Activartis received a boost this week when the US Food and Drug Administration (FDA) granted orphan drug status to its investigational cancer immunotherapy AV0113.
The Austria-based biotech said the US regulator gave the designation for AV0113's use in the treatment malignant glioma, a rare and very aggressive type of brain cancer.
This adds to the existing orphan drug designation for AV0113 granted by the European Medicines Agency (EMA).
The two regulatory bodies reserve orphan status for drugs to treat diseases that affect only a limited proportion of the population.
This designation is intended to support research efforts in these less lucrative markets, and provide companies with motivations, such as tax credits and marketing incentives.
These incentives appear to be working, with the EMA reporting last year that orphan drug applications were up 30 per cent for the first six months of 2012.
A Thomson Reuters' report also described orphan drugs as an economically viable market for pharma.
Activartis' AV0113 is part of this growing trend, and the drug has shown promise in early-stage trials.
The company has now completed recruitment of 78 brain cancer patients to a multi-centre, randomised, phase II clinical trial, which aims to deliver safety and efficacy data for the first time.
According to Activartis, preliminary results revealed a “very promising trend” towards overall survival benefit in patients taking AV0113.
The drug acts as an immunotherapy, activating the patient's immune system to fight tumour cells.
It does this by convincing the body's dendritic cells that there are dangerous molecules, causing these cells to go into a pro-inflammatory mode of action.
This activates tumour-specific T-cells, which are able to recognise and destroy tumour cells.
Other companies banking on immunotherapies in the fight against cancer include Merck Serono, which last month set up a dedicated R&D unit for such therapies.
Cancer immunotherapies already on the market include Bristol-Myers Squibb's Yervoy (ipilimumab) for melanoma, which posted sales of $706m in 2012, its first full-year on the market after debuting in the US in March 2011.