Glaxo and Prosensa have not disclosed the breakdown by age of the 186 patients enrolled in the phase III study, so we won't know if any of this impacts the final results, if at all, until data are presented.
One more item of note from my Prosensa meeting: The company (and Glaxo) have a "contingency plan" if the phase III study fails, Schikan said. Data from three studies -- DEMAND-2, the phase III and DEMAND-5 will be aggregated and analyzed with the goal of demonstrating an overall benefit or a benefit for certain relevant subsets of DMD patients i.e. boys 7 and older. This aggregated data analysis, if positive, would be submitted to the FDA for review and potential approval, Schikan said.
The contingency plan is not unreasonable and might succeed, depending (obviously) on what the efficacy and safety data look like. To be fair, Sarepta Therapeutics (SRPT - Get Report) is counting on FDA accommodation and leniency in order to get eteplirsen approved on a small amount of phase II data. It's not crazy to assume FDA might also be cooperative with Glaxo and Prosensa.
I probably speak for a lot of investors when I say I just want to see the new drisapersen data already so that we can finally get some answers to pressing questions about Prosensa and Sarepta.Kevin L. writes: How do you feel about Rockwell Medical (RMTI - Get Report) now that the CRUISE-1 and CRUISE-2 studies have come back positive? I like the Rockwell Medical story. Administering small doses of maintenance iron to patients via the dialysate used during the hemodialysis procedure makes a lot of sense, more so than waiting for a patient's iron stores to be depleted and replacing it all at once with a large IV iron infusion. Rockwell believes SFP, if approved, will be a superior and safer method for maintaining hemoglobin levels in kidney dialysis patients. By switching to SFP, dialysis centers will reduce costs because use of IV iron and ESAs (Amgen's Epogen) will decrease. I want to hear more of the Rockwell/SFP bear story -- I'm sure it exists -- but until then, the company's pitch for the potential of SFP is really compelling.
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