Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in the United States. The company is headquartered in Novato, California.
| Revenue (TTM) | $717.00M |
| Gross Profit (TTM) | $-149.00M |
| EBITDA | $-465.00M |
| Operating Margin | -35.00% |
| Return on Equity | -608.00% |
| Return on Assets | -24.20% |
| Revenue/Share (TTM) | $7.13 |
| Book Value | $-2.96 |
| Price-to-Book | 19.68 |
| Price-to-Sales (TTM) | 2.00 |
| EV/Revenue | 1.149 |
| EV/EBITDA | -6.46 |
| Quarterly Earnings Growth (YoY) | 0.00% |
| Quarterly Revenue Growth (YoY) | 28.10% |
| Shares Outstanding | $98.59M |
| Float | $80.42M |
| % Insiders | 3.44% |
| % Institutions | 112.80% |
Volatility is currently contracting
Ultragenyx agrees to sell its Fayuvi-linked PRV for $203 million, marking its second voucher sale in one week and adding non-dilutive funding for rare-disease programs.
Ultragenyx's established rare-disease treatments continue to support revenue growth, while two newly approved gene therapies offer additional commercial opportunities.
Agreement marks the Company's second PRV sale within one week, following the previously announced GENGLYCOS™ PRV transaction Agreement marks the Company's second PRV sale within one week, following the previously announced GENGLYCOS™ PRV transaction
RARE enters into an agreement to sell a Rare Pediatric Disease PRV for $210M, adding non-dilutive cash as it advances rare-disease therapies.
Ultragenyx Pharmaceutical (NASDAQ:RARE) shares rose over 3.2% on Wednesday morning after the biopharmaceutical company agreed to sell a Rare Pediatric Disease Priority Review Voucher for $210 million. The voucher was awarded to Ultragenyx after the US Food and Drug Administration approved GENGLYCOS, also known as DTX401, for glycogen storage disease (GSD) type Ia.
NOVATO, Calif., Oct. 07, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that it has entered into a definitive agreement to sell a Rare Pediatric Disease Priority Review Voucher (PRV) for $210 million. Ultragenyx received the PRV upon U.S. Food and Drug Administration (FDA) approval of GENGLYCOS™ (pariglasgene brecaparvovec-opnr), also known as DTX401, the first treatment designed to address the underlying cause of glycogen storage disease type Ia (GSDIa).
NEW YORK, Oct. 06, 2026 (GLOBE NEWSWIRE) -- Pomerantz LLP is investigating claims on behalf of investors of Ultragenyx Pharmaceutical Inc. (“Ultragenyx” or the “Company”) (NASDAQ: RARE). Such investors are advised to contact Danielle Peyton at newaction@pomlaw.com or 646-581-9980, ext. 7980.
RARE's gene therapy for Sanfilippo syndrome wins EMA validation, advancing the treatment toward formal review in Europe.
BASEL, Switzerland, Oct. 02, 2026 (GLOBE NEWSWIRE) -- Today Ultragenyx announced that the European Medicines Agency (EMA) has validated the Company's Marketing Authorisation Application (MAA) for its investigational gene therapy, rebisufligene etisparvovec, for the treatment of mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo syndrome Type A.
NEW YORK, Oct. 1, 2026 /PRNewswire/ -- Pomerantz LLP is investigating claims on behalf of investors of Ultragenyx Pharmaceutical Inc. ("Ultragenyx" or the "Company") (NASDAQ: RARE). Such investors are advised to contact Danielle Peyton at newaction@pomlaw.com or 646-581-9980, ext.
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