Once again a group of outside FDA experts has shrugged off the agency's concerns about Chelsea Therapeutics' drug Northera (droxidopa) and voted to recommend an approval. And once again investors were thrilled to see the endorsement, sending shares of the biotech up about 150% on the lopsided 16-to-1 win.
Now it's up to the FDA to decide whether it will once again send Chelsea ($CHTP) back to the drawing board for more and better data on the drug, which is designed to prevent dizziness and fainting among patients with neurodegenerative ailments like Parkinson's, or finally give it a green light on marketing.
The experts, though, clearly believe that Northera's time has arrived, satisfied that investigators had nailed down data demonstrating a short-term benefit for patients--a considerable improvement for a population without much to choose from.
"I tend to lower the standard a little bit if there's nothing else that really works," said panel member Michael Proschan, a statistician from the National Institute of Allergy and Infectious Diseases, according to Bloomberg.
Chelsea has been on a roller coaster ride with Northera. The drug was initially rejected following a panel vote in its favor, then investors rallied on the news that the FDA had reversed its position and would consider a new application with data from an ongoing study. But FDA insider Shari Targum spurned the new application, saying that the biotech had failed to deliver the goods.
"If we give the applicant the benefit of the doubt, and consider study 306B to support efficacy, the results do not meet the criteria as a "robust" or "strongly positive" single study to support a symptom benefit," Targum noted. "This conclusion is based on the small treatment effect, exceeded by the three-fold higher intra-subject variability."
FDA panels, though, often are open to the argument that patients need some help, even if it might not amount to much help. And a number of the experts reportedly concluded that Chelsea could hunt down long-term effects in a study after marketing began.
Now the ball is back in the hands of the FDA for a formal decision, but at least for now, the roller coaster is headed back up.
- here's the story from Bloomberg