(By Balaseshan) Chelsea Therapeutics International Ltd. (NASDAQ:CHTP) said it has received FDA response for the study to treat neurogenic orthostatic hypotension that is unlikely to provide sufficient confirmatory evidence to support New Drug Application (NDA).
The company has got a written response from the U.S. Food and Drug Administration (FDA) to its modified proposal for utilizing data from ongoing Study 306B to support its application for marketing approval of Northera (droxidopa) in the U.S.
Chelsea is seeking approval of Northera for the treatment of symptomatic neurogenic orthostatic hypotension (known as Neurogenic OH) in patients with primary autonomic failure (Parkinson's disease, multiple system atrophy and pure autonomic failure), dopamine beta hydroxylase deficiency and non-diabetic autonomic neuropathy.
[Related -Stocks End Mixed Amid 'Cliff' Talks, Data; Apple (AAPL) Plunges]
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 agency cannot be confident that this information did not influence an amendment of the statistical analytic plan.
And therefore the agency believes, as presently planned, Study 306B is unlikely to provide sufficient confirmatory evidence to support a Northera (droxidopa) Capsules NDA.
The Advice Letter from the FDA noted that 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, the agency might regard this as a positive trial.
[Related -Futures Advance As China, Data Weigh; Pandora Media (P) Slumps]
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.
Droxidopa is a synthetic catecholamine that is directly converted to norepinephrine (NE) via decarboxylation, resulting in increased levels of NE in the nervous system, both centrally and peripherally.
The most common adverse event (greater than or equal to 5%) during placebo controlled trials was headache. Droxidopa is also being investigated for and completed Phase 2 trials in intradialytic hypotension and adult attention deficit hyperactivity disorder and fibromyalgia.
Neurogenic OH is a chronic neurogenic disorder resulting from deficient release of norepinephrine, the neurotransmitter used by sympathetic autonomic nerves to send signals to the blood vessels and the heart to regulate blood pressure. This deficiency results in lightheadedness, dizziness, blurred vision, fatigue, poor concentration and fainting episodes when a person assumes a standing position.
CHTP closed Monday's regular session down 1.35% at $1.46. The stock has been trading between $1.05 and $6.06 for the past 52 weeks.