Ultragenyx Pharmaceutical

RAREleader

Ultragenyx is a rare-disease biopharmaceutical company with multiple approved therapies and a gene therapy pipeline, focusing on metabolic and bone diseases with no or limited treatment options.

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产品与营收

产品营收占比

营收构成 ($652M)

静态数据（加载实时财务…）

Crysvita Royalties + Direct (60%)

Other Products (40%)

业务板块构成与主要客户

产品详情

Mepsevii (MPS VII)18%

IV enzyme replacement therapy for mucopolysaccharidosis VII — ultra-rare metabolic disorder.

Crysvita (XLH / TIO)45%

Burosumab monoclonal antibody for X-linked hypophosphatemia, the most common inherited form of rickets.

GTX-102 / setrusumab (bone diseases)15%

Gene therapy pipeline targeting osteogenesis imperfecta (brittle bone disease) and related disorders.

UX111 / delandistrogene (UCD, other)22%

AAV gene therapy programs for rare liver-based metabolic disorders and DMD.

供应链关系

合作伙伴

kyowa

Crysvita (burosumab) co-development

影响: critical

宏观与市场背景

헬스케어行业新闻

메디케어 수가 압박 vs AI 진단 효율화 — 선별적 접근 필요

미국 헬스케어 지출 (2026E)

$4.8T

AI 진단 정확도 개선

+23% vs 기존

UNH 의료손해율

87.3%

약가 협상 대상 의약품

20종 (IRA)

近期催化剂

◆CMS 메디케어 최종 수가 발표 (2026.Q2)◆UNH/HUM/CVS Q1 실적 (2026.04)◆IRA 약가 협상 2차 품목 공개

AI 分析

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企业信息

总部

Novato, California, USA

成立时间

2010

员工人数

1,200

官网

ultragenyx.com

行业概况 — 헬스케어

行业新闻