FDA’s short-term view helps Chelsea rise from the ashes

In a move that caught many investors on the hop the FDA has offered Chelsea Therapeutics a way back from the brink, agreeing to use its recently published Study 306b as the basis for a resubmission of the company's lead drug, Northera. Chelsea's share price more than doubled yesterday.

Having previously received a complete response letter for Northera, this latest decision by the FDA says a lot about the unmet medical need in neurogenic orthostatic hypotension (NOH), the sudden drop in blood pressure on standing up that is most often associated with Parkinson's disease. What Chelsea must do now is quickly capitalise on this unexpected reversal in the regulator's position and hope that what will be a forensic review of the data will not find it wanting.

Get out of jail card

Previously Chelsea had fallen foul the FDA’s concern over Northera's durability of response. The drug has proved itself effective in reducing NOH symptoms of dizziness and light-headedness for a week, but the benefit is not statistically significant in subsequent weeks. It was this that caused the regulator to refuse approval, despite a positive advisory committee decision.

This short treatment horizon could also continue to be a thorn in Chelsea’s side. In its guidance to the company, after a meeting with some of its senior FDA officials, the regulator said there could be “a possible requirement to verify durable clinical benefit post-approval”.

As such Chelsea will not get out of conducting a longer trial of Northera, which will have to address the big problem of placebo effect, something that has devilled it in the past.

But this surprising Hail Mary pass could not have come at a better time for Chelsea, which was hovering on the cusp of being delisted from the Nasdaq for breaching its $1 share price rule. If Northera had been delayed because of  longer trial there were few other candidates in the pipeline to turn to and even fewer funding options to move another candidate through the pipeline.

Moving quickly

A filing is now expected by the end of the second quarter, and with a class two submission a six-month review is possible, meaning Northera could have approval by the end of the year. The post-marketing study is also due to start in the fourth quarter of 2013.

Many are now expecting the drug to get a pass by the FDA, which equals some impressive persuasion on the part of the management team during their formal appeal. But even with approval under its belt it is unclear how the drug will perform.

Its only competition, midodrine, has side effects that are unpopular with patients, which could be an advantage for Northera despite doctors’ comfort in prescribing midodrine. But the effective treatment duration of only a week, for patients who face many years of NOH and possible injury, might limit sales until Chelsea can demonstrate that the drug does have longer-term efficacy.

While the FDA may on this occasion have taken a short-term view on the drug, doctors might want to take the long view on Northera.

To contact the writer of this story please email Lisa Urquhart in London at lisau@epvantage.com or follow @LisaEPVantage on Twitter.

EP Vantage would like to apologise to subscribers for the absence of yesterday’s news alert. This was caused by an electrical fault and subsequent power loss at the London office.

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