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Drug Repurposing and Rare Diseases

Treatments for rare and genetic conditions are often few and far between, so it is important for us to be examining all the different ways that therapies might become available to our community in the future. To that end, Richard Le has written a blog about repurposing medicines for rare diseases. Richard Le is a Junior Scientist at SOM Biotech, a clinical-stage biopharmaceutical company specialising in the repurposing of drugs for new indications. Richard is part of the R&D unit, responsible for identifying potential therapeutic candidates for specific rare diseases.

Drug repurposing is the process by which a treatment already used for a certain condition is used to treat other diseases, expanding the range of use of the medicine.

Drug development incurs a high level of risk and is prone to failure, the process normally spans between 12-16 years. Drug repurposing can have many advantages, including bringing this time scale down by roughly 8 years. This is because the repurposing of a previously licensed drug, for a different indication, reduces the time and money spent in research, development (R&D) and clinical trials. Phase I clinical trials can often be bypassed as a result of previously-established drug safety profiles for their original indications. Although still prone to risks, these are significantly lower in comparison to the traditional drug development processes.

Examples of successful repurposing include thalidomide, formerly used for morning sickness, now used to target multiple myelomas (a group of rare cancers). And perhaps one of the most successful examples in medical history: sildenafil (better known as Viagra), which was originally destined for angina but is now utilised for erectile dysfunction and pulmonary arterial hypertension (a rare disease). Various groups are exploring drug repositioning opportunities throughout the scientific community, including in the pharmaceutical/biotechnology industry.

Within the last 20 years, R&D investment by the pharmaceutical industry has increased while the number of therapeutics approved has gone down. This is an obvious problem for patient communities with little or no treatments, such as the rare disease community. In order to be more efficient and to maximise investment, big pharma has turned towards repurposing – many companies have now created specialised research units or departments, and numerous large and medium size pharmaceutical and biopharmaceutical companies are using this developmental approach. Small companies specialising in drug repurposing have recently appeared, such as: Biovista, Anaxomics Biotech SL, Horizon Discovery and SOM Biotech – whom I represent.

Recently SOM Biotech successfully completed a Phase IIa clinical study for a repurposed therapeutic product for transthyretin amyloidosis. At SOM Biotech, we are now in the midst of launching a crowdfunding campaign to start new R&D projects in pediatric rare diseases. Numerous pharmaceutical companies, large and small, have invested heavily within the sector.

Despite not being a replacement for traditional therapeutics development, drug repurposing certainly holds promise for the rare disease community.

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