Download (FDA)顧問小組26日經過表決，建議讓羅氏藥廠(Roche)的藥物

糖尿病水腫藥 可治失明
聯邦食品藥物管理局(FDA)顧問小組 26 日經過表決，建議讓羅氏藥廠(Roche)的藥物 Lucentis 擴
大用於治療糖尿病導致的失明。
顧問小組建議 FDA 核准用劑量 0.5 毫克和 0.3 毫克的 Lucentis，治療糖尿病黃斑部水腫(diabetic
macular edema, DME)。
FDA 預定下個月就此做決定，如它接受建議，Lucentis 將成為第一種獲准用於治療 DME 的藥
物。美國有 50 多萬人罹患這種疾病，而目前只能採用雷射治療。
Lucentis 去年為羅氏藥廠帶來 17 億元營收。紐約州的 Regeneron 製藥公司正研發治療 DME 的
藥物 Eylea，喬治亞州的 Alimera 科學公司和麻州的 Psivida 公司，也在發展治療 DME 的藥物
Iluvien。
FDA 審核羅氏藥廠兩次臨床實驗的結果，發現使用 0.3 毫克劑量的患者，兩年後接受視力測驗，
有 39%的人可以多看清三行；使用 0.5 毫克劑量的人，有 41%視力獲得同樣的改善。接受每月
注射的患者，達到足以維持開車能力的 20/40 視力的機率也顯著提高。
羅氏藥廠的治癌藥癌思停(Avastin)，也被廣泛用於治療濕性老年性黃斑部退化，雖然 FDA 並未
正式核准這種用途。Lucentis 的費用比癌思停高出 40 倍，而專家表示還沒有足夠證據用以判斷
兩者的優劣。
U.S. health advisers back two new eye treatments
By David Morgan | Reuters – Thu, Jul 26, 2012
WASHINGTON (Reuters) - A U.S. Food and Drug Administration panel on Thursday recommended
two drugs for combating different forms of vision loss that can lead to blindness among a range of
adults, including diabetics and the elderly.
The FDA advisory committee of outside experts gave its support to Swiss-based Roche Holding AG's
Lucentis eye drug for treatment of diabetic macular edema and Belgium-based ThromboGenics'
treatment for viteromacular adhesion, a condition that often develops with aging.
The recommendations will now be considered by FDA regulators as they decide whether to approve
the two injectable drugs for sale in the United States.
The news emerged after the companies' shares had ended trading in Europe. Roche shares closed
slightly higher at 168 Swiss francs, while ThromboGenics fell 4.82 percent to 23.30 euros.
The panel gave its blessing to both 0.3 milligram and 0.5 milligram doses of Roche's drug, known
generically as ranibizumab, after reviewing clinical research showing it clearly helps diabetic patients
who suffer from diabetic macular edema, or DME.
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However, some panel members expressed concern about evidence linking higher doses of the drug to
adverse events, including death.
Lucentis, which is made by Roche unit Genentech and administered by monthly injection, is already
approved for two other eye ailments.
"The committee's recommendation is an important step towards improving the care of Americans with
diabetic macular edema. If approved by the FDA, this will be the first major development for treating
diabetic macular edema in more than 25 years," Genentech said in a statement.
The leading cause of vision loss among diabetics, DME occurs when leaking blood vessels in the eye
cause swelling at the center of the retina. The result is a progressive blurring of images near the center
of a patient's field of vision, and sometimes blindness.
About 75,000 new cases of DME are estimated to develop among Americans each year. Genentech
says that about 560,000 in total are affected by the disease.
There are currently no FDA-approved drugs for DME. The condition is currently treated by laser
surgery to stop blood vessels from leaking. The treatment can slow vision loss, but it rarely leads to
vision improvement.
Lucentis is already approved to treat wet age-related macular degeneration - the leading cause of
blindness among the elderly. It is also approved for macular edema following retinal vein occlusion
(RVO), a swelling or thickening of the part of the retina responsible for central vision.
If approved for DME, Lucentis will likely compete with a highly promising drug being tested by
Regeneron Pharmaceuticals Inc and Roche's own cancer drug, Avastin, which works in a similar
manner as Lucentis.
Regeneron shares closed 5.3 percent higher at $135.64 on Nasdaq following the advisory panel's
decision.
The same FDA committee later concluded that ThromboGenics' injectable drug, known generically as
ocriplasmin, helped enough people in clinical trials to warrant a favorable review, despite evidence of
adverse effects, including eye pain, swelling and blurred vision.
Several panel members recommended further studies to monitor safety and determine more fully the
drug's effectiveness.
Vitreomacular adhesion often results from aging and can lead to visual impairment or blindness. But it
currently can be treated only through surgery.
It occurs when vitreous gel detaches at the back of the eye but adheres to the retina as it pulls away,
distorting vision and causing small breaks to develop in the macula, a portion of the retina that is
important for central vision.
Ocriplasmin is ThromboGenics' main drug. The company has secured a deal to sell ocriplasmin in 40
countries outside the United States through Novartis AG's opthalmic division, Alcon.
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ThromboGenics plans to market the product itself within the United States.
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