Antifungal Drug Candidate Given US Orphan Drug Status

Submitted by GAtherton on 13 January 2016

New antifungal drugs are needed, preferably those which may provide a new alternative to treat Aspergillus infections already resistant to existing drugs. There are many possible new drugs under development but we know that not all of those will reach the point were they will be available in clinic. Some will be too toxic when given to animals or people in clinical trials, some will not be effective when given to try to control an infection and more. Those that make it as far as a clinical trial still have quite a low chance of success (around 21% make it out of phase 1 and progress to the clinic) but they have already come a long way – they have to have a reasonable chance of success as clinical trials get VERY expensive and represent a big gamble with investors money for the pharmaceutical companies involved.

Drug development is a long process (drug development review) and even if successful a new drug has a limited amount of time to earn money. Considering costs are estimated at $900 million for a typical drug there are clearly huge amounts to make before profits can be considered! When a drug has a limited market (and thus limited scope for paying back the investment in its development) it can be given orphan status by the regulatory bodies of the countries in which it will be marketed, This extends the time that the drug has exclusive access to its market and thus increases the incentive to investors to develop that drug.

This month antifungal candidate drug VL-2397 (also known as ASP2397) has been granted orphan drug status in the US and will now go into phase 1 clinical trials. In testing up until now VL-2397 has demonstrated good activity against strains of Aspergillus that are already resistant to antifungals already in use in the clinic. We can only hope it will be as successful in clinic, and that it gets that far!