Thursday, February 6, 2014

藥華 布局罕見疾病: Polycythemia vera真性紅血球增多症

Polycythemia vera is a bone marrow disease that leads to an abnormal increase in the number of blood cells (primarily red blood cells).

Causes Polycythemia vera is a disorder of the bone marrow. It mainly causes too much production of red blood cells, although the numbers of white blood cells and platelets are also increased. It is a rare disease that occurs more often in men than women, and is rare in patients under age 40. It is usually associated with a gene mutation called JAK2V617F. The cause of this mutation is unknown.

Treatment The goal of treatment is to reduce the thickness of the blood and prevent bleeding and clotting. A method called phlebotomy is used to decrease blood thickness. One unit of blood (about 1 pint) is removed weekly until the hematocrit level is less than 45 (males) or 42 (females). Then therapy is continued as needed. Occasionally, chemotherapy (specifically hydroxyurea) may be given to reduce the number of red blood cells made by the bone marrow. Interferon may also be given to lower blood counts. A medicine called anagrelide may be given to lower platelet counts. Some patients are advised to take aspirin to reduce the risk of blood clots, though it increases the risk for stomach bleeding. Ultraviolet-B light therapy can reduce the severe itching some patients experience.

生技大咖楊育民 任藥華獨董 2014-01-06 01:48 工商時報 記者杜蕙蓉/台北報導 國際生技專家楊育民將擔任藥華醫藥獨董一職,預期將對國內研發蛋白質新藥添推升動力。圖/本報資料照片 國際生技專家楊育民將擔任藥華醫藥獨董一職,預期將對國內研發蛋白質新藥添推升動力。圖/本報資料照片生醫產業再添人才動能!國際知名生技專家楊育民昨(5)日證實將擔任藥華醫藥獨立董事,由於楊育民專長是蛋白質藥、單株抗體的生產,是國內目前最欠缺的一環,預期他的加入,將引動海外生技專家回流潮,對台灣發展蛋白質新藥領域將有極大助力。已通過公開發行的藥華,預計2月底可望登錄興櫃,該股未上市行情約200元,藥華目前最具潛力的新藥是已進入三期臨床用於治療真性紅血球增多症的P1101,預計在2015年底或2016年初,同步取得歐盟EMA和美國FDA上市許可藥證。藥華總經理林國鐘表示,由於真性紅血球增多症全球尚無藥可醫,目前美國FDA傾向直接以歐盟三期數據為主,因此藥華去年募資18億元,其中15億(5千萬美元),原本用來進行FDA三期臨床,如今看來,該筆花費應可「省下來」。楊育民表示,藥華的新藥開發已具有相當基礎。他很贊成藥華的「新藥研發,並製造優良有效的治療藥物」的宗旨和願景。希望自己可以對藥華實質的營運和新藥開發做一點點小幫助。藥華目前有5個新藥研發中,進度最快P1101,該藥在台灣TFDA的臨床實驗是以治療慢性C型肝炎和B型肝炎為主。由於P1101的適應症還包括血小板增生症、慢性骨髓性白血病和真性紅血球增多症等2009P1101授權給奧地利藥廠AOP後,AOP將其適應症應用於治療真性紅血球增多症,並取得歐洲EMA及美國FDA的孤兒藥認證。藥華董事長詹青柳表示,真性紅血球增多症,為罕見疾病,病患由於紅血球增生太快,常常需要放血,目前歐洲病患數約12萬人,美國10萬人。由於全球尚無藥可醫治中,P1101的臨床是採開放式(Open Label),即病患的治療效果3個月即可測出是否繼續放血?紅血球質是否正常?為此,P1101有效性很容易在3個月內就發現,如歐盟臨床三期數據正面,基於國際臨床ICH的原則,加上全球缺藥,美國FDA初步考量將共用歐盟數據,讓P1101可免做三期臨床,並同步取得歐盟EMA和美國FDA上市許可藥證,進軍全球初估約6.15億美元市場。

 

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