Description of Ultragenyx Pharmaceutical Inc's Business Segments

Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company dedicated to developing innovative therapies for rare and ultra-rare genetic diseases. Their commitment is reflected in a diverse product portfolio divided into several segments, with a focus on addressing unmet medical needs in these specialized fields. Below is an extensive overview of Ultragenyxs segments, products, and services.

Segments

1. Metabolic Disorders
Ultragenyx’s metabolic disorders segment targets rare inherited metabolic diseases. This includes conditions such as:
- Gaucher Disease: A genetic disorder resulting from the deficiency of the enzyme glucocerebrosidase, leading to harmful substance accumulation.
- Fabry Disease: Caused by the deficiency of the enzyme alpha-galactosidase A, leading to pain, kidney dysfunction, and other systemic issues.
- Mucopolysaccharidosis Types: These are a group of lysosomal storage disorders resulting from the bodys inability to break down glycosaminoglycans.
- Ultragenyx’s flagship product in this segment is Mepsevii (velmanase alfa), an enzyme replacement therapy approved by the FDA for the treatment of Mucopolysaccharidosis VII (MPS VII), highlighting the company’s focus on therapies for ultra-rare conditions.

2. Skeletal Disorders
The skeletal disorders segment focuses on genetic conditions affecting bone development and metabolism. Significant conditions include:
- X-Linked Hypophosphatemia (XLH): A genetic disorder characterized by renal phosphate wasting, leading to skeletal deformities.
- Osteogenesis Imperfecta: A group of genetic disorders causing fragile bones due to connective tissue deficiencies.
- Achondroplasia: The most common form of skeletal dysplasia, which leads to disproportionate short stature.
- Crysvita (burosumab-twza) is the leading product in this segment. It is a recombinant human monoclonal antibody designed to target and inhibit the activity of fibroblast growth factor 23 (FGF23), improving phosphate metabolism in patients with X-linked hypophosphatemia.

3. Neurological Disorders
Ultragenyx also focuses on rare neurological diseases that pose significant challenges to patients and clinicians. This segment includes:
- Niemann-Pick Disease Type C: A genetic lipid storage disorder that causes neurological decline.
- CDKL5 Deficiency Disorder: A rare genetic condition leading to severe epilepsy and developmental delays.
- Wilson’s Disease: A genetic disorder leading to copper accumulation in the body, often resulting in severe liver and neurological issues.
- Dojolvi (triheptanoin) is a key product in this category, being the first FDA-approved treatment for long-chain fatty acid oxidation disorders, which are critical metabolic diseases affecting energy production in the body.

Products

1. Crysvita
- Indication: Used for the treatment of X-linked hypophosphatemia.
- Mechanism of Action: It works by inhibiting FGF23, a hormone that regulates phosphate and vitamin D metabolism, thereby increasing serum phosphate levels and improving skeletal health.

2. Mepsevii
- Indication: Approved for the treatment of Mucopolysaccharidosis VII (MPS VII).
- Mechanism of Action: It provides the missing enzyme, beta-glucuronidase, allowing for the degradation of glycosaminoglycans that accumulate in the body due to the deficiency.

3. Dojolvi
- Indication: For the treatment of long-chain fatty acid oxidation disorders.
- Mechanism of Action: As a structural fat, it compensates for the inability to utilize long-chain fatty acids, providing an efficient energy source.

Services

Ultragenyx Pharmaceutical Inc. supports its patients, healthcare providers, and the broader community through various complementary services, such as:

1. Patient Support Services
- These include comprehensive assistance for patients enrolled in their therapies. Services offered involve:
- Reimbursement support: Helping patients navigate insurance processes to access medications.
- Medication delivery: Coordinating the timely delivery of prescribed therapies to patients homes.
- Disease education: Providing resources that inform patients and caregivers about their conditions and treatment plans.

2. Medical Education
- Ultragenyx offers educational programs aimed at healthcare professionals. These programs cover the complexities of rare genetic diseases, emphasizing recent advances in treatment options and the importance of recognizing these conditions in clinical practice.

3. Clinical Trial Support
- The company is involved in designing and supporting clinical trials to evaluate new therapies for rare diseases. They provide expertise in trial management and regulatory compliance, and they offer a clinical trial matching service to assist patients in finding suitable clinical studies for their conditions.

Through its focused segments, innovative products, and supportive services, Ultragenyx Pharmaceutical Inc. continues to make significant strides in the treatment of rare and ultra-rare genetic diseases, striving to improve the lives of patients facing these challenging conditions.

Download as CSV

Commercial Data Licensing & OEM

Annual commercial subscriptions for corporate use, internal BI systems, management reporting, AI integration, and client-facing platforms

API & Data Feeds

Programmatic access to normalized fundamentals & ratios.

