Therachon’s medicine in development for achondroplasia, TA-46, was granted Orphan Drug designation by the medicine agencies FDA and EMA.
TA-46 is a type 3 fibroblast growth factor receptor antagonist addressing achondroplasia. This medicine is an inactive form of the receptor that binds to available fibroblast growth factors (FGFs) and with this stops the abnormal receptors from working. Through its action as a decoy, the medicine is expected to reduce the activity of these receptors, thereby helping to restore normal patterns of growth (EMA, 2017).
To understand the evolution of the process, these are the latest known timelines:
- 27 Feb 2017 – TA 46 received Orphan Drug status for Achondroplasia by the European Medicines Agency, EMA
- 2 Jun 2017 TA 46 received Orphan Drug status for Achondroplasia by the US Food and Drug administration, FDA
- 15 Jun 2017 Therachon signed an agreement with Catalent Pharma Solutions, to support the preclinical and clinical development of TA-46, heading to plan of clinical trials in paediatric patients with Achondroplasia
- 20 Jun 2017 Press release of Therachon receiving orphan drug designation for TA-46
About Orphan Designation
FDA: The Orphan Drug Act (ODA) provides for granting special status to a drug or biological product (“drug”) to treat a rare disease or condition upon request of a sponsor.
EMA: The European Medicines Agency is responsible for reviewing applications from companies /pharmaceutical industry, who intend to develop medicines for rare diseases, known as ‘orphan drugs'(EMA, 2017). When an Orphan Drug designation is granted, EMA provides incentives for the drug’s development as:
- Market exclusivity: 10 years with no competition by similar products,after the drug is approved for sale
- Protocol assistance: The agency provides scientific advice to optimize development and guidance on preparing a dossier that will meet European/US regulatory requirements
- Fee reductions
- EU-funded research
Designation as an orphan medicinal product does not indicate that the product has already satisfied the efficacy, safety and quality criteria necessary for the granting of a marketing authorization. As with any medicine, these criteria can only be assessed once the application for marketing authorization has been submitted.
Next steps – clinical trial Phase 1
At the time of submission of the application for orphan designation in EMA, the evaluation of the effects of the
medicine in experimental models was ongoing (EMA, 2017).
Phase I trial (short video)
Is the first in a series of four stages in testing new therapies in humans. The primary goal of these studies is to determine whether the therapy can be given safely, so one of the main evaluations is to watch for harmful side effects that may be caused by the treatment. The doctors who lead the clinical trial also will try to determine the best way to give an experimental drug (e.g., by mouth, IV drip, or injection) and how often and how much should be given, which is called dosing (John Hopkins Medicine, 2017).
Phase I clinical study for TA-46 is scheduled for the beginning of 2018
In further phases of the clinical trial, TA-46 will be evaluated in children that will receive 1 single subcutaneous (under the skin) injection per week to reduce the effects of achondroplasia.