Re: [新聞] 藥華藥 獲美國 FDA核准一線用藥

[ichitakajoe (joe)]

※ 引述《sandya ()》之銘言：
: 原文標題：FDA Approves Treatment for Rare Blood Disease
: 原文連結：https://www.prnewswire.com/news-releases/fda-approves-treatment-fo
r-
: rare-blood-disease-301423378.html
: 發布時間：Nov 12, 2021, 16:55 ET
: 原文內容：SILVER SPRING, Md., Nov. 12, 2021 /PRNewswire/ -- Today, the U.S.
Fo
: od and Drug Administration approved Besremi (ropeginterferon alfa-2b-njft) i
nj
: ection to treat adults with polycythemia vera, a blood disease that causes t
he
:  overproduction of red blood cells. The excess cells thicken the blood, slow
in
: g blood flow and increasing the chance of blood clots.
: "Over 7,000 rare diseases affect more than 30 million people in the United S
ta
: tes. Polycythemia vera affects approximately 6,200 Americans each year," sai
d
: Ann Farrell, M.D., director of the Division of Non-Malignant Hematology in t
he
:  FDA's Center for Drug Evaluation and Research. "This action highlights the
FD
: A's commitment to helping make new treatments available to patients with rar
e
: diseases."
: Besremi is the first FDA-approved medication for polycythemia vera that pati
en
: ts can take regardless of their treatment history, and the first interferon
th
: erapy specifically approved for polycythemia vera.
: Treatment for polycythemia vera includes phlebotomies (a procedure that remo
ve
: s excess blood cells though a needle in a vein) as well as medicines to redu
ce
:  the number of blood cells; Besremi is one of these medicines. Besremi is be
li
: eved to work by attaching to certain receptors in the body, setting off a ch
ai
: n reaction that makes the bone marrow reduce blood cell production. Besremi
is
:  a long-acting drug that patients take by injection under the skin once ever
y
: two weeks. If Besremi can reduce excess blood cells and maintain normal leve
ls
:  for at least one year, then dosing frequency may be reduced to once every f
ou
: r weeks.
: The effectiveness and safety of Besremi were evaluated in a multicenter, sin
gl
: e-arm trial that lasted 7.5 years. In this trial, 51 adults with polycythemi
a
: vera received Besremi for an average of about five years. Besremi's effectiv
en
: ess was assessed by looking at how many patients achieved complete hematolog
ic
: al response, which meant that patients had a red blood cell volume of less t
ha
: n 45% without a recent phlebotomy, normal white cell counts and platelet cou
nt
: s, a normal spleen size, and no blood clots. Overall, 61% of patients had a
co
: mplete hematological response.
: Besremi can cause liver enzyme elevations, low levels of white blood cells,
lo
: w levels of platelets, joint pain, fatigue, itching, upper airway infection,
 m
: uscle pain and flu-like illness. Side effects may also include urinary tract
 i
: nfection, depression and transient ischemic attacks (stroke-like attacks).
: Interferon alfa products like Besremi may cause or worsen neuropsychiatric,
au
: toimmune, ischemic (not enough blood flow to a part of the body) and infecti
ou
: s diseases, which could lead to life-threatening or fatal complications. Pat
ie
: nts who must not take Besremi include those who are allergic to the drug, th
os
: e with a severe psychiatric disorder or a history of a severe psychiatric di
so
: rder, immunosuppressed transplant recipients, certain patients with autoimmu
ne
:  disease or a history of autoimmune disease, and patients with liver disease
.
: People who could be pregnant should be tested for pregnancy before using Bes
re
: mi due to the risk of fetal harm.
: Besremi received orphan drug designation for this indication. Orphan drug de
si
: gnation provides incentives to assist and encourage drug development for rar
e
: diseases.
: The FDA granted the approval of Besremi to PharmaEssentia Corporation.
: 心得/評論： 台灣之光
: 市場之大 後續看好
: 一線用藥股價才便宜的100
: 感謝全額交割股 讓小股民有機會進場
有人表示 一線用藥跟放血阿斯匹靈一樣
醫師不一定要使用

但事實是，放血跟阿斯匹靈根本只能治標，不會治療PV ET疾病，許多病人後來只好吃HU
（便宜），但是承受副作用。

負擔得起的病人就用Pegasys （無FDA）or Jakafi （第二線）
病友論壇之前很多人是使用Pegasys，但是在FDA唯一許可Besremi一線使用後，這個局勢
應該會改變。

加上原本使用Jakafi的二線病患，也有可能轉換過來（之後Besremi還有ET MPN的研究）

營收樂觀不是空穴來風。
至於歐洲為什麼賣不好，要問AOP

至少美國這部分FDA放一線指引一定是有影響力的。

--
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推 kairiyu     : 反觀高X
噓 SRadiant    : 這個疾病本來就只能治標啊，能治本？
→ SRadiant    : 治本要找基因療法囉
→ ichitakajoe : SR大馬上出來囉～
→ ichitakajoe : 干擾素可以減少PV轉成MPN的機率 不是嗎？
→ ichitakajoe : 所以你說的其他一線跟Besremi一樣嗎 科科
→ ichitakajoe : 研究都還在做 你就知道不能改善？
→ KID0924     : 明天市價買買的到嬤
→ s821088     : 裝睡的人叫不醒
推 zks6699     : 歐洲的用量用法值得研究一下，不要一昧聽新聞講的，
→ zks6699     : 一針250ug，在歐洲真的有人打過嗎
推 woods0598   : 用量沒差，美國是賣療程的
→ woods0598   : SR大 基因是改變不了沒錯 但類似TKI在CML中的角色
→ woods0598   : 能夠達到分子學反應且停藥後可維持 基本就等於治本
→ woods0598   : 可以看incyte的營收, 美國自己做, 全球給諾華做
→ woods0598   : 結果諾華在jakavi的全球營收還低美國15%..
推 woods0598   : https://reurl.cc/Xlyg2j
→ woods0598   : 干擾素可抑制疾病進展 https://reurl.cc/Okngz9
→ woods0598   : 去年就在ASH發表，今年3月正式在Leukemia發表
→ ichitakajoe : 看來woods大懂我的意思 （握）