Dynamics of patents, orphan drug designation, licensing, and revenues from drugs for rare diseases: The market expansion of eculizumab
Dynamics of patents, orphan drug designation, licensing, and revenues from drugs for rare diseases: The market expansion of eculizumab
Data
2021
Autores
Caetano, Rosângela
Correa, Marilena Cordeiro Dias Villela
Villardi, Pedro
Rodrigues, Paulo Henrique Almeida
Osorio-de-Castro, Claudia Garcia Serpa
Journal Title
Journal ISSN
Volume Title
Publisher
PLOS ONE
Resumo
Background
This study examines the dynamics of the eculizumab patenting, orphan designation, and
marketing authorization process in different countries and regulatory systems and analyzes
drug revenues since its first marketing authorization.
Methods
A retrospective case study was conducted. Multiple information sources were used to:
determine the status of eculizumab patents; examine the designation of orphan drug status
by US, European, Japanese, and Brazilian regulatory authorities to determine registration
status and approved clinical indications; estimate the prevalence of associated clinical con-
ditions; investigate the history of the drug manufacturer, Alexion Pharmaceuticals, Inc., and
its financialized business model; and examine global eculizumab sales revenues since its
first marketing authorization.
Results
Our search yielded 32 patent families divided into 98 applications. The first patent granted
was filed in 1995 by Alexion Pharmaceuticals, Inc. in the US. Eculizumab has always been
as an orphan drug, except in the Brazilian regulatory agency. All clinical indications
approved thus far refer to rare diseases (e.g., paroxysmal nocturnal hemoglobinuria syn-
drome, atypical hemolytic-uremic syndrome, refractory and generalized myasthenia gravis,
and neuromyelitis optica spectrum disorder). Alexion’s revenues amounted to more than US
$25 billion between 2007 and 2019, showing a growing trend. Eculizumab led sales from the beginning, being the only product in the company’s portfolio until 2015. In 2019, the drug
accounted for 79.1% of all revenues.
Discussion
Our findings show that a strategy focused on obtaining orphan drug designation, expanding
therapeutic indications and the geographic range of marketing approvals, extending monop-
oly periods, and prioritizing public procurement niches has enhanced revenues and helped
the company achieve leadership in a highly specific and profitable market.
Description
Palavras-chave
Antibodies, Monoclonal, Humanized*, Brazil, Drug Approval*, Humans, Orphan Drug Production*, Retrospective Studies
Citação
Caetano R, Cordeiro Dias Villela Correa M, Villardi P, Almeida Rodrigues PH, Garcia Serpa Osorio-de-Castro C. Dynamics of patents, orphan drug designation, licensing, and revenues from drugs for rare diseases: The market expansion of eculizumab. PLoS One. 2021 Mar 5;16(3):e0247853. doi: 10.1371/journal.pone.0247853.