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The global orphan drugs market was valued at US$ 168.9 Billion in 2021.
We expect the global orphan drugs market to exhibit a CAGR of 12.3% during 2022-2027.
The rising demand for orphan drugs for the diagnosis, prevention, and treatment of rare medical
disorders and cater to public health requirement is primarily driving the global orphan drugs market.
The sudden outbreak of the COVID-19 pandemic had led to postponement in the treatment of several
rare medical ailments to reduce the risk of the coronavirus infection upon hospital visits or interaction
with healthcare professionals, thereby limiting the demand for orphan drugs.
Based on the drug type, the global orphan drugs market has been segmented into biological and non-
biological. Currently, biological holds the majority of the total market share.
Based on the disease type, the global orphan drugs market can be divided into oncology, hematology,
neurology, cardiovascular, and others. Among these, oncology currently exhibits a clear dominance in
Based on the top selling drugs, the global orphan drugs market has been categorized into Revlimid,
Rituxan, Copaxone, OPDIVO, Keytruda, Imbruvica, Avonex, Sensipar, Soliris, and others. Currently,
Keytruda accounts for the majority of the global market share.
Based on the distribution channel, the global orphan drugs market can be segregated into hospital
pharmacies, retail pharmacies, online stores, and others. Among these, hospital pharmacies currently
hold the largest market share.
On a regional level, the market has been classified into North America, Asia Pacific, Europe, Latin
America, and Middle East and Africa, where North America currently dominates the global market.
Some of the major players in the global orphan drugs market include AbbVie Inc., Alexion
Pharmaceuticals Inc., Amgen Inc., Biogen Inc., Bristol-Myers Squibb Company, F. Hoffmann-La Roche AG
(Roche Holding AG), Jazz Pharmaceuticals Plc, Johnson & Johnson, Merck & Co. Inc., Novartis AG, Pfizer
Inc., Sanofi S.A., Takeda Pharmaceutical Company Limited, and Teva Pharmaceutical Industries Ltd.
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