---

原文標題：FDA Approves Treatment for Rare Blood Disease

原文連結：https://www.prnewswire.com/news-releases/fda-approves-treatment-for-
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) inj
ection to treat adults with polycythemia vera, a blood disease that causes the
 overproduction of red blood cells. The excess cells thicken the blood, slowin
g blood flow and increasing the chance of blood clots.

"Over 7,000 rare diseases affect more than 30 million people in the United Sta
tes. Polycythemia vera affects approximately 6,200 Americans each year," said
Ann Farrell, M.D., director of the Division of Non-Malignant Hematology in the
 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 rare
diseases."

Besremi is the first FDA-approved medication for polycythemia vera that patien
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 remove
s excess blood cells though a needle in a vein) as well as medicines to reduce
 the number of blood cells; Besremi is one of these medicines. Besremi is beli
eved to work by attaching to certain receptors in the body, setting off a chai
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 every
two weeks. If Besremi can reduce excess blood cells and maintain normal levels
 for at least one year, then dosing frequency may be reduced to once every fou
r weeks.

The effectiveness and safety of Besremi were evaluated in a multicenter, singl
e-arm trial that lasted 7.5 years. In this trial, 51 adults with polycythemia
vera received Besremi for an average of about five years. Besremi's effectiven
ess was assessed by looking at how many patients achieved complete hematologic
al response, which meant that patients had a red blood cell volume of less tha
n 45% without a recent phlebotomy, normal white cell counts and platelet count
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 infectiou
s diseases, which could lead to life-threatening or fatal complications. Patie
nts who must not take Besremi include those who are allergic to the drug, thos
e with a severe psychiatric disorder or a history of a severe psychiatric diso
rder, immunosuppressed transplant recipients, certain patients with autoimmune
 disease or a history of autoimmune disease, and patients with liver disease.

People who could be pregnant should be tested for pregnancy before using Besre
mi due to the risk of fetal harm.

Besremi received orphan drug designation for this indication. Orphan drug desi
gnation provides incentives to assist and encourage drug development for rare
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




--
--
※ 發信站: 批踢踢實業坊(ptt.cc), 來自: 101.9.138.102 (臺灣)
※ 文章代碼(AID): #1XZlDXpk (Stock)
※ 文章網址: https://www.ptt.cc/bbs/Stock/M.1636758369.A.CEE.html