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FDA再次驳回Chelsea的新药Northera

  1. Chelsea
  2. FDA
  3. Northera

来源:生物谷 2014-01-14 09:00

2014年1月13日讯 /生物谷BIOON/ --最近一年,Chelsea生物医药公司经历了一段肯困难的时期。公司生产的用于治疗患者因为神经系统紊乱而导致的昏厥症药物Northera在提交FDA审批时遭遇了很大的阻力。尽管公司积极地补充相关研究数据,FDA在上周仍然再次驳回了公司的上市申请。FDA认为公司此次补充的数据仍然不能证明该药物的有效性。FDA负责这次审批的工作人员Shari Targum认为Chelsea公司此次提交的306B研究虽然加上了一些可靠的研究数据,但这些研究数据并未能达到FDA要求的标准,因而不具有说服力。受此消息影响公司股价重挫26%。

这也使Chelsea公司处于极其艰难的境地,公司不得不继续进行进一步的研究以说明Northera的有效性。然而即使最终FDA通过了其上市申请,FDA对Northera不甚积极的态度也会影响其未来的市场推广。(生物谷Bioon.com)

详细英文报道:

Chelsea Therapeutics was struggling to overcome a series of setbacks a year ago, which had been crowned by the FDA's rejection of Northera (droxidopa). And then the biotech made a surprise announcement: The agency was willing to consider data from another study it had been working on rather than force the company to mount a new clinical trial. And the news inspired a long run-up in the biotech's stock price ($CHTP).

Until today.

After reading the FDA's internal review of Chelsea's new pitch, some investors are likely to question just why they had become so optimistic about the therapy, which is designed to prevent dizziness and fainting among patients with disorders of the nervous system.

Ahead of the Tuesday advisory committee meeting on Northera, the FDA's Shari Targum concludes that a fresh batch of data failed to deliver the proof needed for an approval, recommending that the experts vote for a rejection.

Shares of the company quickly fell 26%.

The crux of the FDA's problem gets down to the new trial data that Chelsea had hoped would provide the basis for an approval. The biotech had hoped that its 306B study would add additional pivotal data that could convince the agency the drug is effective--after the agency had initially turned its thumb down on the drug based on inadequate efficacy and safety data. But a changeup in the trial design may have fatally damaged the data.

"If we give the applicant the benefit of the doubt, and consider study 306B to support efficacy, the results do not meet the criteria as a "robust" or "strongly positive" single study to support a symptom benefit," writes Targum. "This conclusion is based on the small treatment effect, exceeded by the 3-fold higher intra-subject variability. In addition, more patients on droxidopa (vs. placebo) discontinued prior to the first post-randomization OHSA-item 1 (even if patients discontinuing from 306A are counted in discontinuations in 306B), presenting a dilemma in how to interpret the missing OHSA item-1 data."

Not only that, she adds, but data from two studies "support a lack of effect durability in this chronic condition."

The bottom line: "This reviewer recommends a Complete Response action (rejection) for droxidopa in the treatment of symptomatic neurogenic orthostatic hypotension (NOH), because of inadequate evidence of effectiveness."

Chelsea clearly faces an uphill climb during the panel session ahead. It will have to bring a great game to the court if it expects to overcome these kinds of doubts. And even if it does pull off a surprise win next week, this kind of review raises serious doubts about the drug's near-term future.

 

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