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Survey about achondroplasia
We have designed a questionnaire with the purpose to better know the population connected to achondroplasia.
The questionnaire is optional and anonymous. The data obtained will be used for statistical purposes and to better understand the natural history of achondroplasia. Please consider answering.
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A new approach by the company Ascendis Pharma, through their innovative TransCon technology with the TransCon CNP for achondroplasia.
What is TransCon CNP and how does it work?

This technology combines the benefits of a prodrug and a sustained-release technology. A prodrug is a masked form of an active drug (in this case, CNP linked to another molecule, the TransCon linker) which is converted into the active drug when it reaches its therapeutic target in a predictable pattern (sustained release) due to the nature of the linker used to form the prodrug [1].

prodrug 1
How a prodrug acts in the body. Credits: Cutting Edge Approach on Prodrug: Contrivance for Target Drug Delivery. Varsha Y et al., 2011

The therapeutic molecule is a CNP, same basic compound as Vosoritide, so its mechanism of action is similar, except on the delivery, requiring fewer doses and less quantity of CNP to elicit the therapeutic effects (more on how CNP works here). CNP is, in theory, more efficient with this technology because it's degraded more slowly since it only assumes a form that our cells can degrade closer to its target (it would otherwise be degraded as soon as it entered the body), which also reduces toxicity.
In what stage of development is TransCon CNP in?

Ascendis Pharma plans to submit an Investigational New Drug Application for TransCon CNP in the fourth quarter of 2017 and TransCon CNP will most probably enter in phase 1 clinical trial in the second quarter of 2018.


  1. Ascendis Pharma (2017). "TransconCNP." Product Pipeline. Retrieved 25/05, 2017.
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