Composition of Ultragenyx Pharmaceutical Inc Revenues by Segments


Product Revenue	65.44 %	of total Revenue
Product Revenue Crysvita	33.82 %	of total Revenue
Product Revenue Dojolvi	13.24 %	of total Revenue
Product Revenue Evkeeza	13.24 %	of total Revenue
Product Revenue Mepsevii	5.15 %	of total Revenue
Royalty revenue	34.56 %	of total Revenue
Royalty revenue Crysvita	34.56 %	of total Revenue
North America	43.38 %	of total Revenue
Latin America	30.88 %	of total Revenue
Europe Middle East and Africa	22.79 %	of total Revenue
Asia-Pacific	2.94 %	of total Revenue
Royalty revenue Kyowa Kirin Collaboration Royalty Revenue in North American Territory	19.85 %	of total Revenue
Non-cash royalty revenue Kyowa Kirin Collaboration Royalty Revenue in North American Territory	8.82 %	of total Revenue
Non-cash royalty revenue Kyowa Kirin Collaboration Royalty Revenue in European Territory	5.88 %	of total Revenue
Collaboration and Royalty Kyowa Kirin Collaboration	68.38 %	of total Revenue
Collaboration and Royalty Kyowa Kirin Collaboration Royalty Revenue in North American Territory	28.68 %	of total Revenue
Product sales	65.44 %	of total Revenue
Product sales Kyowa Kirin Collaboration	33.82 %	of total Revenue

Q1 three months ended (Mar 31 2026)
Revenues by Business Segments	Revenues (in millions $)	% (of total Revenues)
Product Revenue	89.00	65.44 %
Product Revenue Crysvita	46.00	33.82 %
Product Revenue Dojolvi	18.00	13.24 %
Product Revenue Evkeeza	18.00	13.24 %
Product Revenue Mepsevii	7.00	5.15 %
Royalty revenue	47.00	34.56 %
Royalty revenue Crysvita	47.00	34.56 %
North America	59.00	43.38 %
Latin America	42.00	30.88 %
Europe Middle East and Africa	31.00	22.79 %
Asia-Pacific	4.00	2.94 %
Royalty revenue Kyowa Kirin Collaboration Royalty Revenue in North American Territory	27.00	19.85 %
Non-cash royalty revenue Kyowa Kirin Collaboration Royalty Revenue in North American Territory	12.00	8.82 %
Non-cash royalty revenue Kyowa Kirin Collaboration Royalty Revenue in European Territory	8.00	5.88 %
Collaboration and Royalty Kyowa Kirin Collaboration	93.00	68.38 %
Collaboration and Royalty Kyowa Kirin Collaboration Royalty Revenue in North American Territory	39.00	28.68 %
Product sales	89.00	65.44 %
Product sales Kyowa Kirin Collaboration	46.00	33.82 %
Total	136.00	100 %

Q1 three months ended (Mar 31 2026)
Revenue Growth rates by Segment	Y/Y Revenue %	Q/Q Revenue %
Product Revenue	-2.2 %	-
Product Revenue Crysvita	-16.36 %	-
Product Revenue Dojolvi	5.88 %	-
Product Revenue Evkeeza	63.64 %	-
Product Revenue Mepsevii	-12.5 %	-
Royalty revenue	-2.08 %	-
Royalty revenue Crysvita	-2.08 %	-
North America	1.72 %	-
Latin America	-23.64 %	-
Europe Middle East and Africa	34.78 %	-
Asia-Pacific	33.33 %	-
Royalty revenue Kyowa Kirin Collaboration Royalty Revenue in North American Territory	-6.9 %	-
Non-cash royalty revenue Kyowa Kirin Collaboration Royalty Revenue in North American Territory	0 %	-
Non-cash royalty revenue Kyowa Kirin Collaboration Royalty Revenue in European Territory	14.29 %	-
Collaboration and Royalty Kyowa Kirin Collaboration	-9.71 %	-
Collaboration and Royalty Kyowa Kirin Collaboration Royalty Revenue in North American Territory	-4.88 %	-
Product sales	-2.2 %	-
Product sales Kyowa Kirin Collaboration	-16.36 %	-
Total	-2.36 %	-

Q1 three months ended (Mar 31 2026)
Income by Business Segments	Income (in millions $)	% (Profit Margin)
Total	-185.00	-

Q1 three months ended (Mar 31 2026)
Income Growth rates by Segment	Y/Y Income %	Q/Q Income %
Total	-	-

Annual Report on Ultragenyx Pharmaceutical Inc Divisions, Sales by Country


Product Revenue	54.83 %	of total Revenue
Product Revenue Crysvita	26.3 %	of total Revenue
Product Revenue Dojolvi	14.26 %	of total Revenue
Product Revenue Evkeeza	8.77 %	of total Revenue
Product Revenue Mepsevii	5.5 %	of total Revenue
Royalty revenue	45.17 %	of total Revenue
Royalty revenue Crysvita	45.17 %	of total Revenue
North America	56.02 %	of total Revenue
Latin America	25.26 %	of total Revenue
Europe Middle East and Africa	16.05 %	of total Revenue
Asia-Pacific	2.67 %	of total Revenue
Non-cash royalty revenue Kyowa Kirin Collaboration Royalty Revenue in European Territory	4.31 %	of total Revenue
Collaboration and Royalty Kyowa Kirin Collaboration	71.47 %	of total Revenue
Product sales	54.83 %	of total Revenue
Product sales Kyowa Kirin Collaboration	26.3 %	of total Revenue
Royalty revenue Kyowa Kirin Collaboration Profit Share Territory	28.53 %	of total Revenue
Non-cash royalty revenue Kyowa Kirin Collaboration Profit Share Territory	12.33 %	of total Revenue
Collaboration and Royalty Kyowa Kirin Collaboration Profit Share Territory	40.86 %	of total Revenue

Twelve months ended 2025
RARE s Annual Revenue by Geography and Business Segments	Sales (in millions $)	% (of total Sales)
Product Revenue	369.00	54.83 %
Product Revenue Crysvita	177.00	26.3 %
Product Revenue Dojolvi	96.00	14.26 %
Product Revenue Evkeeza	59.00	8.77 %
Product Revenue Mepsevii	37.00	5.5 %
Royalty revenue	304.00	45.17 %
Royalty revenue Crysvita	304.00	45.17 %
North America	377.00	56.02 %
Latin America	170.00	25.26 %
Europe Middle East and Africa	108.00	16.05 %
Asia-Pacific	18.00	2.67 %
Non-cash royalty revenue Kyowa Kirin Collaboration Royalty Revenue in European Territory	29.00	4.31 %
Collaboration and Royalty Kyowa Kirin Collaboration	481.00	71.47 %
Product sales	369.00	54.83 %
Product sales Kyowa Kirin Collaboration	177.00	26.3 %
Total	673.00	100 %
Royalty revenue Kyowa Kirin Collaboration Profit Share Territory	192.00	28.53 %
Non-cash royalty revenue Kyowa Kirin Collaboration Profit Share Territory	83.00	12.33 %
Collaboration and Royalty Kyowa Kirin Collaboration Profit Share Territory	275.00	40.86 %

Twelve months ended 2025
RARE s Annual Income by Country and Business Segments	Income (in millions $)	% (Profit Margin)
Total	-575.00	-

Twelve months ended 2025
Annual Revenue and Income Growth by Country and Business Segments	% Y/Y Sales Growth	% Y/Y Income Growth
Product Revenue	29.47 %	-
Product Revenue Crysvita	31.11 %	-
Product Revenue Dojolvi	9.09 %	-
Product Revenue Evkeeza	84.38 %	-
Product Revenue Mepsevii	23.33 %	-
Royalty revenue	10.55 %	-
Royalty revenue Crysvita	10.55 %	-
North America	10.88 %	-
Latin America	29.77 %	-
Europe Middle East and Africa	35 %	-
Asia-Pacific	100 %	-
Non-cash royalty revenue Kyowa Kirin Collaboration Royalty Revenue in European Territory	11.54 %	-
Collaboration and Royalty Kyowa Kirin Collaboration	17.32 %	-
Product sales	29.47 %	-
Product sales Kyowa Kirin Collaboration	31.11 %	-
Total	20.13 %	-
Royalty revenue Kyowa Kirin Collaboration Profit Share Territory	10.34 %	-
Non-cash royalty revenue Kyowa Kirin Collaboration Profit Share Territory	10.67 %	-
Collaboration and Royalty Kyowa Kirin Collaboration Profit Share Territory	10.44 %	-

Help

About us

Products

Data downloads

API

CSIMarket Company, Sector, Industry, Market Analysis, Stock Quotes, Earnings, Economy, News and Research.

Intraday data delayed per exchange requirements. All quotes are in local exchange time. Intraday data delayed 15 minutes for Nasdaq, and other exchanges. Fundamental and financial data for Stocks, Sector, Industry, and Economic Indicators provided by CSIMarket.com

Disclaimer: Information provided by CSIMarket.com is for informational purposes only and does not constitute investment advice, recommendation, or solicitation to buy or sell any security.