SPECIALTY RX FACTS

27Sep/100

BLA vs NDA What are the differences?

We are seeing more and more specialty products coming to market via the Biologic License Application ( BLA) route, so what are the key differences?  For a layman's definition look below:

"After clinical trials have shown that the new agent is safe and effective, there is reason to make the agent generally available to patients and physicians. The formal process in the U.S. by which this occurs is the approval by FDA of a marketing application (New Drug Application for cytotoxic/cytostatic agents or a Biologic License Application for biological agents) submitted by a private firm.  The applicant seeks approval from FDA for one or more specific indication(s). Review and approval of an NDA or BLA are based on the demonstration of safety and efficacy assessed from detailed reports of the clinical trials; particularly randomized controlled studies. The contribution of a new agent in the treatment of a disease is demonstrated unambiguously if the agent is the only variable between the treatments."

I'll add something below from fda.gov

"In june 30, 2003 FDA transferred some of the therapeutic biological products that had been reviewed and regulated by the Center for Biologics Evaluation and Research (CBER) to the Center for Drug Evaluation and Research (CDER).

Categories of Biological Products Transferred to CDER :

>Monoclonal antibodies for in vivo use.
>Proteins intended for therapeutic use, including cytokines (e.g. interferons), enzymes (e.g. thrombolytics), and other novel proteins, except for those that are specifically assigned to CBER (e.g., vaccines and blood products). This category includes therapeutic proteins derived from plants, animals, or microorganisms, and recombinant versions of these products.
>Immunomodulators: proteins or peptides that are not antigen specific (e.g., cytokines, growth factors, chemokines, etc.) that are intended to treat disease by inhibiting or modifying a pre-exisiting immune response; and proteins or peptides intended to act in antigen-specific fashion to treat or prevent autoimmune diseases by inhibiting or modifying pre-existing immune responses.
>Growth factors, cytokines, and monoclonal antibodies intended to mobilize, stimulate, decrease or otherwise alter the production of cells in vivo.1 This category includes growth factors, cytokines, and monoclonal antibodies, as well as non-biological agents, administered as mobilizing agents for their direct therapeutic effect on the recipient, as well as growth factors, cytokines, and monoclonal antibodies administered for the purpose of subsequently harvesting the mobilized, stimulated, decreased or otherwise altered cells for use in a human cellular or tissue-based product (HCT/P).

Categories of Biological Products Remaining in CBER

>Cellular products, including products composed of human, bacterial or animal cells (such as pancreatic islet cells for transplantation), or from physical parts of those cells (such as whole cells, cell fragments, or other components intended for use as preventative or therapeutic vaccines).
>Gene therapy products. Human gene therapy/gene transfer is the administration of nucleic acids, viruses, or genetically engineered microorganisms that mediate their effect by transcription and/or translation of the transferred genetic material, and/or by integrating into the host genome. Cells may be modified in these ways ex vivo for subsequent administration to the recipient, or altered in vivo by gene therapy products administered directly to the recipient.
>Vaccines and vaccine-associated products: products, regardless of their composition or method of manufacture, intended to induce or enhance a specific immune response to prevent or treat a disease or condition, or to enhance the activity of other therapeutic interventions.
>Allergenic extracts used for the diagnosis and treatment of allergic diseases and allergen patch tests.
>Antitoxins, antivenins, and venoms
>Blood, blood components, plasma derived products (for example, albumin, immunoglobulins, clotting factors, fibrin sealants, proteinase inhibitors), including recombinant and transgenic versions of plasma derivatives, (for example clotting factors), blood substitutes, plasma volume expanders, human or animal polyclonal antibody preparations including radiolabeled or conjugated forms, and certain fibrinolytics such as plasma-derived plasmin, and red cell reagents.
>Human cells, tissues and cellular and tissue-based products (HCT/P’s). This category includes HCT/P’s containing cells that have been harvested following in vivo administration of a CDER-regulated growth factor, cytokine, or monoclonal antibody,2 as well as HCT/P’s requiring ex vivo manipulation.

For more information go to:  http://www.fda.gov/Drugs/informationondrugs/ucm079436.htm

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