RARE

BASEL, Switzerland, Nov. 06, 2026 (GLOBE NEWSWIRE) -- Ultragenyx (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialisation of novel therapies for rare and ultra-rare genetic diseases, today announced that the UK Medicines and Healthcare products Regulatory Agency (MHRA) accepted on 28th October 2026 the Company's Marketing Authorisation Application (MAA) for rebisufligene etisparvovec (also known as rebi-vec or UX111), an investigational gene therapy for the treatment of mucopolysaccharidosis Type IIIA (MPS IIIA), also known as Sanfilippo syndrome Type A.

Ultragenyx Pharmaceutical remains a cautious 'Hold,' as profitability targets are driven by deep R&D cuts, not robust revenue growth. RARE's Q3 revenue rose 8% Y/Y to $173M, but business remains highly concentrated in CRYSVITA, with new launches contributing minimally. Pipeline setbacks and terminated programs have forced a pivot from high-risk optionality to a leaner rare-disease commercial model.

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) stock is trading lower on Wednesday after the company initiated substantial expense reductions to align expenses with revenue and achieve profitability by 2027.

Ultragenyx Pharmaceutical Inc. ( RARE ) Q3 2026 Earnings Call November 3, 2026 5:00 PM EST Company Participants Joshua Higa - Director of Investor Relations & Corporate Communications Emil Kakkis - Founder, President, CEO & Director Howard Horn - Executive VP of Corporate Strategy & CFO Erik Harris - Executive VP & Chief Commercial Officer Eric Crombez - Chief Medical Officer & Executive VP Conference Call Participants Maurice Raycroft - Jefferies LLC, Research Division Kristen Kluska - Cantor Fitzgerald & Co., Research Division Joon Lee Jaena Han - TD Cowen, Research Division Tazeen Ahmad - BofA Securities, Research Division Maxwell Skor - Morgan Stanley, Research Division Lydia Erdman - Goldman Sachs Group, Inc., Research Division Jack Allen - Robert W. Baird & Co. Incorporated, Research Division Luca Issi - RBC Capital Markets, Research Division Benjamin Burnett - Wells Fargo Securities, LLC, Research Division Lachlan Hanbury-Brown - William Blair & Company L.L.C.

Third quarter total revenue of $173 million, CRYSVITA® revenue of $109 million and DOJOLVI® revenue of $30 million GENGLYCOS™ and FAYUVI™ BLAs approved by FDA in the third quarter Two Priority Review Vouchers (PRVs) under agreement for combined sales price of $413 million Substantial reduction of expenses and headcount underway, expected to drive at least a 50% reduction in 2027 R&D expenses vs 2025 NOVATO, Calif., Nov. 03, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today reported its financial results for the quarter ended September 30, 2026, announced substantial expense reductions, and reaffirmed its guidance for 2026 revenue and 2027 profitability.

NOVATO, Calif., Oct. 27, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that it will host a conference call at 5:00 p.m.

Ultragenyx (RARE) doesn't possess the right combination of the two key ingredients for a likely earnings beat in its upcoming report. Get prepared with the key expectations.

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