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An orphan drug is a pharmaceutical agent that is developed to treat certain rare medical conditions. An orphan drug would not be profitable to produce without government assistance, due to the small population of patients affected by the conditions. The conditions that orphan drugs are used to treat are referred to as orphan diseases. The assignment of orphan status to a disease and to drugs developed to treat it is a matter of public policy that depends on the legislation of the country.
A cancer vaccine, or oncovaccine, is a vaccine that either treats existing cancer or prevents development of cancer. Vaccines that treat existing cancer are known as therapeutic cancer vaccines or tumor antigen vaccines. Some of the vaccines are "autologous", being prepared from samples taken from the patient, and are specific to that patient.
Avicine, tested and developed by AVI BioPharma, and also known as CTP-37 was trialled as a possible cancer vaccine to treat a number of different cancers. These included colorectal cancer, pancreatic cancer and prostate cancer. The treatment was trialled as and intended to be induced via intramuscular injection into the bloodstream, the location dependent on the treatment area.
Iduronidase, sold as Aldurazyme, is an enzyme with the systematic name glycosaminoglycan α-L-iduronohydrolase. It catalyses the hydrolysis of unsulfated α-L-iduronosidic linkages in dermatan sulfate.
Rintatolimod, sold under the tradename Ampligen, is a medication intended for treatment of myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS). There is some evidence it may improve some ME/CFS symptoms.
Blinatumomab, sold under the brand name Blincyto, and known informally as blina, is a biopharmaceutical medication used as a second-line treatment for Philadelphia chromosome-negative relapsed or refractory acute lymphoblastic leukemia. It belongs to a class of constructed monoclonal antibodies, bi-specific T-cell engagers (BiTEs), that exert action selectively and direct the human immune system to act against tumor cells. Blinatumomab specifically targets the CD19 antigen present on B cells. In December 2014, it was approved by the US Food and Drug Administration under the accelerated approval program; marketing authorization depended on the outcome of clinical trials that were ongoing at the time of approval. Blinatumomab is given via intravenous infusion.
Biovest International, Inc was a Minneapolis-based biotechnology company. Their active immunotherapy, BiovaxID, is a cancer vaccine whose first indication was intended to be consolidation/adjuvant therapy of follicular Non-Hodgkin's Lymphoma. Biovest filed to reorganize under chapter 11 bankruptcy in 2014, BiovaxID was refused European marketing authorization in 2015, and Biovest's stock listing was revoked in 2017.
Merck & Co., Inc. is an American multinational pharmaceutical company headquartered in Rahway, New Jersey, and is named for Merck Group, founded in Germany in 1668, of which it was once the American arm. The company does business as Merck Sharp & Dohme or MSD outside the United States and Canada. It is one of the largest pharmaceutical companies in the world, generally ranking in the global top five by revenue.
The Orphan Drug Act of 1983 is a law passed in the United States to facilitate development of orphan drugs—drugs for rare diseases such as Huntington's disease, myoclonus, ALS, Tourette syndrome or muscular dystrophy which affect small numbers of individuals residing in the United States.
Jennerex Biotherapeutics, Inc. was an American private biopharmaceutical company that developed the oncolytic viruses JX-594 and JX-929 among others. By creating oncolytic viruses that can (1) kill tumor cells directly through lysis, (2) activate the immune system by delivering genes that encode immunostimulants and by overcoming tumor cell-induced immunological tolerance, and (3) reduce tumor nutrient supply through the destruction of blood vessels, Jennerex aimed to create a novel approach to treating and possibly curing cancer.
Pembrolizumab, sold under the brand name Keytruda, is a humanized antibody used in cancer immunotherapy that treats melanoma, lung cancer, head and neck cancer, Hodgkin lymphoma, stomach cancer, cervical cancer, and certain types of breast cancer. It is administered by slow intravenous injection.
Kite Pharma is an American biotechnology company that develops cancer immunotherapy products with a primary focus on genetically engineered autologous CAR T cell therapy - a cell-based therapy which relies on chimeric antigen receptors and T cells. Founded in 2009, and based in Santa Monica, California, it was acquired by Gilead Sciences in 2017.
Arcturus Therapeutics Holdings Inc. is an American RNA medicines biotechnology company focused on the discovery, development and commercialization of therapeutics for rare diseases and infectious diseases. Arcturus has developed proprietary lipid nanoparticle RNA therapeutics for nucleic acid medicines including small interfering RNA (siRNA), messenger RNA (mRNA), gene editing RNA, DNA, antisense oligonucleotides, and microRNA.
Avelumab, sold under the brand name Bavencio, is a fully human monoclonal antibody medication for the treatment of Merkel cell carcinoma, urothelial carcinoma, and renal cell carcinoma.
Remune was the first therapeutic HIV vaccine based on the killed whole virus approach. Remune was initially invented by Jonas Salk in 1987 and was developed by Immune Response BioPharma, Inc. (IRBP)
Tonix Pharmaceuticals is a pharmaceutical company based in Chatham, New Jersey that focuses on repurposed drugs for central nervous system conditions and as of 2020 was also pursuing a vaccine for COVID-19 and a biodefense project.
YM BioSciences Inc. was a Canadian drug development company primarily focused on advancing CYT-387, an orally administered inhibitor of both the JAK1 and JAK2 kinases, which have been implicated in a number of hematological and immune cell disorders including myeloproliferative neoplasms and inflammatory diseases as well as certain cancers. YM BioSciences also had several preclinical programs underway with candidates from its library of compounds identified through internal research conducted at YM BioSciences Australia.
Axicabtagene ciloleucel, sold under the brand name Yescarta, is a medication used for the treatment for large B-cell lymphoma that has failed conventional treatment. T cells are removed from a person with lymphoma and genetically engineered to produce a specific T-cell receptor. The resulting chimeric antigen receptor T cells (CAR-Ts) that react to the cancer are then given back to the person to populate the bone marrow. Axicabtagene treatment carries a risk for cytokine release syndrome (CRS) and neurological toxicities.
bluebird bio, Inc., based in Somerville, Massachusetts, is a biotechnology company that develops gene therapies for severe genetic disorders.
Infigratinib, sold under the brand name Truseltiq, is an anti-cancer medication used to treat cholangiocarcinoma.
References
- 1 2 "Search Orphan Drug Designations and Approvals" . Retrieved 13 January 2015.
- 1 2 3 "Neurovax" . Retrieved 13 January 2015.
