GlobalRare Disease Drugs Market is impacted by various key trends which aredriven by supply as well as demand side. Wide gap between market needs andpotential market offerings is the main driving force behind an increasinginterest of the manufacturers towards providing therapies in this segment ofthe healthcare industry. According to the U.S. Food & Drug Administration(FDA), in 2016, 582 requests for orphan drug designation were filed by thebiopharma companies. Furthermore, the sales of orphan drug diseases has been ona rise due to availability of highly effective drug therapies. Moreover,potential drug therapies are present in pipeline for various cancers and rarediseases such as Niraparib by Tesaro, Inc. in phase I for the treatment ofovarian cancer, Emicizumab by Roche in phase III for the treatment ofhemophilia, and LentiGlobin by Bluebird Bio in different phases for thetreatment of sickle cell anemia and beta-thalassemia major. Positive resultsfrom these clinical trials are expected to change the outlook of the globalrare disease drugs market over the foreseeable future.
Rare diseases or orphan diseasesare conditions that affect a small percentage of population at any given time.Orphan drug designation is given to the drugs indicated for the treatment ofrare diseases. According to the U.S. Food & Drug Administration (FDA), anorphan disease is defined as a disease or condition affecting less than 200,000people in the country. Orphan Drugs Act under the U.S. legislation allowsmanufacturer to request FDA to grant an orphan drug status to drugs intended totreat a rare disease or condition. According to the FDA, 2017, number of orphandrug designation requests have steadily increased over the past few years.
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The global rare disease drugsmarket was not considered lucrative until recently, owing to variousuncertainties associated with these diseases. Small percentage of affectedpopulation provided limited opportunity for study. Moreover, diseases werepoorly understood, owing to huge differentiation and heterogeneity among thegroup of diseases which discouraged manufacturers to address unmet needs inthis space. However, introduction of some highly priced orphan drugs e.g.spinal muscular atrophy drug Spinraza priced at US$ 125,000 per vial, and highunmet needs has drawn attention of manufacturers towards rare disease drugsmarket.
Novartis AG is a globalpharmaceutical organization with a diverse product folio ranging from drugtherapies for cancer treatment to ophthalmic products. The company operatesbusiness through Innovative Medicines Division, which consists of oncologybusiness unit and Novartis pharmaceutical business units. Innovative medicinesOncology division offers products for oncology diseases and rare diseases, andpharmaceutical business consists of ophthalmology, neuroscience, immunology anddermatology, respiratory, cardio-metabolic, and established medicines. Novartislost patent protection for its orphan drug Gleevec for the treatment of chronicmyeloid leukemia (CML) in 2015 and Sun Pharma brought out generic Gleevec in2016 hampering the sales of Novartis for Gleevec.
Celgene Corporation specializesin therapies for cancer treatment and inflammatory diseases which includesdrugs such as Revlimid, Pomalyst, and Otezla. The company currently isconcentrated on expanding the indication range of the present therapies and hasa robust pipeline for oncology conditions. For instance, Revlimid(lenalidomide), in phase III clinical trials for diffuse large B-cell lymphoma,indolent lymphoma, and follicular lymphoma.
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Furthermore, the company hasdeveloped its own target-identification and drug-discovery technology platformswhich enable the company to proceed rapidly from target identification andvalidation to lead identification and optimization.
Shire Plc. became global leaderin orphan drug products category (excluding oncology products) in year 2016 andgrossed almost US$ 11 billion. The company acquired Baxalta in 2016, and itsthree therapeutic segments namely hematology, immunology, and oncology. ShirePlc. is also focused on providing therapies for rare diseases as it investedUS$ 1.4 billion in research and development. The company had 37 therapies inresearch and the pipeline has further been strengthened by acquisition of Dyaxand Baxalta in 2016.
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