What does NME stand for?

NME stands for New Molecular Entity — a drug with an active ingredient that has never been previously approved by the FDA. FDA's annual novel drug approvals list includes NMEs (small molecules approved via NDA) and novel BLAs (biologics approved via BLA), but excludes new formulations, new indications, generics, and biosimilars of already-approved active ingredients.

What is an FDA approval package?

When the FDA approves a new drug, the agency releases an 'approval package' on accessdata.fda.gov containing the documents that describe the basis for the decision. Typical contents include the medical review, statistical review, pharmacology review, chemistry review, the FDA-approved label, the approval letter, summary basis of approval, and various administrative correspondence.

What is the difference between an NDA and a BLA?

An NDA (New Drug Application) is the regulatory pathway for small-molecule drugs, reviewed by FDA's Center for Drug Evaluation and Research (CDER). A BLA (Biologics License Application) is the pathway for biologics — proteins, antibodies, cell therapies, gene therapies — reviewed by either CDER or the Center for Biologics Evaluation and Research (CBER). Application numbers follow different conventions: NDAs are typically six digits starting with 0 or 2, BLAs start with 1 or 7.

What is accelerated approval?

Accelerated approval is one of four FDA expedited-review programs (along with priority review, breakthrough therapy designation, and fast track). It allows the FDA to approve a drug based on a surrogate endpoint reasonably likely to predict clinical benefit, with the requirement that the sponsor conduct post-approval confirmatory trials. Accelerated approval is most common in oncology and rare disease.

Where do I find FDA approval-package documents?

Approval packages live on accessdata.fda.gov for CDER products and on per-product pages under fda.gov/vaccines-blood-biologics/ for CBER cell and gene therapies. The trove FDA index gives you the right approval-package URL for each of 218 novel drug approvals from 2021-2024 (192 CDER + 26 CBER) so you don't have to guess at it.

Are CBER cell and gene therapy approvals in trove's FDA index?

Yes, as of v0.2. Lenmeldy (atidarsagene autotemcel), Casgevy and Lyfgenia (sickle cell gene therapies), Beqvez, Hemgenix, Roctavian, Adstiladrin, and other recent CBER-regulated gene therapies are included alongside CDER novel drug approvals. Each row carries a regulatory_center column distinguishing CDER from CBER. The trove FDA index now covers 218 novel approvals from 2021-2024 — 192 CDER plus 26 CBER.

What is an FDA novel drug approval (NME)? Approval packages explained

Each year FDA publishes a curated list of Novel Drug Approvals — first-time approvals of drugs with active ingredients that have never been approved before. In 2024 the list had 50 drugs; in 2023, 55; 2022, 37; 2021, 50. These are mostly NMEs (New Molecular Entities, approved via NDA) plus a handful of novel BLAs (Biologics License Applications for first-of-kind biologics). The list excludes generics, biosimilars, new formulations of existing drugs, and supplemental approvals adding indications to already-approved drugs.

The list is what most public discourse about "what drugs got approved this year" refers to. It's also the cohort with the largest, most-detailed approval packages on accessdata.fda.gov.

CDER vs. CBER. The Novel Drug Approvals page is curated by CDER (Center for Drug Evaluation and Research), which reviews small-molecule drugs and most antibody biologics. Cell and gene therapy products — Lenmeldy, Casgevy, Lyfgenia, Beqvez, Hemgenix, Roctavian, and other recent gene therapies — are regulated by CBER (Center for Biologics Evaluation and Research) and appear on a separate FDA page. As of v0.2, trove ingests both. Each row carries a regulatory_center column distinguishing CDER from CBER.

What an approval is

When the FDA approves a new drug for marketing in the U.S., several things happen at once:

FDA sends an approval letter to the sponsor authorizing the drug to be marketed for the indication(s) listed.
FDA finalizes the label (also called prescribing information) — the legally binding description of the drug's indications, dosing, adverse reactions, warnings, contraindications, and use in specific populations.
FDA releases the approval package — the agency's internal review documents — on accessdata.fda.gov, usually within a few weeks to a few months of approval.

What's in an FDA approval package

The approval package is the most detailed public record of why a drug got approved. Typical contents:

Medical Review. Written by the medical officer who reviewed the application. Discusses the disease, the clinical trials, the efficacy endpoints, the adverse events, and the medical officer's recommendation. For most questions of the form "what was the basis for approval?" the medical review is the right document.
Statistical Review. Written by the statistician. Analyzes the trial design, primary and secondary endpoints, subgroup analyses, missing-data handling, and the statistical evidence supporting the efficacy claims.
Pharmacology Review. Covers pharmacokinetics, pharmacodynamics, ADME (absorption, distribution, metabolism, excretion), drug-drug interactions, and dose justification.
Chemistry Review (CMC). Manufacturing, formulation, stability, specifications. Most clinically interesting questions don't require the chemistry review, but it's where you'd look to understand the manufacturing process.
Summary Basis of Approval / Cross-Discipline Team Leader Review. A condensed integrated review that the FDA team leader writes summarizing the cross-disciplinary view.
Administrative documents. Approval letter, label, post-marketing requirements, REMS (Risk Evaluation and Mitigation Strategy) documents.

A common confusion: the label is what the drug company writes (subject to FDA approval). The medical and statistical reviews are what FDA reviewers write. The label is what's on Drugs.com and in clinical decision-support tools; the reviews tell you why the FDA agreed with what's on the label. They often disagree about emphasis, especially for adverse events and subgroup efficacy.

Regulatory pathways

FDA has several expedited-review programs, often combined for the same drug:

Standard review — 10-month FDA review clock from filing.
Priority review — 6-month review clock for drugs that would offer significant improvement in safety or effectiveness.
Breakthrough therapy designation — applies during development; gives the sponsor more FDA interaction.
Fast track — also during development; allows rolling review of the application.
Accelerated approval — approval based on a surrogate endpoint reasonably likely to predict clinical benefit, with required confirmatory trials.

The specific pathways used for a given approval are documented in the medical review and the approval letter.

How to find an approval's documents

FDA's front door for CDER products is the Drugs@FDA database at accessdata.fda.gov/scripts/cder/daf/. Each approval has an application-overview page that lists every document FDA released — original approval plus any post-approval supplements. The application-overview URL has the form ?event=overview.process&ApplNo=XXXXXX. For CBER cell and gene therapies, each product has its own page under fda.gov/vaccines-blood-biologics/ that links to the package insert, approval letter, clinical review memo, and Summary Basis for Regulatory Action. The trove FDA index gives you the right URL for each of 218 novel drug approvals from 2021–2024 (192 CDER + 26 CBER) so you don't have to guess at it.

What's not on FDA's approval-package page

Raw clinical trial data. Approval packages summarize the trials. The trial protocols and clinical-trial data themselves live on ClinicalTrials.gov for the registered trials.
Post-approval safety surveillance. Once a drug is on the market, adverse event reports go to FAERS. The approval package describes pre-approval safety; FAERS describes real-world safety.
The reviewer's view of competing drugs. Some medical reviews include comparative tables, but FDA can't approve a drug "vs." another drug — only against the legal standard of safety and efficacy for the proposed indication.
Internal deliberations and dissents. Many approvals involve internal disagreement among reviewers. The published reviews reflect the final agency position, sometimes with redacted dissent.

How to use FDA approval-package data

Use trove's FDA novel drug approvals index — search any of 218 approvals from 2021–2024 (CDER NMEs + CBER cell/gene therapies) and click straight through to the approval package: /drugs/. Each drug has its own permalink, e.g. /drugs/lenmeldy/, /drugs/leqembi/, /drugs/casgevy/.
Install the fda-analyst Claude Code skill to ask natural-language questions like "What was the basis for Lenmeldy's approval?" or "What adverse events did the FDA flag in the Elahere medical review?" The skill fetches and reads the relevant PDF at query time.
Browse Drugs@FDA directly at accessdata.fda.gov if you want to dig through everything yourself.

Citations

U.S. Food and Drug Administration, Novel Drug Approvals at FDA.
U.S. Food and Drug Administration, Drugs@FDA database.
FDA expedited programs: Fast Track, Breakthrough Therapy, Accelerated Approval, Priority Review.
FDA documents are U.S. government work, public domain.