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  • Published In : Oct 2023
  • Code : CMI1200
  • Formats :
      Excel and PDF
  • Industry : Pharmaceutical

Rare diseases or orphan diseases are conditions that affect a small percentage of population at any given time. Orphan drug designation is given to the drugs indicated for the treatment of rare diseases. According to the U.S. Food & Drug Administration (FDA), an orphan disease is defined as a disease or condition affecting less than 200,000 people in the country. Orphan Drugs Act under the U.S. legislation allows manufacturer to request FDA to grant an orphan drug status to drugs intended to treat a rare disease or condition. According to the FDA, 2017, number of orphan drug designation requests have steadily increased over the past few years.

The global rare disease drugs market was not considered lucrative until recently, owing to various uncertainties associated with these diseases. Small percentage of affected population provided limited opportunity for study. Moreover, diseases were poorly understood, owing to huge differentiation and heterogeneity among the group of diseases which discouraged manufacturers to address unmet needs in this space. However, introduction of some highly priced orphan drugs e.g. spinal muscular atrophy drug Spinraza priced at US$ 125,000 per vial, and high unmet needs has drawn attention of manufacturers towards rare disease drugs market.

The total revenue of top 10 global rare disease drugs market for 2016 accounted for US$ 62.5 billion.

Competitive Analysis of top 10 organizations in the global rare disease drugs market

Top 10 organizations in the global rare disease drugs market in 2016, based on the 2016 revenue include:

  • Novartis AG
  • Hoffmann-La Roche Ltd.
  • Celgene Corporation
  • Bristol-Myers Squibb Company
  • Shire plc
  • Pfizer, Inc.
  • Sanofi S.A.
  • Bayer
  • Alexion Pharmaceuticals, Inc.
  • Biogen, Inc.
  1. Novartis AG is a global pharmaceutical organization with a diverse product folio ranging from drug therapies for cancer treatment to ophthalmic products. The company operates business through Innovative Medicines Division, which consists of oncology business unit and Novartis pharmaceutical business units. Innovative medicines Oncology division offers products for oncology diseases and rare diseases, and pharmaceutical business consists of ophthalmology, neuroscience, immunology and dermatology, respiratory, cardio-metabolic, and established medicines. Novartis lost patent protection for its orphan drug Gleevec for the treatment of chronic myeloid leukemia (CML) in 2015 and Sun Pharma brought out generic Gleevec in 2016 hampering the sales of Novartis for Gleevec.
  2. Celgene Corporation specializes in therapies for cancer treatment and inflammatory diseases which includes drugs such as Revlimid, Pomalyst, and Otezla. The company currently is concentrated on expanding the indication range of the present therapies and has a 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. Furthermore, the company has developed its own target-identification and drug-discovery technology platforms which enable the company to proceed rapidly from target identification and validation to lead identification and optimization.
  3. Shire Plc. became global leader in orphan drug products category (excluding oncology products) in year 2016 and grossed almost US$ 11 billion. The company acquired Baxalta in 2016, and its three therapeutic segments namely hematology, immunology, and oncology. Shire Plc. is also focused on providing therapies for rare diseases as it invested US$ 1.4 billion in research and development. The company had 37 therapies in research and the pipeline has further been strengthened by acquisition of Dyax and Baxalta in 2016.

Top 10 orphan drugs U.S. sales (US$ Mn) in rare disease drugs market (2016):

Orphan Drug Product Company U.S. Sales,(US$ million) 2016
Revlimid Celgene 4,417
Rituxan Roche 3,970
Copaxone Teva Pharm 3,257
Opdivo Bristol-Myers Squibb 2,664
Avonex Biogen 1,675
Imbruvica AbbVie 1,580
Sensipar Amgen 1,240
Gleevec Novartis 1,214
Velcade Takeda 1,133
Xyrem Jazz Pharmaceuticals 1,114


Global rare disease drugs market is impacted by various key trends which are driven by supply as well as demand side. Wide gap between market needs and potential market offerings is the main driving force behind an increasing interest of the manufacturers towards providing therapies in this segment of the healthcare industry. According to the U.S. Food & Drug Administration (FDA), in 2016, 582 requests for orphan drug designation were filed by the biopharma companies. Furthermore, the sales of orphan drug diseases has been on a rise due to availability of highly effective drug therapies. Moreover, potential drug therapies are present in pipeline for various cancers and rare diseases such as Niraparib by Tesaro, Inc. in phase I for the treatment of ovarian cancer, Emicizumab by Roche in phase III for the treatment of hemophilia, and LentiGlobin by Bluebird Bio in different phases for the treatment of sickle cell anemia and beta-thalassemia major. Positive results from these clinical trials are expected to change the outlook of the global rare disease drugs market over the foreseeable future.

This study provides a detailed competitive landscape and competitive analysis for leading players in this market. This will the aid one in understanding the operations of successful companies in this market and their long term goals.

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