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Orphan Drugs

13:42 EST 24th November 2017 | BioPortfolio

An orphan drug is a pharmaceutical product aimed at rare diseases or disorders. The development of orphan drugs has been financially incentivised through US law via the ‘Orphan Drug Act of 1983’. The National Organization for Rare Disorders (NORD), which was instrumental in establishing the 1983 Act, currently estimates 30 million Americans suffer from 7,000 rare diseases. Prior to the 1983 Act, 38 orphan drugs were approved. To date, 425 indication designations covering 347 drugs have been approved.

The success of the original Orphan Drug Act in the US led to it being adopted in other key markets, most notably in Japan in 1993 and the European Union in 2000.

Rare Disease Patient Populations are Defined in Law as:

  • USA: <200,000 patients (<6.37 in 10,000, based on US population of 314m)
  • EU: <5 in 10,000 (<250,000 patients, based on EU population of 506m)
  • Japan: <50,000 patients (<4 in 10,000 based on Japan population of 128m)

Projected top 30 Orphan Drugs

Product ~ Generic Name ~ Company
Rituxan ~ rituximab ~ Roche
Revlimid ~ lenalidomide ~ Celgene
Soliris ~ eculizumab ~ Alexion Pharmaceuticals
Afinitor ~ everolimus ~ Novartis
Tasigna ~ nilotinib hydrochloride ~ Novartis
Velcade ~ bortezomib ~ J&J/Takeda
Avonex ~ interferon beta-1a ~ Biogen Idec
Alimta ~ pemetrexed disodium ~ Eli Lilly
Yervoy ~ ipilimumab ~ Bristol-Myers Squibb
Sprycel ~ dasatinib ~ Bristol-Myers Squibb
Rebif ~ interferon beta-1a ~ Merck/Pfizer
Kalydeco ~ ivacaftor ~ Vertex Pharmaceuticals
Jakavi ~ ruxolitinib phosphate ~ Incyte/Novartis
Sutent ~ sunitinib malate ~ Pfizer
Kyprolis ~ carfilzomib ~ Onyx Pharmaceuticals
Kogenate ~ octocog alfa ~ Bayer
NovoSeven ~ eptacog alfa ~ Novo Nordisk
Nexavar ~ sorafenib tosylate ~ Bayer/Onyx
Copaxone ~ glatiramer acetate ~ Teva/Sanofi
Ibrutinib ~ ibrutinib ~ Pharmacyclics
Cerezyme ~ imiglucerase ~ Sanofi
Myozyme ~ alglucosidase alfa ~ Sanofi
Gleevec ~ imatinib mesylate ~ Novartis
Sandostatin LAR ~ octreotide acetate ~ Novartis
Exjade ~ deferasirox ~ Novartis
Opsumit ~ macitentan ~ Actelion
Xalkori ~ crizotinib ~ Pfizer
Xyrem ~ sodium oxybate ~ Jazz Pharmaceuticals
Pomalyst ~ pomalidomide ~ Celgene

Source: Evalute Pharma

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