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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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Products & Revenue

Product Revenue Share

Revenue Breakdown ($652M)

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Crysvita Royalties + Direct (60%)
Other Products (40%)

Segment Breakdown & Key Customers

Product Details

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.

Supply Chain Relationships

Partners

kyowa

Crysvita (burosumab) co-development

Impact: critical

Macro & Market Context

헬스케어Sector News

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)

Upcoming Catalysts

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

Latest News

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AI Analysis

Click "Get AI Analysis" for an AI-powered supply chain analysis of Ultragenyx Pharmaceutical.

Company Info

Headquarters

Novato, California, USA

Founded

2010

Employees

1,200

Sector Overview — 헬스케어

Sector News

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)

Key Themes

  • •CMS 메디케어 어드밴티지 수가 인하 → 보험사 마진 압박
  • •AI 영상진단·병리학 도입 가속 — Tempus, Veracyte 수혜
  • •GLP-1 보험 적용 확대 논의 → UNH·CVS 비용 증가 리스크

Upcoming Catalysts

  • ◆CMS 메디케어 최종 수가 발표 (2026.Q2)
  • ◆UNH/HUM/CVS Q1 실적 (2026.04)