RegulatoryGlossary Term
A regulatory submission to the FDA requesting approval to market a biological product in the United States, demonstrating that the product meets standards for safety, purity, and potency.
The Biologics License Application is the regulatory pathway for approval of biological products, a category that includes vaccines, blood products, gene therapies, cellular therapies, and therapeutic proteins produced through biotechnology. Unlike chemically synthesized drugs covered by NDAs, biological products are derived from living organisms and characterized by their manufacturing process as much as by their molecular structure. The BLA provides evidence that the biological product is safe, pure, and potent for its intended use.
The BLA shares the same Common Technical Document format as the NDA, but with additional emphasis on manufacturing characterization reflecting the complexity of biological products. Because slight variations in manufacturing can alter the biological activity of these products, the process is considered part of the product, requiring detailed description and control of all aspects of production. The BLA must demonstrate that manufacturing processes consistently produce a product meeting established specifications and that appropriate testing ensures each lot meets release criteria before distribution.
FDA review of BLAs involves both the Center for Biologics Evaluation and Research (CBER) and, for certain therapeutic biologics, the Center for Drug Evaluation and Research (CDER). The review evaluates the totality of evidence regarding safety and efficacy from clinical trials, the adequacy of manufacturing processes and controls, and the appropriateness of proposed labeling. Facility inspections verify that actual manufacturing conditions match the described processes and that current Good Manufacturing Practice regulations are followed. Upon approval, the biological product receives a license number, and each manufacturing facility must be separately licensed.
Therapeutic protein approval
"The BLA for the monoclonal antibody included extensive manufacturing characterization demonstrating consistent production of the glycoprotein across multiple production batches and three manufacturing facilities."
Cell therapy approval
"The CAR-T cell therapy BLA presented data from two pivotal trials showing durable responses in patients with relapsed lymphoma, along with detailed procedures for cell collection, modification, and infusion."
A premarket submission to the FDA demonstrating that a medical device is substantially equivalent to a legally marketed predicate device and therefore does not require premarket approval.
An FDA pathway allowing approval of drugs for serious conditions based on a surrogate endpoint or intermediate clinical endpoint reasonably likely to predict clinical benefit, with post-marketing requirements to confirm the expected benefit.
An FDA program for drugs intended to treat serious conditions where preliminary clinical evidence indicates substantial improvement over available therapies on clinically significant endpoints.
An FDA program designed to expedite the development and review of drugs intended to treat serious conditions and fill an unmet medical need, providing increased communication with FDA and eligibility for Rolling Review.
An FDA approval that allows an investigational medical device to be used in a clinical study to collect safety and effectiveness data required to support a premarket approval application or 510(k) submission.
A regulatory submission to the FDA requesting approval to market a biological product in the United States, demonstrating that the product meets standards for safety, purity, and potency.
The Biologics License Application is the regulatory pathway for approval of biological products, a category that includes vaccines, blood products, gene therapies, cellular therapies, and therapeutic proteins produced through biotechnology. Unlike chemically synthesized drugs covered by NDAs, biological products are derived from living organisms and characterized by their manufacturing process as much as by their molecular structure. The BLA provides evidence that the biological product is safe, pure, and potent for its intended use.
The BLA shares the same Common Technical Document format as the NDA, but with additional emphasis on manufacturing characterization reflecting the complexity of biological products. Because slight variations in manufacturing can alter the biological activity of these products, the process is considered part of the product, requiring detailed description and control of all aspects of production. The BLA must demonstrate that manufacturing processes consistently produce a product meeting established specifications and that appropriate testing ensures each lot meets release criteria before distribution.
FDA review of BLAs involves both the Center for Biologics Evaluation and Research (CBER) and, for certain therapeutic biologics, the Center for Drug Evaluation and Research (CDER). The review evaluates the totality of evidence regarding safety and efficacy from clinical trials, the adequacy of manufacturing processes and controls, and the appropriateness of proposed labeling. Facility inspections verify that actual manufacturing conditions match the described processes and that current Good Manufacturing Practice regulations are followed. Upon approval, the biological product receives a license number, and each manufacturing facility must be separately licensed.
Therapeutic protein approval
"The BLA for the monoclonal antibody included extensive manufacturing characterization demonstrating consistent production of the glycoprotein across multiple production batches and three manufacturing facilities."
Cell therapy approval
"The CAR-T cell therapy BLA presented data from two pivotal trials showing durable responses in patients with relapsed lymphoma, along with detailed procedures for cell collection, modification, and infusion."
A premarket submission to the FDA demonstrating that a medical device is substantially equivalent to a legally marketed predicate device and therefore does not require premarket approval.
An FDA pathway allowing approval of drugs for serious conditions based on a surrogate endpoint or intermediate clinical endpoint reasonably likely to predict clinical benefit, with post-marketing requirements to confirm the expected benefit.
An FDA program for drugs intended to treat serious conditions where preliminary clinical evidence indicates substantial improvement over available therapies on clinically significant endpoints.
An FDA program designed to expedite the development and review of drugs intended to treat serious conditions and fill an unmet medical need, providing increased communication with FDA and eligibility for Rolling Review.
An FDA approval that allows an investigational medical device to be used in a clinical study to collect safety and effectiveness data required to support a premarket approval application or 510(k) submission.