UPDATED: FDA rejects Chelsea’s Northera, demands additional study data

Posted under Biotech Stock, Blog, Chelsea Therapeutics, Companies, Diagnostics, Droxidopa, FDA approval, Funding, Medical Devices, Medical Supply, neurogenic orthostatic hypotension, Northera, Pharmaceuticals, Pipeline, Regulatory, Startups, Universities, Videos by john

After the markets closed on Wednesday, Chelsea Therapeutics ($CHTP) put out the word that the FDA had rejected Northera, a troubled program that drew regulatory frowns for an absence of long-term efficacy data and troubling safety signals. In handing Chelsea a complete response letter, the agency opted to overlook the majority vote in favor of Northera by a panel of outside experts, where Northera earned some grudging respect. 

The FDA concluded that Chelsea needed to file positive data from an additional two- to three-month study, and Chelsea immediately raised the prospect that an ongoing 10-week trial could provide the data it needs. That didn't sit well with investors, though. Chelsea's shares plunged more than 30% in a matter of minutes.

"We believe there continues to be an important unmet medical need in addressing the symptoms associated with Neurogenic OH and remain committed to working with the FDA to determine the appropriate next steps required to bring a much-needed new therapy to the market as quickly as possible," said CEO Simon Pedder in a statement.

Analysts had been kept guessing on the outcome throughout the day, balancing the official stance of the FDA with the 7-4 panel vote and the simple fact that this drug has been available for years in Japan. For Chelsea, that track record had initially allowed the biotech to claim that their program had been significantly de-risked. Advocates maintained that Northera--or droxidopa--offered perhaps the best hope for patients with symptomatic neurogenic orthostatic hypotension, the sudden drop in blood pressure that can trigger fits of dizziness for some 180,000 patients.

For some analysts, the first of two Phase III studies was considered something of a cakewalk. But Northera flunked that study. A subsequent Phase III produced positive data, adding to the biotech's reputation for treating investors to a roller coaster ride on its share price.

Even if it is approved at a later date, the FDA is already considering a black box warning on safety concerns. FDA reviewers have raised concerns about links to a neurological condition.

- here's the release
- here's the Bloomberg report

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Conflicting signals cloud Chelsea’s chances on D-Day for Northera

Posted under Blog, Chelsea Therapeutics, Companies, Diagnostics, Droxidopa, FDA, Funding, Medical Devices, Medical Supply, neurogenic orthostatic hypotension, Northera, Pharmaceuticals, Phase III, rare diseases, Startups, Universities, Videos by John Carroll

It's D-Day for Chelsea Therapeutics and its rare disease drug Northera. The FDA is due to deliver its official verdict on a therapy that's been in use for years in Japan. But this therapy caused more than one case of heartburn for Chelsea ($CHTP) with split results in two Phase III studies, dour safety warnings from FDA staffers and only a majority 7-to-4 vote in its favor by an FDA expert panel.

The last cliffhanger for the Charlotte, NC-based biotech occurred about a month ago when a majority of the agency's panel concluded, rather reluctantly, that Northera--or droxidopa--offered perhaps the best hope for patients with symptomatic neurogenic orthostatic hypotension, the sudden drop in blood pressure that can trigger fits of dizziness for some 180,000 patients. Advocates concluded that the risks could be managed and the potential benefits were significant. But the agency had earlier recommended against an approval, noting that the treatment is linked to lethal cases of neuroleptic malignant syndrome while there's no data to support a case for long-term efficacy.

The mixed signals are giving investors a bad case of heartburn of their own today. As Brian Orelli at Motley Fool remarks, the company's share price has been trading down a bit following the spike that occurred on the panel vote in its favor, indicating that there's a lot of bets being made for a rejection in the form of a complete response letter. So if it is approved, the thumbs-up is likely to trigger a neck-breaking ride straight up.

For Chelsea, an approval would give the biotech its first marketed drug, giving it a green light to set up its own marketing effort in the U.S. while finding partners to handle international markets.

But it could go either way. We'll keep you posted when the news breaks.

- MedCity News lines up the arguments for and against
- here's the story from Motley Fool

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Chelsea Therapeutics surges on FDA panel nod for Northera
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Chelsea Therapeutics reports positive efficacy results in PhII fibromyalgia study

Posted under Blog, Chelsea Therapeutics, Companies, Dainippon Sumitomo, Diagnostics, Droxidopa, Fibromyalgia, Funding, Medical Devices, Medical Supply, Pharmaceuticals, Startups, Universities, Videos by John Carroll

Chelsea Therapeutics ($CHTP) has racked up some more positive efficacy data for its lead drug droxidopa, which is now up for review at the FDA. Investigators reported the drug spurred a statistically significant response on some key measures of fibromyalgia in the Phase II study, which was designed to test a range of doses as well as a combo approach with carbidopa. The biotech recruited 120 patients for the study, ultimately zeroing in on 7 separate arms.

Charlotte, NC-based Chelsea designed the trial with the understanding that droxidopa, a synthetic amino acid, is converted by the body into norepinephrine. By boosting levels of norepinephrine, Chelsea believes it can directly address some of the disease's severe side effects. Shares of Chelsea were up slightly this morning.

"Since norepinephrine is a key neurotransmitter involved with the modulation of chronic pain, we were not surprised to see evidence of droxidopa's therapeutic benefit in treating patients with fibromyalgia," stated Dr. Art Hewitt, Chelsea's chief scientific officer. "As we continue to evaluate potential indications for droxidopa such as fibromyalgia and adult attention deficit disorder, trials like this provide insight into how to optimize dosing for more robust future clinical evaluations. Given the broad biologic activity of norepinephrine, we continue to believe there are wide-ranging therapeutic applications for a first-in-class oral prodrug of norepinephrine. We also continue to be encouraged by the remarkable safety profile of droxidopa which, even at its highest dose, again proved not to be associated with any serious or significant adverse events."

A few weeks ago the FDA accepted Chelsea's application for droxidopa as a new treatment for hypotension. The drug, in-licensed from Japan's Dainippon Sumitomo 5 years ago, flunked its first Phase III, but Chelsea regrouped and announced positive data in 2010. Regulators are expected to make its final decision on the program by March 28 under a priority review.

- here's the press release
- read the Reuters story

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Chelsea shares rocket up after lead drug clears PhIII