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Ultragenyx Pharmaceutical Inc.: Business Overview, Financials & Competitive Analysis

Ultragenyx Pharmaceutical Inc. scores 6/10 on the AskCyborg Cyborg Score (Mixed). Ultragenyx is a binary 2026 story with approvals and clean Angelman data determining whether it rerates or raises capital from weakness. Ultragenyx positions itself as a diversified rare disease company with four approved products generating approximately $670 million in annual revenue, but recent Phase 3 failure of setrusumab highlights.

What is Ultragenyx Pharmaceutical Inc.'s industry? Ultragenyx Pharmaceutical Inc. (ultragenyx.com) operates in Biopharmaceutical - Rare and Ultra-Rare Genetic Disease Therapeutics. AskCyborg classifies Ultragenyx Pharmaceutical Inc. under Biopharmaceutical - Rare and Ultra-Rare Genetic Disease Therapeutics in its company registry.

Ultragenyx is a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies a...

6/ 10Mixed

Cyborg Score thesis

Ultragenyx is a binary 2026 story with approvals and clean Angelman data determining whether it rerates or raises capital from...

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Industry Biopharmaceutical - Rare and Ultra-Rare Genetic Disease Therapeutics
3 named competitors
3 recent developments
11 analyst dimensions
Synthesized from analyst-debate stress-testing across 11 dimensions and hundreds of data points. Methodology →
Cyborg Score recalibrated: November 2, 2026
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Ultragenyx Pharmaceutical Inc. OverviewStrategic ProfileCompetitive LandscapeIndustry ContextKey facts
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Ultragenyx Pharmaceutical Inc. Overview

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Ultragenyx is a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need for which there are typically no approved therapies treating the underlying disease.

Strategic Profile

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Ultragenyx's strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency. The company's advancing late-stage pipeline, successful product launches, and gene therapy innovation position it for diversified growth and improved profitability.

Competitive Landscape

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Ultragenyx is covered by 34 analysts, with 18 submitting estimates used in coverage reports. The company competes in the rare disease therapeutic space with other biotech firms developing treatments for genetic metabolic disorders, lysosomal storage diseases, and neurological conditions. Heavy reliance on a limited product portfolio and potential biosimilar competition threaten long-term revenue growth and margin expansion.

Industry Context

Ultragenyx Pharmaceutical Inc. operates in Biopharmaceutical - Rare and Ultra-Rare Genetic Disease Therapeutics.

Key facts

Founded: 2010 · Headquarters: Novato, CA, USA · Revenue: $670M · Market cap: $2.27B

What the AI analysts debated

AI-generated and not independently verified: selected findings from the multi-agent debate on Ultragenyx Pharmaceutical Inc..

  • February 2026: FDA acceptance and priority review of DTX401 AAV gene therapy BLA for Glycogen Storage Disease Type Ia
  • Company faces pivotal 2026 defined by two gene therapy PDUFA decisions (UX111 for Sanfilippo A and DTX401 for GSDIa in Q3 2026) and Phase 3 data for GTX-102 in Angelman syndrome in H2 2026
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Recent developments

Material events tracked by the AskCyborg corpus.

  • February 2026FDA Acceptance and Priority Review of DTX401 (AAV gene therapy) BLA for Glycogen Storage Disease Type Ia
  • August 2022Completion of GeneTx Biotherapeutics acquisition for $75 million upfront plus up to $115 million in milestone payments
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Named competitors

Pulled from the analyst-debate framework. Click any competitor to see their Cyborg Score.

Pompe Disease/Lysosomal Storage Disease TherapiesGene therapies and enzyme replacement therapies for rare genetic diseases→Gene Therapy PlatformsAAV-based gene therapies for rare genetic diseases→Rare Disease TherapeuticsSmall molecule and enzyme therapies for rare metabolic disorders→

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Industry context

Ultragenyx Pharmaceutical Inc. operates in Biopharmaceutical - Rare and Ultra-Rare Genetic Disease Therapeutics. See how the rest of the sector scores on the Cyborg framework.

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Frequently asked questions

What does Ultragenyx Pharmaceutical Inc. do?+
Ultragenyx is a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies a...
What is Ultragenyx Pharmaceutical Inc.'s Cyborg Score?+
Ultragenyx Pharmaceutical Inc. has a Cyborg Score of 6/10 (Mixed). The score synthesizes business model, financials, leadership, competitive position, technology, marketing, and ESG dimensions across hundreds of data points.
Who are Ultragenyx Pharmaceutical Inc.'s main competitors?+
Named competitors include Pompe Disease/Lysosomal Storage Disease Therapies, Gene Therapy Platforms, Rare Disease Therapeutics. The full competitive landscape is in the AskCyborg report.
How can I get the full Ultragenyx Pharmaceutical Inc. research report?+
Get the report plus analyst-debate audio at askcyborg.com -- no credit card required for preview access. Pro adds saved-company audio playlists and monitoring.
How current is the Ultragenyx Pharmaceutical Inc. research?+
The Cyborg Score is recalibrated weekly. Recent developments are tracked continuously and surfaced in every report.

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