帕金森症患者的福音！FDA批准艾伯维帕金森症长效疗法上市

随着老年化社会的进程，医药企业都越来越重视如帕金森症、阿尔兹海默症等神经退行性疾病的研究。然而，限于目前医学界对这类疾病认识水平，这类药物也一直是医药产业界的重灾区。不过，最近艾伯维宣布公司开发的用于治疗晚期帕金森症患者运动波动的药物DUOPA获得FDA的上市批准，这一利好消息无疑对今后生物医药产业界更好地开发这类药物打了一针强心剂。

DUOPA是目前市面上第一种畅销治疗帕金森症患者运动波动的药物。它主要是通过一个小型药物泵将卡比多巴和左旋多巴注入人体，以达到治疗效果。这种疗法的持续时间可以长达16个小时，极大程度上降低了患者的负担和痛苦。与传统的口服卡比多巴和左旋多巴不同，DUOPA是将药物直接泵入到小肠部位，避过了患者的胃部，从而避免了胃部的酸性环境和酶类对药物的破坏，保证药效。

此次FDA批准DUOPA作为罕见病药物上市。研究人员称，这一决定将在很大程度上缓解目前重度帕金森症患者对治疗运动波动的疗法的需求。临床三期研究显示，使用了DUOPA的患者临床症状获得了明显缓解。

所谓帕金森症，是一种神经退行性疾病，它可以造成患者出现震颤、肌肉僵硬、动作缓慢等等症状，这主要是与大脑中一种分泌多巴胺的细胞数量减少直接相关。目前，科学家们尚没有办法治愈这种疾病，仅能通过药物缓解这种疾病的症状。据估计，美国目前有一百万人患有这种疾病，而每年还有6万例新增病例出现。

详细英文报道：

NORTH CHICAGO, Ill., Jan. 12, 2015 -- The U.S. Food and Drug Administration (FDA) has approved AbbVie's (NYSE: ABBV) DUOPA? (carbidopa and levodopa) enteral suspension for the treatment of motor fluctuations for people with advanced Parkinson's disease. DUOPA is administered using a small, portable infusion pump that delivers carbidopa and levodopa directly into the small intestine for 16 continuous hours via a procedurally-placed tube.

DUOPA was approved by the FDA as an orphan drug, a designation granted to products intended for the treatment of rare diseases or conditions affecting fewer than 200,000 patients in the U.S.

"There is unmet need for treatment options for patients with advanced Parkinson's disease. As the disease advances, it can be difficult to control motor features," said C. Warren Olanow, M.D., Professor, Department of Neurology and Department of Neuroscience, Mount Sinai School of Medicine, and lead investigator of the DUOPA pivotal trial. "In clinical trials, DUOPA was shown to significantly reduce the amount of off time advanced Parkinson's disease patients experienced."

In the advanced stages of Parkinson's disease, patients may begin to experience "off" time, or periods of poor mobility, slowness and stiffness. Additionally, in Parkinson's disease patients, the spontaneous emptying of the stomach becomes delayed and unpredictable, which can affect the timing of when orally administered medicines leave the stomach and are absorbed in the small intestine. DUOPA provides patients with the same active ingredients as orally-administered carbidopa and levodopa immediate release, but is delivered in a suspension that goes directly into the small intestine via a tube placed by a percutaneous endoscopic gastrostomy procedure with jejunal extension (PEG-J). This type of administration is intended to bypass the stomach.

"The FDA approval of DUOPA is another significant milestone for AbbVie's pipeline," saidMichael Severino, M.D., Executive Vice President, Research and Development and Chief Scientific Officer, AbbVie. "This advancement is important for patients with advanced Parkinson's disease and their care teams, as it provides a new therapeutic option to help manage motor symptoms."

"Due to the progressive nature of Parkinson's disease, it can be difficult to treat over time, especially in the advanced stages," said Joyce Oberdorf, President and CEO, National Parkinson Foundation. "Our organization is encouraged by the introduction of a new therapy that may provide another treatment option for affected patients and families."

About the Duopa Clinical Trial Program

The DUOPA approval is based on a Phase 3, 12-week, double-blind, double-placebo, active control, parallel group, multi-center trial (N=71) that compared the efficacy and safety of DUOPA to oral, immediate-release (IR) carbidopa-levodopa tablets in advanced Parkinson's disease patients. The study showed that DUOPA significantly reduced daily (per 16 waking hours) mean "off" time at 12 weeks by four hours, which resulted in an average of 1.9 fewer hours of "off time" when compared to carbidopa-levodopa IR tablets. Treatment with DUOPA was also associated with an improved mean "on" time (periods when the medication is working and symptoms are controlled) without troublesome dyskinesia (uncontrolled movement that does not interfere with normal daily activities) by four hours at 12 weeks, which resulted in an average of 1.9 hours more "on" time when compared to carbidopa-levodopa IR tablets. The most common adverse events (>7% and greater than carbidopa and levodopa immediate release) were complication of device insertion, nausea, constipation, incision site erythema, dyskinesia, depression, post procedural discharge, peripheral edema, hypertension, upper respiratory tract infection, oropharyngeal pain, atelectasis, confusional state, anxiety, dizziness and hiatal hernia.

About Parkinson's Disease

Parkinson's disease is a progressive and chronic movement disorder1 characterized by tremor, muscle rigidity, slowness of movement and difficulty with balance.2 It is classified as a movement disorder resulting from the loss of dopamine-producing brain cells.3 The motor symptoms of Parkinson's disease begin when approximately 60-80 percent of the dopamine-producing cells in the brain are lost and symptoms continue to worsen slowly over the course of time.4 While there is no known cure for the disease, there are treatments available to help reduce symptoms.3

As Parkinson's disease progresses, patients may experience fluctuations from an "on" state to an "off state," during which they are slower, stiffer and experience more difficulty moving. Patients may also experience dyskinesias (involuntary movements).

In the United States, it is estimated that 60,000 new cases of Parkinson's disease are reported each year, adding to as many as 1 million people who currently have the disease.

AbbVie has launched a product support program, called DuoConnect?, which is intended to provide a broad range of support for DUOPA patients. The DuoConnect program can be accessed soon by calling 1-844-DUO-4YOU (1-844-386-4968).

Additionally, for people living with advanced Parkinson's disease who face financial difficulties, the AbbVie Patient Assistance Program may provide DUOPA at no cost. A co-pay assistance program will be available for commercially-insured patients being treated with DUOPA.

AbbVie also supports independent non-profit organizations that assist eligible patients enrolled in federal and private insurance plans with their out-of-pocket medication costs.

About DUOPA (carbidopa and levodopa) enteral suspension

DUOPA is a new approach to the administration of carbidopa and levodopa for the treatment of motor fluctuations for people with advanced Parkinson's disease. Carbidopa-levodopa is widely recognized as an effective treatment for motor fluctuations of the disease.5 DUOPA was reviewed and approved by the FDA as a combination product with use of the CADD Legacy 1400 pump.

Carbidopa and levodopa enteral suspension is currently approved in 41 countries and is marketed by AbbVie as DUODOPA? outside the United States.