American biotech BioCryst Pharmaceuticals (BCRX 2.65%) scored an important regulatory win across the Atlantic Ocean on Wednesday. As a result, happy shareholders pushed the company's stock almost 5% higher on the day.
BioCryst announced that the European Medicines Agency (EMA) -- a regulator for the sprawling, multi-country European Union -- has granted the company's BCX9250 investigational drug access to the EMA's Priority Medicines (PRIME) initiative. This is a treatment targeting fibrodysplasia ossificans progressiva (FOP), an affliction in which patients suffer the ossification of ligaments and tendons.
The EMA describes PRIME as an effort "to enhance support for the development of medicines that target an unmet medical need." It is similar to efforts in other jurisdictions aimed at encouraging the development and commercialization of such treatments. FOP certainly fits the bill, as it is a very rare disease throughout the world.
BioCryst didn't hesitate to point out that BCX9250 is the first investigational drug for its indication to enter the PRIME program.
The biotech quoted its chief research and development officer Helen Thackray as saying that "Promising results from non-clinical data and the first-in-human Phase 1 safety, tolerability and pharmacokinetics study in healthy subjects formed the basis of the application for PRIME eligibility."
"We look forward to applying the benefits available to us through PRIME as we continue to advance our ALK-2 inhibitor program," she added.
BCX9250 certainly has potential, but investors should keep in mind that it is early days for the pipeline drug. According to BioCryst, in a phase 1 clinical trial it was demonstrated to be safe and well-tolerated by the participants. Significantly more research is forthcoming.