Chelsea制药1月15日宣布，FDA心血管和肾脏药物顾问委员会（CRDAC）以16比1的投票结果，建议批准该公司的药物Northera（droxidopa，屈昔多巴），用于原发性自主神经衰弱（帕金森病，多系统萎缩症和纯自主神经衰弱）、多巴胺β羟化酶缺乏症、非糖尿病性自主神经病变等患者有症状神经源性体位性低血压（NOH）的治疗。
上周五，FDA内部审查员得出结论，认为Chelsea制药提交的一批新数据不足以支持Northera的获批，并建议FDA心血管和肾脏药物顾问委员会投票拒绝批准该药。
FDA并不受CRDAC建议的约束，但在审查Northera新药申请（NDA）时会加以考虑，该药的处方药用户收费法（PDUFA）目标日期为2014年2月14日。
此前，FDA已授予Northera孤儿药地位，并进入FDA特殊快速通道。特殊快速通道旨在加快那些用于治疗严重或潜在危险生命的疾病的尚未满足医疗需要的药物的审评。
关于
Northera是Chelsea制药的主打研究药物，目前处于临床III期，开发用于治疗原发性自主神经衰弱（如帕金森病、多系统萎缩和单纯性自主神经衰弱）患者的有症状神经源性直立性低血压症状（NOH）。Droxidopa是一合成儿茶酚胺，通过脱羧直接转化为去甲肾上腺素，使得中枢和外周神经系统去甲肾上腺素水平升高。
关于
据估计，在美国和欧盟，有近30万患者患有慢性有症状NOH。有症状NOH是一种慢性疾病，由内在性神经性疾病导致，如帕金森氏病、多系统萎缩症或纯自主神经衰弱。NOH的症状包括头晕、胸闷、视力模糊、乏力、注意力不集中以及站立时昏厥发作。这些症状往往严重限制一个人开展日常活动的能力，患者不得不依靠轮椅生活。
CHARLOTTE, N.C., Jan. 14, 2014 (GLOBE NEWSWIRE) -- Chelsea Therapeutics International, Ltd. (Nasdaq:CHTP) today announced that the Food and Drug Administration's (FDA) Cardiovascular and Renal Drugs Advisory Committee (CRDAC) voted 16-1 to recommend approval of NORTHERA™ (droxidopa) for the treatment of symptomatic neurogenic orthostatic hypotension (nOH) 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.
The FDA is not bound by the CRDAC's recommendation but will take it into consideration when reviewing the New Drug Application (NDA) for Northera. A Prescription Drug User Fee Act (PDUFA) action date for the Northera NDA has been scheduled for February 14, 2014.
"Chelsea is committed to improving the lives of patients with nOH, a debilitating disorder which often severely limits a person's ability to perform routine daily activities," said Joseph G. Oliveto, Interim Chief Executive Officer of Chelsea Therapeutics. "We are appreciative of the FDA bringing our Northera NDA before the Cardiovascular and Renal Drugs Advisory Committee, and for the Committee's thoughtful discussion of the application."
Northera was previously granted Orphan Drug Designation, which is granted by the FDA to treatments for rare diseases/disorders.
About Symptomatic nOH
It is estimated that nearly 300,000 patients suffer from chronic symptomatic nOH in the U.S. and EU combined. Symptomatic nOH is a chronic disorder that is caused by an underlying neurogenic disorder, such as Parkinson's disease, multiple system atrophy or pure autonomic failure. Symptoms of nOH include dizziness, lightheadedness, blurred vision, fatigue, poor concentration, and fainting episodes when a person assumes a standing position. These symptoms often severely limit a person's ability to perform routine daily activities that require standing or walking for both short and long periods of time.
About Northera
NORTHERA™ (droxidopa), the lead investigational agent in Chelsea Therapeutics' pipeline, is currently in Phase III development for the treatment of symptomatic neurogenic orthostatic hypotension (nOH) in patients with primary autonomic failure — an indication that includes a significant number of patients with Parkinson's disease, multiple system atrophy (MSA) and pure autonomic failure (PAF). 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.
Droxidopa, developed by and licensed from Dainippon Sumitomo Pharma Co., Ltd. (DSP), initially received Japanese approval in 1989 for the treatment of frozen gait and dizziness on standing associated with Parkinson's Disease and for the treatment of orthostatic hypotension, syncope or dizziness on standing associated with Shy-Drager syndrome and Familial Amyloidotic Polyneuropathy. In 2000, Droxidopa received expanded marketing approval to include prevention of vertigo, dizziness and weakness associated with orthostatic hypotension in hemodialysis patients.
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