Top 10 Organizations Competitive Analysis In The Global Rare Disease Drugs Market

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

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Top 10 organizations in the global rare disease drugs market in 2016:

  1. Novartis AG

  2. Hoffmann-La Roche Ltd.

  3. Celgene Corporation

  4. Bristol-Myers Squibb Company

  5. Shire plc

  6. Pfizer, Inc.

  7. Sanofi S.A.

  8. Bayer

  9. Alexion Pharmaceuticals, Inc.

  10. Biogen, Inc.

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 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.

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