FDA核准吸入型Aztreonam治療囊腫纖維症
作者：Yael Waknine
出處：WebMD醫學新聞
February 24, 2010 — 美國食品藥物管理局(FDA)核准aztreonam吸入型溶液(商品名Cayston，Gilead Sciences公司)，以改善綠膿桿菌(Pseudomonas aeruginosa)感染之囊腫纖維症(cystic fibrosis，CF)病患的呼吸道症狀，此項產品預計在下週末前於特定藥局上市。
大約90%的CF病患一生中有某個時間點感染有綠膿桿菌，這是造成感染與發炎之惡性循環的一個重要因子，導致支氣管擴張症，最後造成呼吸衰竭。
作為十多年來第一個被核准用於CF病患的新型吸入型抗生素，aztreonam代表的是那些對現有治療產生阻抗性之CF病患殷殷期盼的治療選項。
囊腫纖維症基金會總裁暨執行長Robert J. Beall博士在新聞稿中表示，我們樂見在我們的「武器清單」中多一種新的抗生素，來對抗此一疾病的相關致命感染威脅，如同我們持續推動其他治療來解決CF的潛在原因，該基金會投資了100萬美元來幫助發展治療。
Aztreonam吸入型溶液是在28天療程中，給予每天3次、每次劑量75-mg，透過一個藥物專屬攜帶式吸入系統(商品名Altera，PARI Pharma GmbH公司)給予此抗生素，該系統使用專利的eFlow技術在3分鐘內給予每次劑量，因而可以減輕CF病患一般需要每天3-4小時治療的負擔。
該產品之核准是根據一個28天研究的資料，研究對象是164名7歲以上、預測一秒內用力呼氣容積(FEV1)25%-75%的病患(平均為55%)，他們被隨機指派接受每天3次的75 mg aztreonam吸入型溶液或者安慰劑，加上標準的CF照護。
治療最後一天時的結果顯示，相較於安慰劑，aztreonam顯著改善了呼吸道症狀；小兒病患的平均改善程度大於成人病患。
相較於安慰劑，使用aztreonam治療之病患的肺功能也有顯著改善(FEV1治療差異為10%；95%信心區間[CI]為 6% - 14%)，小兒病患與成人病患之間沒有差異。
完成治療後兩週，治療組之間的呼吸道症狀有一點點不同，FEV1的差異降低到6% (95% CI，2% - 9%)。
與aztreonam治療有關的最常見(發生率> 5%)副作用包括咳嗽、鼻塞、呼吸喘、咽喉疼痛、發燒、胸部不適、腹部疼痛與嘔吐。
為了協助與支持病患，該公司運用囊腫纖維症基金會的非營利補助成立一個客服中心，「Cayston Access Program」這項計畫將協助CF病患與照護團隊成員進行保險確認、共同給付輔助以及權利支持等。可於該計畫的網站或來電1-877-7CAYSTON begin_of_the_skype_highlighting 1-877-7CAYSTON end_of_the_skype_highlighting (877-722-9786 begin_of_the_skype_highlighting 877-722-9786 end_of_the_skype_highlighting)獲得更多資訊。
FDA Approves Inhaled Aztreonam for Cystic Fibrosis
By Yael Waknine
Medscape Medical News
February 24, 2010 — The US Food and Drug Administration (FDA) has approved aztreonam inhalation solution (Cayston, Gilead Sciences, Inc) to improve respiratory symptoms in patients with cystic fibrosis (CF) with Pseudomonas aeruginosa. The product is expected to be available in specialty pharmacies by the end of next week.
P aeruginosa colonization occurs in about 90% of patients with CF at some point during their lives and is an important contributor to the vicious cycle of infection and inflammation leading to bronchiectasis and eventual respiratory failure.
As the first new inhaled antibiotic to be approved for CF patients in more than a decade, aztreonam represents a much-needed therapeutic alternative for CF patients who have developed resistance to current therapies.
"We are delighted to have a new antibiotic in the arsenal to help fight the life-threatening infections associated with this disease, as we continue to push forward with other therapies that address the underlying cause of CF," said Robert J. Beall, PhD, president and chief executive officer of the Cystic Fibrosis Foundation, in a news release. The foundation invested $1 million in helping develop the therapy.
Aztreonam inhalation solution is administered as a 75-mg dose 3 times daily during a 28-day period. The antibiotic is delivered via a drug-specific portable nebulizer system (Altera, PARI Pharma GmbH) that uses proprietary eFlow technology to deliver each dose in less than 3 minutes, thereby reducing the burden for CF patients who often require 3 to 4 hours of treatment daily.
The product's approval was based on data from a 28-day study of 164 patients aged 7 years and older with forced expiratory volume in 1 second (FEV1) of 25% to 75% predicted (mean, 55%), who were randomly assigned to receive either 75 mg aztreonam inhalation solution or placebo 3 times daily, in addition to standard CF care.
Results on the last day of treatment showed that aztreonam significantly improved respiratory symptoms from baseline relative to placebo; mean improvements were substantially greater for pediatric compared with adult patients.
Pulmonary function was also significantly improved in aztreonam-treated patients relative to placebo (FEV1 treatment difference, 10%; 95% confidence interval [CI], 6% - 14%), and no differences were noted between adult and pediatric patients.
Two weeks after completion of therapy, a smaller difference in respiratory symptoms between treatment groups remained and the difference in FEV1 had decreased to 6% (95% CI, 2% - 9%).
Adverse events most commonly associated with aztreonam therapy (prevalence > 5%) included cough, nasal congestion, wheezing, pharyngolaryngeal pain, pyrexia, chest discomfort, abdominal pain, and vomiting.
To support patients, the company is launching a call center that was developed with a nonprofit subsidiary of the Cystic Fibrosis Foundation. The Cayston Access Program will assist people with CF and members of their care team with insurance verification, copay assistance, and claims support. Additional information is available online on the program's Web site or by calling 1-877-7CAYSTON begin_of_the_skype_highlighting
