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

Haendel, 2020, How many rare diseases are there?, Nat Rev Drug Discov, 19, 77, 10.1038/d41573-019-00180-y Scherman, 2020, Drug repositioning for rare diseases: Knowledge-based success stories, Therapie, 75, 161, 10.1016/j.therap.2020.02.007 Phillips, 2013, Big Pharma's new model in orphan drugs and rare diseases, Expert Opin Orphan Drugs, 1, 1, 10.1517/21678707.2013.752128 Kakkar, 2014, The evolving drug development landscape: from blockbusters to niche busters in the orphan drug space, Drug Dev Res, 75, 231, 10.1002/ddr.21176 Heemstra, 2009, Translation of rare disease research into orphan drug development: disease matters, Drug Discov Today, 14, 1166, 10.1016/j.drudis.2009.09.008 Boycott, 2019, International collaborative actions and transparency to understand, diagnose, and develop therapies for rare diseases, EMBO Mol Med, 11, e10486, 10.15252/emmm.201910486 Miller, 2018, Investigating the landscape of US orphan product approvals, Orphanet J Rare Dis, 13, 183, 10.1186/s13023-018-0930-3 Jayasundara, 2019, Estimating the clinical cost of drug development for orphan versus non-orphan drugs, Orphanet J Rare Dis, 14, 12, 10.1186/s13023-018-0990-4 Meekings, 2012, Orphan drug development: an economically viable strategy for biopharma R&D, Drug Discov Today, 17, 660, 10.1016/j.drudis.2012.02.005 Lanthier, 2019, An Analysis of Follow-On Development in New Drug Classes, January 1986-June 2018, Clin Pharmacol Ther, 106, 1125, 10.1002/cpt.1554 Pearson, 2022, The next generation of rare disease drug policy: ensuring both innovation and affordability, J Comp Eff Res, 11, 999, 10.2217/cer-2022-0120 Mullard, 2021, FDA approves 100th monoclonal antibody product, Nat Rev Drug Discov, 20, 491, 10.1038/d41573-021-00079-7 Kulkarni, 2021, The current landscape of nucleic acid therapeutics, Nat Nanotechnol, 16, 630, 10.1038/s41565-021-00898-0 Yamada, 2019, Recent Chemical Approaches for Site-Specific Conjugation of Native Antibodies: Technologies toward Next-Generation Antibody-Drug Conjugates, Chembiochem, 20, 2729, 10.1002/cbic.201900178 Sedykh, 2018, Bispecific antibodies: design, therapy, perspectives, Drug Des Devel Ther, 12, 195, 10.2147/DDDT.S151282 Hair, 2013, Mipomersen sodium: first global approval, Drugs, 73, 487, 10.1007/s40265-013-0042-2 Hoy, 2018, Patisiran: First Global Approval, Drugs, 78, 1625, 10.1007/s40265-018-0983-6 Anguela, 2019, Entering the Modern Era of Gene Therapy, Annu Rev Med, 70, 273, 10.1146/annurev-med-012017-043332 Jogalekar, 2022, Front Immunol, 13, 10.3389/fimmu.2022.925985 Lazo, 2017, New Approaches to Difficult Drug Targets: The Phosphatase Story, SLAS Discov, 22, 1071, 10.1177/2472555217721142 Yu, 2020, RNA Drugs and RNA Targets for Small Molecules: Principles, Progress, and Challenges, Pharmacol Rev, 72, 862, 10.1124/pr.120.019554 Caetano, 2021, Dynamics of patents, orphan drug designation, licensing, and revenues from drugs for rare diseases: The market expansion of eculizumab, PLoS One, 16, e0247853, 10.1371/journal.pone.0247853 Seki, 2022, Lifecycle management of orphan drugs approved in Japan, Orphanet J Rare Dis, 17, 299, 10.1186/s13023-022-02456-w Simoens, 2011, Pricing and reimbursement of orphan drugs: the need for more transparency, Orphanet J Rare Dis, 6, 42, 10.1186/1750-1172-6-42 Villa, 2022, Orphan drug prices and epidemiology of rare diseases: A cross-sectional study in Italy in the years 2014–2019, Front Med, 17 Bourdoncle, 2019, Patient access to orphan drugs in France, Orphanet J Rare Dis, 14, 47, 10.1186/s13023-019-1026-4 Eichler, 2023, Orphan drugs' clinical uncertainty and prices: Addressing allocative and technical inefficiencies in orphan drug reimbursement, Front Pharmacol, 14, 1074512, 10.3389/fphar.2023.1074512 Gertz, 2019, Advances in the treatment of hereditary transthyretin amyloidosis: A review, Brain Behav, 9, e01371, 10.1002/brb3.1371 Lamb, 2021, Tafamidis: A Review in Transthyretin Amyloid Cardiomyopathy, Am J Cardiovasc Drugs, 21, 113, 10.1007/s40256-020-00461-7 Porcari, 2023, Prevalence, characteristics and outcomes of older patients with hereditary versus wild-type transthyretin amyloid cardiomyopathy, Eur J Heart Fail, 10.1002/ejhf.2776 Singh, 2020, The First Orally Deliverable Small Molecule for the Treatment of Spinal Muscular Atrophy, Neurosci Insights, 15, 10.1177/2633105520973985 Finkel, 2017, Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy, N Eng J Med, 377, 1723, 10.1056/NEJMoa1702752 Syed, 2018, Lanadelumab: First Global Approval, Drugs, 78, 1633, 10.1007/s40265-018-0987-2 Gao, 2022, A review of oral kallikrein inhibitor berotralstat for hereditary angioedema, Drugs Today, 58, 59, 10.1358/dot.2022.58.2.3369191 Gower, 2021, Considerations for transition from subcutaneous to oral prophylaxis in the treatment of hereditary angioedema, Allergy Asthma Clin Immunol, 17, 100, 10.1186/s13223-021-00603-9 Zorzi, 2017, Cyclic peptide therapeutics: past, present and future, Curr Opin Chem Biol, 38, 24, 10.1016/j.cbpa.2017.02.006 Moon, 2018, Chemically Induced Cellular Proteolysis: An Emerging Therapeutic Strategy for Undruggable Targets, Mol Cells, 41, 933 Ku, 2015, Recent trends in specialty pharma business model, J Food Drug Anal, 23, 595, 10.1016/j.jfda.2015.04.008 Miller, 2016, Trends In Orphan New Molecular Entities, 1983–2014: Half Were First In Class, And Rare Cancers Were The Most Frequent Target, Health Aff, 35, 464, 10.1377/hlthaff.2015.0921 Brabers, 2011, Orphanet J Rare Dis, 6, 59, 10.1186/1750-1172-6-59 Cossins, 2015, Small Molecule Targeting of Protein-Protein Interactions through Allosteric Modulation of Dynamics, Molecules, 20, 16435, 10.3390/molecules200916435 Ojala, 2021, In Search of a Cure: The Development of Therapeutics to Alter the Progression of Spinal Muscular Atrophy, Brain Sci, 11, 194, 10.3390/brainsci11020194