Several years after its US approval, Ultragenyx's therapy for long-chain fatty acid oxidation disorders, Dojolvi, has been greenlit in the UK.

MHRA approves triheptanoin to treat adult and paediatric patients with a group of rare inherited disorders.

<img src="https://www.govwire.co.uk/og/cache/news/3057502.png"/>MHRA approves triheptanoin to treat adult and paediatric patients with a group of rare inherited disorders. | Medicines and Healthcare products Regulatory Agency.

<img src="https://www.govwire.co.uk/og/cache/news/3057502.png"/>MHRA approves triheptanoin to treat adult and paediatric patients with a group of rare inherited disorders. | Medicines and Healthcare products Regulatory Agency.

MHRA approves triheptanoin to treat adult and paediatric patients with a group of rare inherited disorders.

Click to view the official SEC document.

Ultragenyx ($RARE): FDA full approval of FAYUVI (UX111) on September 17, 2026 for Sanfilippo type A, a priority review voucher and EMA validation.

Click to view the official SEC document.

Ultragenyx (NASDAQ: RARE) reported second quarter 2026 total revenue of $214 million, up from $167 million a year ago, led by Crysvita revenue of $156 million and Dojolvi revenue of $27 million. Evkeeza contributed $21 million and Mepsevii $10 million.According to Ultragenyx, net loss narrowed to $92 million, or $0.90 per share, versus a $115 million loss, or $1.17 per share, in Q2 2025. Total operating expenses were $289 million, including $34 million of non‑cash stock-based compensation.The company reaffirmed 2026 guidance for total revenue of $730–$760 million, Crysvita revenue of $500–$520 million, and Dojolvi revenue of $100–$110 million, and expects combined R&amp;D and SG&amp;A expenses in 2026 to be flat to down low-single digits versus 2025 and to decrease by at least 15% in 2027, supporting its stated path to profitability in 2027.Ultragenyx highlighted near‑term catalysts including two FDA PDUFA action dates for DTX401 (August 23, 2026) and UX111 (September 19, 2026), and Phase 3 GTX‑102 Aspire data for Angelman syndrome expected in September or October 2026. As of June 30, 2026, cash, cash equivalents, and marketable securities were $436 million, with net cash used in operations of $97 million in the quarter.

Ultragenyx (NASDAQ: RARE) reported Q1 2026 revenue of $136 million with Crysvita $93M, Dojolvi $18M and reaffirmed 2026 guidance of $730–$760M total revenue. The company reaffirmed flat-to-slightly-down 2026 combined R&amp;D and SG&amp;A versus 2025 and a path to profitability in 2027.Key clinical and regulatory milestones include FDA acceptance and PDUFA dates for DTX401 (Aug 23, 2026) and UX111 (Sep 19, 2026), Phase 3 Aspire data for GTX-102 expected H2 2026, and $534M cash and marketable securities as of March 31, 2026.

Ultragenyx (NASDAQ: RARE) reported preliminary unaudited 2025 results and a corporate update at J.P. Morgan Healthcare Conference on January 12, 2026. Preliminary 2025 total revenue was $672M–$674M, roughly +20% vs 2024, with Crysvita revenue $480M–$482M (+17% vs 2024) and Dojolvi revenue $95M–$97M (+9% vs 2024). Cash and investments were ~ $735M at December 31, 2025. The company completed a rolling BLA submission for DTX401 and expects a PDUFA in Q3 2026; UX111 BLA resubmission is planned for early 2026 with up to a 6-month review. Key 2026 catalysts include potential approvals for two gene therapies and pivotal Phase 3 Aspire data for GTX-102 in Angelman syndrome in H2 2026.

Ultragenyx (NASDAQ: RARE) reported Q2 2025 financial results with total revenue of $166 million, representing 13% growth year-over-year. Key revenue drivers include Crysvita at $120 million and Dojolvi at $23 million.The company reaffirmed its 2025 guidance, expecting total revenue between $640-670 million. Despite a net loss of $115 million ($1.17 per share), Ultragenyx maintains its path to profitability target for 2027. Cash position stands at $539 million as of June 30, 2025.Notable pipeline updates include: UX143 Phase 3 data for osteogenesis imperfecta expected by year-end, GTX-102 receiving FDA Breakthrough Therapy Designation for Angelman syndrome with Phase 3 fully enrolled, and planned BLA submission for DTX401 in Q4 2025.

Ultragenyx Pharmaceutical (NASDAQ: RARE) reported Q1 2025 financial results with total revenue of $139 million, up 28% year-over-year. Key revenue drivers include Crysvita at $103 million (25% growth) and Dojolvi at $17 million. The company reported a net loss of $151 million ($1.57 per share), improved from $171 million loss in Q1 2024. Cash position stands at $563 million. Ultragenyx reaffirmed its 2025 guidance, expecting total revenue between $640-670 million. Notable pipeline progress includes UX111's BLA review for Sanfilippo syndrome with PDUFA date of August 18, 2025, and advancement of multiple Phase 3 programs including UX143 for osteogenesis imperfecta and GTX-102 for Angelman syndrome.

Ultragenyx Pharmaceutical (NASDAQ: RARE) reported strong financial results for Q4 and full year 2024, with total revenue of $560 million, representing 29% growth compared to 2023. Key revenue drivers included Crysvita at $410 million (+25% YoY) and Dojolvi at $88 million (+25% YoY).The company provided 2025 guidance projecting total revenue between $640-670 million, with Crysvita revenue of $460-480 million and Dojolvi revenue of $90-100 million. Despite revenue growth, Ultragenyx reported a net loss of $569 million ($6.29 per share) for 2024, though improved from $607 million loss in 2023.The company ended 2024 with $745 million in cash and equivalents, with net cash used in operations of $414 million for the year. