Results 41 to 50 of about 110,217 (215)

Modality diversification and best-in-class small-molecule drugs: Recent trend of orphan drug development

Medicine in Drug Discovery, 2023
Only 5% of orphan diseases have approved drugs, leading to a high demand for new treatment. As a result, pharmaceutical companies have shifted their focus of drug development to orphan diseases.
Ryo Okuyama
doaj   +1 more source

Estimating the clinical cost of drug development for orphan versus non-orphan drugs

Orphanet Journal of Rare Diseases, 2019
Background High orphan drug prices have gained the attention of payers and policy makers. These prices may reflect the need to recoup the cost of drug development from a small patient pool.
Kavisha Jayasundara, Aidan Hollis, Murray Krahn, Muhammad Mamdani, Jeffrey S. Hoch, Paul Grootendorst   +5 more
doaj   +1 more source

Clinical pharmacology information in regulatory submissions and labeling: A comparative analysis of orphan and non‐orphan drugs approved by the FDA

Clinical and Translational Science, 2022
Clinical pharmacology is an integral discipline supporting the development, regulatory evaluation, and clinical use of drugs for the treatment of both common and rare diseases.
Julie Hsieh, Martina Sahre, Xinning Yang, Rajanikanth Madabushi, Anuradha Ramamoorthy   +4 more
doaj   +1 more source

Successful private–public funding of paediatric medicines research: lessons from the EU programme to fund research into off-patent medicines [PDF]

, 2014
The European Paediatric Regulation mandated the European Commission to fund research on off-patent medicines with demonstrated therapeutic interest for children.
Baiardi, P, Bonifazi, D, Bonifazi, F, Ceci, A, Chiron, C, Davies, E H, Eichler, I, Felisi, M, Giannuzzi, V, Giaquinto, C, Jacqz-Aigrain, E, Neubert, A, on behalf of the GRiP Consortium,, Pressler, R, Rabe, H, Ruggieri, L, Turner, M A, Whitaker, M J   +17 more
core   +1 more source

Profitability and Market Value of Orphan Drug Companies: A Retrospective, Propensity-Matched Case-Control Study.

PLoS ONE, 2016
BackgroundConcerns about the high cost of orphan drugs has led to questions being asked about the generosity of the incentives for development, and associated company profits.MethodsWe conducted a retrospective, propensity score matched study of publicly-
Dyfrig A Hughes, Jannine Poletti-Hughes
doaj   +1 more source

Class IIa HDACs forced degradation allows resensitization of oxaliplatin‐resistant FBXW7‐mutated colorectal cancer

Molecular Oncology, EarlyView.
HDAC4 is degraded by the E3 ligase FBXW7. In colorectal cancer, FBXW7 mutations prevent HDAC4 degradation, leading to oxaliplatin resistance. Forced degradation of HDAC4 using a PROTAC compound restores drug sensitivity by resetting the super‐enhancer landscape, reprogramming the epigenetic state of FBXW7‐mutated cells to resemble oxaliplatin ...
Vanessa Tolotto, Nicolò Gualandi, Ylenia Cortolezzis, Raffaella Picco, Monica Colitti, Francesca D'Este, Mariachiara Gani, Wayne W. Hancock, Giovanni Terrosu, Cristina Degrassi, Francesca Agostini, Claudio Brancolini, Luigi E. Xodo, Eros Di Giorgio   +13 more
wiley   +1 more source

Impacts of adjustment of National Reimbursement Drug List on orphan drugs volume and spending in China: an interrupted time series analysis

BMJ Open, 2023
Objective To evaluate the impacts of the 2017 adjustment of National Reimbursement Drug List (NRDL) on orphan drugs hospital procurement volumes and spending in China.Design We used an interrupted time series design covering the period from 2016 to 2018 ...
Yu Xie, Hua Xing, Zihao Wang, Hongbin Yi, Fenghao Shi, Liping Kuai, Dongyan Xu   +6 more
doaj   +1 more source

ATF4‐mediated stress response as a therapeutic vulnerability in chordoma

Molecular Oncology, EarlyView.
We screened 5 chordoma cell lines against 100+ inhibitors of epigenetic and metabolic pathways and kinases and identified halofuginone, a tRNA synthetase inhibitor. Mechanistically halofuginone induces an integrated stress response, with eIF2alpha phosphorylation, activation of ATF4 and its target genes CHOP, ASNS, INHBE leading to cell death ...
Lucia Cottone, James Dunford, Eleanor Calcutt, Vicki Gamble, Filiz Senbabaoglu Aksu, Lorena Ligammari, Georgina Wherry, Giorgia Gaeta, John C. Christianson, Adrienne M. Flanagan, Udo Oppermann, Adam P. Cribbs   +11 more
wiley   +1 more source

The photoswitchable cannabinoid azo‐HU308 enables optical control of Ca2+ dynamics in INS‐1 β‐cells via off‐target effects on TRPC channels

FEBS Open Bio, EarlyView.
Light activation of the photoswitchable cannabinoid ligand azo‐HU308 triggers Ca2+ influx in pancreatic β‐cells through TRPC channels, independent of CB2 cannabinoid receptors. This reveals a non‐GPCR pathway for cannabinoid modulation of β‐cell Ca2+ dynamics and establishes azo‐HU308 as an optical tool to study cannabinoid signaling through TRP ...
Alexander E. G. Viray, James A. Frank
wiley   +1 more source

Analysis of the first ten years of FDA’s rare pediatric disease priority review voucher program: designations, diseases, and drug development

Orphanet Journal of Rare Diseases
Background The Rare Pediatric Disease (RPD) Priority Review Voucher (PRV) Program was enacted in 2012 to support the development of new products for children.
Catherine Mease, Kathleen L. Miller, Lewis J. Fermaglich, Jeanine Best, Gumei Liu, Erika Torjusen   +5 more
doaj   +1 more source