Drug Repositioning Program

Identifying and developing new uses for exisiting drugs in neurology


Drug repositioning, or the discovery of new indications of already known drugs has played an increasingly important role in the search for new treatments for incurable and rare diseases in recent years, due to the more limited capital investment requirements involved. This reduces the time and money that pharmaceutical companies have to invest, and means that treatments can be offered more quickly and with reduced risks to the individuals affected. Bearing in mind that the full process between the discovery of a new drug and its launch on the market takes 13 years, and an investment of approximately 1,778 million dollars, drug repurposing can substantially reduce both the time and the cost.

Our drug repositioning program is experienced in proof of concept and phase II trials for drugs in rare diseases. Our Department is one of the most prestigious in Spain in terms of the volume and variety of rare diseases treated. We have recently carried out proof of concept trials for drug repositioning for a promising treatment for familial amyloid polyneuropathy (FAP-TTR). Further trials for Huntington’s disease, amyotrophic lateral sclerosis and X-linked adrenoleukodystrophy (ABCD1) are under way. Phase II clinical trials for rare diseases including chronic inflammatory demyelinating polyneuropathy (CIDP), ALS, myasthenia gravis, Nonaka myopathy, familial amyloid polyneuropathy (FAP-TTR) and spinal muscular atrophy are also ongoing.

Our center is an accredited Reference Center for Neuromuscular and Rare Diseases (CSUR) by the Ministry of Health of the Spanish Government, and a member of the European Network for Neuromuscular and Rare Diseases (ERN-NMD). We can offer biopharmaceutical companies a complete portfolio of services for the clinical process of drug repositioning.