Quantisnow financial glossary. For longer explainers, see the investing guides. Educational content, not investment advice.