European Commission Grants Orphan Designation Designation for Investigative Achondroplasia Therapy

Ascendis Pharma has just announced that their investigative therapy for achondroplasia called TransCon CNP has just received Orphan Drug Designation from the European Commission. This same designation has already been provided by the FDA for this medication (Feb 2019).

As of yet, there are still no approved treatments for this condition. Researchers are hopeful this therapy could become the first option for patients.

Achondroplasia

Achondroplasia is a form of dwarfism. It is caused by a mutation in the FGFR3 gene. This mutation leads to imbalances in the FGFR3 signaling pathway and the CNP signaling pathway (which is responsible for stimulating growth).

An increase in CNP promotes bone growth by counteracting the impacts of the mutation.

The condition is associated with an array of skeletal complications that each are associated with their own comorbidities. Some of these are explained below.

  • Abnormal development of the skeletal system -> sleep apnea
  • Lower spine impingement -> chronic pain in the legs and back
  • Cervical compression -> sudden death in infants
  • Abnormal Eustachian tubes -> chronic ear infections -> hearing loss as well as speech delay

TransCon CNP

TransCon CNP is a long-acting prodrug that is meant to ensure patients have a continuous level of CNP. This is achieved through one subcutaneous dose each week.

TransCon technology stands for transient conjugation. It’s goal is to optimize the efficacy and therapeutic effect of new therapies.

The TransCon molecules have an unmodified drug, a carrier that protects that drug, and a linker that binds the drug and carrier. The drug is not activated until it is released from its carrier. The injection of both drug and carrier into the body initiates the drugs release, due to the physiologic conditions of the body. This release is predictable, and allows the unmodified drug to carry on as designed.

This type of technology can be applied to small molecules, peptides, or proteins. It can be used in many therapeutic areas, either locally or systemically.   

Looking Forward

Ascendis headquarters are in Denmark but they also operate offices in Germany and California. The company is in the process of working on three different therapeutic candidates, all for rare diseases.  

The TransCon CNP product is currently in a Phase 2 clinical trial. This global investigation is called ACcomplisH. This new designation will help the company to stay on track with their development, and hopefully get this new therapy into the hands of patients faster. 

You can read more about this investigative therapy for achondroplasia here.

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