Shining a Light on Drug Repurposing for Rare Diseases

Whole genome sequencing is providing high-level information about new rare diseases putting pressure on drug developers to pick up the pace. Of approximately 7,000 rare diseases, most do not have a cure. Innovations in drug repurposing are necessary to bridge the gap between promises and cures.

Labiotech recently published an article highlighting the advantages of drug repurposing for the treatment of rare diseases.  One of the most obvious problems facing developers concerns clinical trials and the difficulty in enrolling a sufficient number of rare disease patients.

The FDA offers incentives and expedites regulations. The agency points out that drugs undergoing expedited approval are still beneficial and safe for patients. Beyond these incentives, drug development must be more creative in order to increase production.

About Drug Repurposing

The procedure, due to its many benefits, has captured the attention of mainstream drug developers. Biotech companies and researchers are seeking new ways to use applications for drugs that already exist. This includes drugs that were investigated in clinical trials but were not entered into the commercial market.

When compared to normal drug processing, the repurposing method is more economical as it reduces costs and time. The next hurdle is whether it can treat the targeted condition.

Aspirin is an excellent example. Aspirin was originally developed for pain relief. Later the medicine was repurposed to treat heart diseases. More recent examples are the various drugs repurposed by drug companies throughout the world in the race to treat COVID-19.

 Drug-Drug Interaction

A simple explanation would be the drug-drug interaction that occurs when the effect of one drug is changed by the introduction of another drug.

The use of high-throughput screening (HTS) is defined as automated equipment that tests thousands to millions of samples for biological activity.

NovaBiotics, a Scottish biotech company that is primarily focused on immunology assays is repurposing a drug used to treat cystinosis, a rare metabolic disorder. The drug, cysteamine, has been used for this purpose for decades.

NovaBiotics has reformulated cysteamine, now called Lynovex, and is developing it to potentially cure cystic fibrosis, also a rare metabolic disease.

Lynovex has been granted orphan designation in Europe and in the United States. The designation represents a status that is assigned to a drug developed for the treatment of a rare disease.

NovaBiotics has also developed a product called NM001 for cystic fibrosis in dry power (inhaled) and oral form. Initial results found that the oral form is helpful during the episodes where symptoms of cystic fibrosis worsen. The form that can be inhaled supports lung function during treatment.

In addition, cysteamine is being repurposed by NovaBiotics to treat the more common community-acquired pneumonia. Companies are exposed to less risk by repurposing the same drug for multiple conditions.

There is an advantage to repurposing drugs that have similar characteristics as drugs treating the same disease. However, data used in the repurposing process are not always accessible for rare diseases when attempting to search the interactions of drugs and targets.


Computational Drug Repurposing (CDR) is revolutionizing drug discovery by offering faster and more expandable techniques.

Computer Aided Drug Discovery (CADD) reduces cost and time during the initial discovery stage. CADD highlights molecules with the best chance of performing against the drug target with minimal adverse events.

AI-based solutions allow researchers to have a better understanding of the mechanisms of disease and then identify the drugs that may work against them.

New methods for repurposing drugs for individual patients are urgently needed as are improved computational tools that can collect insights from rare disease patients.



Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia (AML) six years ago. During this period of partial remission, Rose researched investigational drugs to be prepared in the event of a relapse. Her husband died February 12, 2021 with a rare and unexplained occurrence of liver cancer possibly unrelated to AML.

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