William SumnerGW Pharmaceuticals plc (GWPH), a biopharmaceutical company dedicated to developing cannabis-based pharmaceuticals, announced today cannabidiol (CBD) has been granted orphan drug status by the European Medicines Agency (EMA) for the treatment of tuberous sclerosis (TS).
Orphan drug designation by the EMA is meant for medicines used to treat rare diseases, as defined by no more than 5 patients in 10,000. The designation allows companies to profit from incentives offered by the European Union to develop medicines for the treatment of life threatening or debilitating rare diseases; including reduced fees and protection from competition once on the market.
Currently the company is recruiting for a Phase 3 clinical trial of Epidiolex, a cannabidiol-based medicine, as an ancillary treatment to TS. The company expects to begin receiving data by the second half of 2018. Pending positive results, the company expects to submit regulatory applications for Epidiolex in the treatment of TS in both the United States and European Union.
“GW‘s decision to evaluate Epidiolex in patients with tuberous sclerosis is based on findings from the physician-led Epidiolex expanded access program where the results of this open-label use of Epidiolex in children with TS have been very encouraging,” said GW CEO, Justin Gover, in a statement.
This latest comes on the heels of news that the company’s pipeline compound GWP42006 failed its Phase 2a proof of concept study. The drug was meant as an ancillary treatment for adults with inadequately controlled focal seizures but recent studies showed a much higher response from patients taking placebos than expected.
In related news, GW competitor Insys Therapeutics (INSY) suffered a setback after news broke that Gavin Awerbuch, a Michigan doctor tied to a federal investigation into the company, was sentenced to 32 months in prison after admitting to writing illegitimate prescriptions for Insys’ fentanyl-based drug Subsys and health care fraud.
According to Reuters, the company used a fraudulent speaker program to pay the doctor $138,435 in kickbacks. In addition to severing 32 months in prison, Awerbuch must oay $4.1 million in restitution and fines.
