Treating achondroplasia: update on the current potential therapies

There has been a while since I published the last article and, as we see some new updates on currently therapeutic approaches and the emrgence of a couple of new ones, I thought it would be good to list the current active (and not so active) research on potential therapies for achondroplasia.

The table below lists the main therapeutic strategies disclosed in the literature. Almost all of them have been reviewed here in the blog, but for two exceptions: 1. B-701, an anti-FGFR3 antibody currently in development by an US based biotech company, about which we do not have much information; and 2. the gene editing strategy (which does deserve a dedicated review). If you are interested in learning more about them, just visit the index page to find out the links for their reviews.

Some of the drugs listed have not been tested in an achondroplasia model, but showed activity against FGFR3 in the lab and could be candidates for testing in achondroplasia as well.

Table. Main potential pharmacological strategies for the treatment of achondroplasia.

Drug	Status	Developer	Reference
Tested in achondroplasia models
Vosoritide	Phase 3	Biomarin	Lorget F et al. (2012)
TA-46	Pre-clinical	Theracon	Garcia S et al. (2012)
Meclizine/meclozine	Pre-clinical	Academy	Matsushita M et al. (2017)
Statins	Pre-clinical	Academy	Yamashita A et al. (2014)
NVP-BGJ398	Pre-clinical	Academy/Novartis	Komla-Ebri D et al. (2016)
B-701 (antibody)	Pre-clinical	Bioclin	No specific publication located
CRISPR-CAS9 (gene editing)	?	Academy	Wojtal D et al. (2016)
PTH	?	Academy	Xie Y et al. (2012); Kaisermann MC (2011, letter on Samson et al. 2011)
P3 (peptide)	?	Academy	Jin M et al. (2012)
Tested against FGFR3 (but not in achondroplasia)
Menaquinones		Academy	Sogabe N et al. (2011); Kaisermann MC (2011) (letter on Sogabe et al.); Cao K et al. (2009)