New Muscular Dystrophy Drug Granted FDA Orphan Designation in US
What's next?
The company Benitec is working hard to continue with the development of the drug by running nonclinical safety studies as well developing production processes. The hope is to continue to expand the approvals worldwide by working with other governing bodies such as, for example, Health Canada to eventually help as many people with OPMD as possible.
From a business perspective, companies such as Benitec do not begin developing orphan drugs to make money, in fact, the economical return is very small, at first. As the drug or treatment options works through the development plan, the goal (aside from developing something to help those suffering from diseases like OPMD) is to develop something that will eventually have a bigger impact. In the case of BB-301, the ‘silence and replace’ technology could potentially work with other forms of MD and other diseases as a whole.
