What happens if a newly developed drug fails in the tested indication? 

Very often such drugs are abandoned if the developers think they will not be able to be used for different indications or therapeutic areas. In such cases these drugs are classified as ‘orphaned drugs’. 

Where the term ‘orphaned drug’ comes from? 

The focus of drug development is shifting towards diseases that affect smaller amount of the population, also known as rare or ‘orphan’ diseases. In USA a disease is considered ‘orphan’ if affects less than 200 000 people or roughly 1 per 1500 people. The term ‘orphan drug’ refers to drugs used to treat orphan diseases and its derived from legislation like Orphan Drug Act of 1983. 

Not surprisingly oncology is viewed as one of the major therapeutic area where orphan drugs are used because more and more evidence suggest that cancer is a collection of orphan diseases. 

Vicus Therapeutics has developed a model which allows adoption of such orphan drugs for new cancer indications.

Step 1:  Hierarchical Network Algorithm (HiNET) – This is an algorithm that allows modelling of the disease by evaluating tissue energetics, homeostatic control and biochemical pathways. 

Step 2: Drug Selection: In this step it is used a data base which contains information for off-patent drugs, their target and human efficacy data in similar diseases, potential adverse events and pharmacokinetic profiles.

Step 3: Due to the complexity of cancer rarely one single drug could be used, therefore the model created potential treatment regimens.  Then the suggested regimens are evaluated for their potential safety and efficacy. 

The use of such models in repurposing the orphan drugs is a novel and smart way of speeding up drug development process and identifying new therapies for rare diseases which in many cases have no treatment options.


Adopting orphaned drugs: developing multidrug regimens using generic drugs

Author: Olga Peycheva

Olga is a clinical research professional who has been working in clinical research since 2005. She has extensive experience in clinical research in Eastern and Western Europe. 

Originally published on 4 July 2019