Development perspectives for curative technologies in primary demyelinating disorders of the central nervous system with neuromyelitis optica spectrum disorder (NMOSD) and myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD) at the forefront

Pitter, János (author), Nagy, László (author), Nagy, Balázs (author), Hren, Rok (author)

Primary demyelinating disorders of the central nervous system (CNS) include multiple sclerosis and the orphan conditions neuromyelitis optica spectrum disorder (NMOSD) and myelin oligodendrocyte glycoprotein IgG-associated disease (MOGAD). Curative technologies under development aim to selectively block autoimmune reactions against specific autoantigens while preserving the responsiveness of the immune system to other antigens. Our analysis focused on target patient selection for such developments, carefully considering the relevant clinical, regulatory, and market-related aspects. We found that the selection of patients with orphan conditions as target populations offers several advantages. Treatments for orphan conditions are associated with limited production capacity, qualify for regulatory incentives, and may require significantly shorter and lower-scale clinical programs. Furthermore, they may meet a higher acceptable cost-effectiveness threshold in order to compensate for the low numbers of patients to be treated. Finally, curative technologies targeting orphan indications could enter less competitive markets with lower risk of generic price erosion and would benefit from additional market protection measures available only for orphan products. These advantages position orphan conditions and subgroups as the most attractive target indications among primary demyelinating disorders of the CNS. The authors believe that after successful proof-of-principle demonstrations in orphan conditions, broader autoimmune patient populations may also benefit from the success of these pioneering developments.

• Language: English
• Keywords: medicine, nevrology, diseases of the central nervous system, treatment
• Work type: Article
• Typology: 1.01 - Original Scientific Article
• Organization: FMF - Faculty of Mathematics and Physics
• Publication version: Version of Record
• Year: 2024
• Number of pages: 13 str.
• Numbering: Vol. 14, iss. 6, art. no. 599
• PID: 20.500.12556/RUL-158310
• UDC: 616.8
• ISSN on article: 2075-4426
• DOI: 10.3390/jpm14060599
• COBISS.SI-ID: 197720579
• Publication date in RUL: 04.06.2024

Record is a part of a journal

• Title: Journal of personalized medicine
• Shortened title: J. pers. med.
• Publisher: MDPI
• ISSN: 2075-4426
• COBISS.SI-ID: 31207641

Licences

• License: CC BY 4.0, Creative Commons Attribution 4.0 International
• Link: http://creativecommons.org/licenses/by/4.0/
• Description: This is the standard Creative Commons license that gives others maximum freedom to do what they want with the work as long as they credit the author.

Secondary language

• Language: Slovenian
• Keywords: medicina, nevrologija, bolezni centralnega živčnega sistema, zdravljenje

Projects

• Funder: EC - European Commission
• Project number: 101080562
• Name: ADVANCED ANTIGEN-SPECIFIC DENDRITIC CELL-BASED THERAPY TO RE-ESTABLISH TOLERANCE IN IMMUNE-MEDIATED DISEASES
• Acronym: IMMUTOL