European Biotech Pharming Group Touts Positive Data From Pivotal Pediatric Study Of FDA-Approved Immune System Disorder Drug
European Biotech Pharming Group Touts Positive Data From Pivotal Pediatric Study Of FDA-Approved Immune System Disorder Drug
Pharming plans to include data from this 4-11-year-old trial in regulatory filings worldwide for the approval of leniolisib for pediatric patients with APDS, beginning in 2025.On Wednesday, Pharming Group N.V. (NASDAQ:PHAR) revealed topline results of data from its Phase 3 trial evaluating leniolisib in children aged 4 to 11 years with activated phosphoinositide 3-kinase delta syndrome (APDS).
在週三，Pharming Group N.V.（納斯達克：PHAR）公佈了其評估leniolisib在4至11歲兒童中治療激活的磷脂酰肌醇3-激酶δ綜合症（APDS）的第三階段試驗的頂線數據。
APDS is a genetic disorder that impairs the immune system. Individuals with this condition often have low numbers of white blood cells, particularly certain types of B and T cells. These cells are necessary to recognize and attack foreign invaders, such as viruses and bacteria, to prevent infection.
APDS是一種損害免疫系統的遺傳疾病。患有這種控件的人通常白細胞數量較少，尤其是某些類型的b細胞和t細胞。這些細胞對於識別和攻擊外來侵入者（如病毒和細菌）以防止感染是必要的。
Leniolisib, marketed under Joenja in the U.S., received FDA approval for APDS in adult and pediatric patients 12 years and older in March 2023.
Leniolisib在美國以Joenja的名義上市，於2023年3月獲得FDA對12歲及以上成人和兒童患者APDS的批准。
Pharming plans to include data from this 4-11-year-old trial in regulatory filings worldwide for the approval of leniolisib for pediatric patients with APDS, beginning in 2025.
Pharming計劃將這項針對4至11歲兒童的試驗數據納入全球監管提交，以申請leniolisib用於患有APDS的兒童患者的批准，預計於2025年開始。
The study enrolled 21 children with APDS ages 4 to 11 years.
該研究招募了21名年齡在4至11歲的APDS兒童。
The study's primary efficacy endpoints are a reduction in index lymph node size and an increased proportion of naïve B cells out of total B cells from baseline at 12 weeks.
該研究的主要療效終點是減少指數淋巴結的大小和基線時在12周時naïve b細胞佔總b細胞的比例增加。
Secondary endpoints include assessing the ability of leniolisib to modify health-related quality of life.
次要終點包括評估leniolisib對健康相關生活質量的影響。
All 21 patients enrolled completed the 12-week treatment period.
所有21名患者均完成了12周的治療週期。
Lymphoproliferation improved as measured by a mean reduction in index lesion size, and immunophenotype correction was demonstrated by an increase in the percent of naïve B cells.
淋巴增生通過平均減少指標病竈大小得到了改善，免疫表型糾正通過幼稚b細胞的百分比增加得到了證明。
The improvement in lymphoproliferation and immunophenotype correction was seen across the four dose levels being investigated and was consistent with the improvements previously reported in adolescent and adult patients.
在研究的四個劑量水平上，淋巴增生和免疫表型糾正的改善是一致的，與以前在青少年和成人患者中報告的改善相符。
All treatment-emergent adverse events were reported to be mild to moderate in nature. There were no drug-related serious adverse events, and all patients completed the 12-week treatment period.
所有治療出現的不良事件均被報告爲輕度至中度。沒有與藥物相關的嚴重不良事件，所有患者完成了爲期12周的治療期。
In October, Pharming started a Phase 2 proof-of-concept trial evaluating leniolisib in primary immunodeficiencies with immune dysregulation linked to altered PI3Kẟ signaling in lymphocytes.
在十月份，Pharming開始了一項評估leniolisib在與淋巴細胞的PI3Kẟ信號改變相關的免疫功能失調的原發免疫缺陷中的概念驗證二期試驗。
Price Action: PHAR stock is up 11.6% at $9.75 at last check Wednesday.
價格動態：PHAR股票在週三最後檢查時上漲了11.6%，報9.75美元。
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譯文內容由第三人軟體翻譯。
