Chelsea Therapeutics Provides an Update on Northera(TM) (droxidopa) Regulatory Status and Development Program

Chelsea Therapeutics International, Ltd.

received a written response from the U.S. Food and Drug Administration (FDA) to Chelsea's modified proposal for utilizing data from ongoing Study 306B to support its application for marketing approval of Northera™ (droxidopa) in the U.S. In its response, FDA advised Chelsea that, based on the theoretical potential for certain patients from Study 306B to have been unblinded in conjunction with the reporting of 306A data, the FDA cannot be confident that this information did not influence an amendment of the statistical analytic plan, and therefore believes, as presently planned, Study 306B is unlikely to provide sufficient confirmatory evidence to support a Northera™ (droxidopa) Capsules New Drug Application (NDA). The Advice Letter from the FDA noted "if an analysis of all subjects enrolled in study 306 after [Chelsea] amended the analytic plan demonstrated a statistically significant benefit on the primary endpoint, [FDA] might regard this as a positive trial." However, as this analytic approach appears to exclude at least 109 patients from Study 306B, the FDA further recommends that Chelsea 'design and conduct an additional trial to demonstrate that droxidopa has a significant and persistent effect' on symptoms of neurogenic orthostatic hypotension. The FDA did not provide feedback or express any concerns regarding Chelsea's proposal to assess efficacy two-weeks post titration using OHSA item 1 (dizziness) nor did the FDA provide further guidance regarding the duration of clinical efficacy data needed to support a "persistent effect." Based on FDA's feedback, along with potential cost and timing considerations, the Company is currently evaluating several scenarios that may provide the supportive data the FDA is seeking while minimizing any delays to the planned resubmission of its Northera NDA. "As we communicated to the FDA during our End-of-Review meeting and subsequently documented in detail as part of our proposal, we remain fully blinded to all efficacy data from Study 306B," commented Dr. Simon Pedder, president and CEO of Chelsea Therapeutics. "We are deeply disappointed that the agency did not find our blinding documentation sufficient to warrant full use of Study 306B as confirmatory evidence of droxidopa's demonstrated benefits. The FDA did acknowledge the 'important need' for a safe and effective therapy for these patients and indicated they were 'anxious to work with [us] in designing a new trial,' specifically noting that many suitable trial options exist, including potentially recruiting patients directly from Chelsea's expanded access program."