I have no employment relationship or receive any compensation or incentive from any company researching medications or treatments for achondroplasia. What I write is based on the knowledge acquired from the scientific literature and the information published by the respective companies.
I have been receiving many questions about how the research for achondroplasia treatments is going and more specifically about vosoritide. As the treatment is a relevant theme to many, I thought it would be useful to share my knowledge with everyone in the form of questions and answers.
All product information in this text is in public domain and referenced. Where there is no bibliographical reference the text reflects my personal opinion.
Visit the index page to learn more about the topics covered here. The blog contains many reviews on achondroplasia and on basically all potential treatments already published in the scientific literature so far.
- What is vosoritide?
- What is the reason for using CNP or vosoritide in achondroplasia?
- What is the risk of using CNP or vosoritide in children with achondroplasia?
- What is the risk of using vosoritide in the long term?
- What to expect from treatment with vosoritide?
- Does vosoritide really work?
- Who will benefit from the use of vosoritide (or any treatment for achondroplasia)?
Growth plates close at the end of puberty, representing the end of the growth period of the body. Unfortunately, CNP, vosoritide or any other treatment aimed at blocking the action of FGFR3 will cease to be useful when the individual reaches adulthood because there will be no more growth activity then.
- When to start treatment with vosoritide for the treatment of achondroplasia (or any other medication that is approved to treat it)?
- As for vosoritide, when will it become available?
Vosoritide is being tested in a Phase 3 study, and the results of this study will allow the laboratory developing it to obtain the license to market it provided it is successful in the tests. The current prediction by the developer is that the results of the phase 3 study will be available in the second half of 2019 to be submitted to the FDA (9). Based on this prediction, if approved, it may be available in the United States at the beginning of 2020. Availability in other countries will depend on the strategy of the developer of vosoritide and the speed of the drug approval process of the regulatory agencies in those other countries.
- Are there other medications being tested?
For example, the developer of the molecule known as TA-46 announced in February 2018 that it wants to start the Phase 1 study soon (10). Another company that is developing a new form of CNP known as TransCon-CNP also intends to request authorization to start clinical studies soon (11). As a consequence of a study in an animal model of achondroplasia in which it showed promising results, a company was created to continue the development of the molecule NVP-BGJ398 (12,13).
These are just a few examples, there are other initiatives underway, but I believe these are the most promising at the moment. All these initiatives are still far from the pharmacy, but some of them, if successful, could be available in about 4-5 years .
I hope this little review helps to clarify some doubts. The blog Treating Achondroplasia is active. New articles will be published whenever there is any theme that may interest readers.
1. Wendt DJ et al. Neutral endopeptidase-resistant C-type natriuretic peptide variant represents a new therapeutic approach for treatment of fibroblast growth factor receptor 3-related dwarfism. J Pharmacol Exp Ther 2015; 353(1):132-49. Free access.
2. Krejci P et al. Interaction of fibroblast growth factor and C-natriuretic peptide signaling in regulation of chondrocyte proliferation and extracellular matrix homeostasis. J Cell Sci 2006; 118: 5089-00.
3. Lorget F et al. Evaluation of the therapeutic potential of a CNP analog in a Fgfr3 mouse model recapitulating achondroplasia. Am J Hum Genet 2012;91(6):1108-14.
4. Nakao K et al. Impact of local CNP/GC-B system in growth plates on endochondral bone growth. Pharmacol Toxicol 2013; 14 (Suppl 1):48.
5.Yasoda A et al. Systemic Administration of C-Type Natriuretic Peptide as a Novel Therapeutic Strategy for Skeletal Dysplasias. Endocrinology 2009;150: 3138–44.
6.Yasoda A and Nakao K. Translational research of C-type natriuretic peptide (CNP) into skeletal dysplasias. Endocrine J 2010; 57 (8): 659- 66.
7. Hoover-Fong J et al. Vosoritide in children with achondroplasia: Updated results from an ongoing Phase 2, open-label, sequential cohort, dose-escalation study. Abstract presented at the American Society of Human Genetics Meeting 2016, Vancouver. Meeting Abstract book p. 1301. Free access.
8. Biomarin R&D Day 2017 presentation.
9. Biomarin press release. BioMarin Announces Fourth Quarter and Full Year 2017 Financial Results.
10. Therachon Feb 14, 2018.Therachon Announces Dosing of First Subject in Phase 1 Clinical Trial Evaluating TA-46, a Novel Investigational Therapy for the Potential Treatment of Achondroplasia.
11. Ascendis Pharma. TransCon-CNP.
12. Komla-Ebri Det al. Tyrosine kinase inhibitor NVP-BGJ398 functionally improves FGFR3-related dwarfism in mouse model. J Clin Invest 2016;126(5):1871-84. Free access.
13. FiercePharma Jan 30, 2018. BridgeBio’s QED Therapeutics picks up discarded Novartis cancer drug.