Product Code Database
A biopharmaceutical, also known as a biological medical product, or biologic, is any pharmaceutical drug product manufactured in, extracted from, or semisynthesis from biology sources. Different from total synthesis pharmaceuticals, they include , whole blood, blood components, , , gene therapy, tissues, recombinant therapeutic protein, and used in cell therapy. Biopharmaceuticals can be composed of , , , or complex combinations of these substances, or may be living cells or tissues. They (or their precursors or components) are isolated from life sources—human, animal, plant, fungal, or microbial. They can be used in both human and animal medicine.
Terminology surrounding biopharmaceuticals varies between groups and entities, with different terms referring to different subsets of therapeutics within the general biopharmaceutical category. The term biologics is often used more restrictively to mean biopharmaceuticals that are produced using recombinant DNA technology. Some regulatory agencies use the terms biological medicinal products or therapeutic biological product to refer specifically to engineered macromolecule products like protein- and nucleic acid-based , distinguishing them from products like blood, blood components, or vaccines, which are usually extracted directly from a biological source. Biopharmaceutics is pharmaceutics that works with biopharmaceuticals. Biopharmacology is the branch of pharmacology that studies biopharmaceuticals. Specialty drugs, a recent classification of pharmaceuticals, are high-cost drugs that are often biologics. The European Medicines Agency uses the term advanced therapy medicinal products (ATMPs) for medicines for human use that are "based on genes, cells, or tissue engineering", including gene therapy medicines, somatic-cell therapy medicines, tissue-engineered medicines, and combinations thereof. Within EMA contexts, the term advanced therapies refers specifically to ATMPs, although that term is rather nonspecific outside those contexts.
Gene-based and cellular biologics, for example, often are at the forefront of biomedicine and biomedical research, and may be used to treat a variety of medical conditions for which no other treatments are available.
Building on the market approvals and sales of recombinant virus-based biopharmaceuticals for veterinary and human medicine, the use of engineered plant viruses has been proposed to enhance crop performance and promote sustainable production.
In some jurisdictions, biologics are regulated via different pathways from other small molecule drugs and .
Some biologics that were previously extracted from animals, such as insulin, are now more commonly produced by recombinant DNA.
Biologics as a class of medications in this narrower sense have had a profound impact on many medical fields, primarily rheumatology and oncology, but also cardiology, dermatology, gastroenterology, neurology, and others. In most of these disciplines, biologics have added major therapeutic options for treating many diseases, including some for which no effective therapies were available, and others where previously existing therapies were inadequate. However, the advent of biologic therapeutics has also raised complex regulatory issues (see below), and significant pharmacoeconomic concerns because the cost for biologic therapies has been dramatically higher than for conventional (pharmacological) medications. This factor has been particularly relevant since many biological medications are used to treat chronic diseases, such as rheumatoid arthritis or inflammatory bowel disease, or for the treatment of otherwise untreatable cancer during the remainder of life. The cost of treatment with a typical monoclonal antibody therapy for relatively common indications is generally in the range of €7,000–14,000 per patient per year.
Older patients who receive biologic therapy for diseases such as rheumatoid arthritis, psoriatic arthritis, or ankylosing spondylitis are at increased risk for life-threatening infection, adverse cardiovascular events, and malignancy.
The first such substance approved for therapeutic use was biosynthetic "human" insulin made via recombinant DNA. Sometimes referred to as rHI, under the trade name Humulin, was developed by Genentech, but licensed to Eli Lilly and Company, who manufactured and marketed it starting in 1982.
Major kinds of biopharmaceuticals include:
Research and development investment in new medicines by the biopharmaceutical industry stood at $65.2 billion in 2008. A few examples of biologics made with recombinant DNA technology include:
| abatacept | Orencia | rheumatoid arthritis | immunoglobin CTLA-4 fusion protein | T-cell deactivation |
| adalimumab | Humira | rheumatoid arthritis, ankylosing spondylitis, psoriatic arthritis, psoriasis, ulcerative colitis, Crohn's disease | monoclonal antibody | TNF antagonist |
| alefacept | Amevive | chronic plaque psoriasis | immunoglobin G1 fusion protein | incompletely characterized |
| erythropoietin | Epogen | anemia arising from cancer chemotherapy, chronic renal failure, etc. | recombinant protein | stimulation of red blood cell production |
| etanercept | Enbrel | rheumatoid arthritis, ankylosing spondylitis, psoriatic arthritis, psoriasis | recombinant human TNF-receptor fusion protein | TNF antagonist |
| infliximab | Remicade | rheumatoid arthritis, ankylosing spondylitis, psoriatic arthritis, psoriasis, ulcerative colitis, Crohn's disease | monoclonal antibody | TNF antagonist |
| trastuzumab | Herceptin | breast cancer | humanized monoclonal antibody | HER2/neu (erbB2) antagonist |
| ustekinumab | Stelara | psoriatic arthritis, psoriasis, ulcerative colitis, Crohn's disease | humanized monoclonal antibody | IL-12 and IL-23 antagonist |
| denileukin diftitox | Ontak | cutaneous T-cell lymphoma (CTCL) | Diphtheria toxin engineered protein combining Interleukin-2 and Diphtheria toxin | Interleukin-2 receptor binder |
| golimumab | Simponi | rheumatoid arthritis, psoriatic arthritis, ankylosing spondylitis, ulcerative colitis | monoclonal antibody | TNF antagonist |
| vedolizumab | Entyvio | ulcerative colitis, Crohn's disease | monoclonal antibody | α4β7 integrin blocker |
| ixekizumab | Taltz | plaque psoriasis, psoriatic arthritis, ankylosing spondylitis, non-radiographic axial spondyloarthritis | humanized monoclonal antibody | IL-17A neutralizer |
Viral gene therapies using engineered plant viruses have been proposed to enhance crop performance and promote sustainable production.
Biosimilars require a different regulatory framework compared to small-molecule generics. Legislation in the 21st century has addressed this by recognizing an intermediate ground of testing for biosimilars. The filing pathway requires more testing than for small-molecule generics, but less testing than for registering completely new therapeutics.
In 2003, the European Medicines Agency introduced an adapted pathway for biosimilars, termed similar biological medicinal products. This pathway is based on a thorough demonstration of comparability of the product to an existing approved product. Within the United States, the Patient Protection and Affordable Care Act of 2010 created an abbreviated approval pathway for biological products shown to be biosimilar to, or interchangeable with, an FDA-licensed reference biological product.; Researchers are optimistic that the introduction of biosimilars will reduce medical expenses to patients and the healthcare system.
One potential approach to this technology is the creation of a transgenic mammal that can produce the biopharmaceutical in its milk, blood, or urine. Once an animal is produced, typically using the pronuclear microinjection method, it becomes efficacious to use cloning technology to create additional offspring that carry the favorable modified genome. The first such drug manufactured from the milk of a genetically modified goat was ATryn, but marketing permission was blocked by the European Medicines Agency in February 2006. This decision was reversed in June 2006 and approval was given August 2006.
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