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 surges on FDA panel nod for Northera

Posted under Blog, Chelsea Therapeutics, Companies, Diagnostics, Funding, Medical Devices, Medical Supply, Northera, Pharmaceuticals, Startups, Universities, Videos by Ryan McBride

FDA panelists' opinions about the benefits of hypotension drug Northera clashed with those in an FDA staff review of the drug, and the agency's advisory committee backed the treatment for U.S. approval. Shares of Chelsea Therapeutics International ($CHTP), the drug's developer, rallied in after-market trading and were up 73% early this morning.

The non-agency experts on the FDA panel voted 7-4 in favor of approval Thursday, deciding that the benefits of the drug for treating symptomatic neurogenic orthostatic hypotension outweighed the risks that were highlighted in the FDA staff review earlier this week, Reuters reported. Despite cases of deadly neuroleptic malignant syndrome in patients who took the drug in Japan, where the drug has been in use for decades, FDA panel members weighed heavily the lack of any effective treatments for the chronic disease in the U.S.

"If there were other treatments available, the answer would be 'no.' But there aren't," said panelist Allan Coukell of Pew Charitable Trusts, as quoted by Reuters.

While the FDA doesn't have to follow suit with the panel in its ultimate decision on Northera's U.S. approval, investors appeared to be betting up the odds of Chelsea winning the agency's nod and bringing its first product to market. And the Charlotte, NC-based drug developer is poised to recover from the big hit its stock took after the agency staff review said loud and clear Tuesday that the drug shouldn't be approved.  

The FDA is expected to decide by March 28 the fate of Chelsea's drug, which aims to combat the blood-pressure drop in some 180,000 patients with the disorder, Bloomberg reported. The symptoms of the disease such as dizziness and fainting can force some patients into wheelchairs.

- here's the Reuters article
- see Bloomberg's report

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FDA staffers advise agency to deny Chelsea's key drug
Chelsea shares nosedive on FDA's questions about key drug

FDA staffers advise agency to deny Chelsea’s key drug

Posted under Blog, Chelsea Therapeutics, Companies, Diagnostics, Funding, Medical Devices, Medical Supply, Northera, Pharmaceuticals, Startups, Universities, Videos by Ryan McBride

There's no ambiguity about this FDA staff review. In briefing docs on Chelsea Therapeutics' ($CHTP) bid to gain approval of its hypotension drug Northera (droxidopa), agency staffers recommended against approval, confirming investors' worries about the drug's prospects in the U.S. market.

The FDA staff noted the "worrisome" safety issues in clinical trials, including deaths, strokes, myocardial infarction and disease progression, according to the agency's briefing docs. Also sparking concern is a post-marketing study in Japan, where the drug is approved, that identified cases of a potentially deadly neurological condition known as neuroleptic malignant syndrome in patients who took the drug.

"On the basis of the safety concerns compounded by absence of evidence of durability of effect, my regulatory recommendation is that we should not grant approval for droxidopa [Northera] at this time," FDA staffers said in their report.

Chelsea's shares were down by 15.7% as of 11:46 am ET today. In fact, its share price has taken a beating since the company alerted investors last week the FDA had questions about the relatively small size of its clinical trials for the drug (along with other concerns). The drug is supposed to treat a drop in blood pressure that happens within three minutes of a patient standing up, Bloomberg reported, noting Dainippon Sumitomo Pharma markets the drug in Japan with annual revenue of $50 million.

Non-FDA experts are due to meet Feb. 23 to review Chelsea's Northera application and decide whether to recommend approval. The FDA doesn't have to--but often does--take such panels' recommendations, and the agency is expected to decide whether to stamp an approval on the drug by March 28.

- here’s the Bloomberg article

- see the update from RTTNews

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Chelsea shares nosedive on FDA's questions about key drug