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Table 1.

Selected aspects related to rare diseases and orphan drugs according to jurisdiction.

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Table 1 Expand

Fig 1.

Active and pending eculizumab patents deposited at patent and trademark offices in the United States, European Union, Japan, and Brazil up to 2019.

Note: Fig 1 provides an overall view of the duration of protection granted to each active patent in each jurisdiction (illustrated by darker horizontal lines) and the estimated duration of pending patents (illustrated by lighter horizontal lines). The patent numbers are displayed in the right column and the duration of protection (in year) is shown at the top.

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Fig 1 Expand

Fig 2.

Timeline of eculizumab orphan drug designation and marketing authorization in international regulatory agencies according to year and indication, 2007–2019.

Paroxysmal Nocturnal Hemoglobinuria (PNH), Atypical Hemolytic Uremic Syndrome (aHUS), Generalized Myasthenia Gravis (gMG), Neuromyelitis Optica Spectrum Disorder (NMOSD), FDA–Food and Drug Administration; EMA–European Medicines Agency; PMDA—Pharmaceutical and Medical Devices Agency; ANVISA–Brazilian Health Surveillance Agency.

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Fig 2 Expand

Table 2.

Prevalence of approved indications for eculizumab (per 100,000).

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Table 2 Expand

Fig 3.

Eculizumab and total revenues of Alexion Pharmaceuticals, Inc. (million US$), 2007–2019.

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Fig 3 Expand

Fig 4.

Eculizumab revenues (in million US$) and total number of indications approved by international regulatory agencies, 2007–2019.

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Fig 4 Expand

Fig 5.

Total eculizumab sales revenue in Brazil and worldwide (in millions of US$).

Alexion Pharmaceuticals, Inc., 2007–2019.

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Fig 5 Expand