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企業プロフィール

Pharming Group 株価・ニュース・カタリスト

ティッカー:
PHAR
取引所:
NASDAQ
セクター:
Biotechnology
所在地:
United States

株価パフォーマンス

11.79 USD (+0.60%)

終値

最終更新: 2026年8月17日

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時価総額

株価変動の要因

As of Aug 17, 2026, PHAR closed up 0.60% at $11.79. No new same-company headline was identified in the current news window.

企業概要

Global Rare-Disease Company

Pharming Group is a commercial-stage biopharmaceutical company that develops and commercializes small-molecule and biologic medicines for rare, debilitating, and life-threatening diseases. Headquartered in Leiden, the Netherlands, the company operates in the United States and Europe and markets two rare-disease products across multiple international markets.

RUCONEST for Hereditary Angioedema

RUCONEST is an intravenous on-demand treatment for acute attacks of hereditary angioedema. Pharming positions the product for patients with high disease burden, including those experiencing severe or frequent attacks or inadequate outcomes with other on-demand therapies. The franchise is now primarily focused on the US market.

Joenja for APDS

Joenja, or leniolisib, is a targeted treatment for activated PI3K delta syndrome, a rare primary immunodeficiency caused by dysregulated PI3K delta signaling. The product is approved in the United States and an expanding group of international markets, with eligible populations varying by jurisdiction. Pharming combines commercialization with genetic diagnosis and patient-identification programs intended to find previously unrecognized APDS patients.

Leniolisib Expansion Beyond APDS

Pharming is evaluating leniolisib in two Phase 2 studies involving broader primary immunodeficiencies with immune dysregulation. One study enrolls patients with genetically defined disorders linked to altered PI3K delta signaling, while the other focuses on common variable immunodeficiency. The company expects the two studies to inform a single registrational Phase 3 strategy in the broader CVID population.

Mitochondrial-Disease Pipeline

Napazimone, formerly KL1333, is Pharming's pivotal-stage candidate for primary mitochondrial diseases caused by mitochondrial DNA mutations. The FALCON study is evaluating the therapy in adults with symptoms such as fatigue and muscle weakness and represents the company's principal clinical program outside the leniolisib franchise.

今後のカタリスト

  • FDA PDUFA decision on the Joenja sNDA for certain children aged 4 to 11 years with APDS on October 24, 2026.
  • Topline Phase 2 data for leniolisib in genetically defined primary immunodeficiencies linked to altered PI3K delta signaling in the fourth quarter of 2026.
  • Topline Phase 2 data for leniolisib in CVID with immune dysregulation in the fourth quarter of 2026.
  • Completion of enrollment in the pivotal FALCON study of napazimone in mitochondrial DNA-driven primary mitochondrial diseases during 2026.

最近のニュース

  1. 01

    Pharming announces commercial launch of Joenja (leniolisib) in Japan for adult and pediatric patients with APDS aged 4 and older

  2. 02

    Pharming Group to participate in Canaccord Genuity 46th Annual Growth Conference

  3. 03

    Pharming reports second quarter and first half 2026 financial results and revises year-end guidance; strong Joenja momentum and near-term clinical readouts to support broader therapeutic use

  4. 04

    Pharming Group to report second quarter and first half 2026 financial results and provide business update on July 30

  5. 05

    Pharming announces U.S. FDA acceptance of sNDA resubmission for Joenja® (leniolisib) to treat children aged 4 to 11 years with APDS

類似銘柄

事業成熟度、治療領域、カタリスト傾向が近い同業銘柄です。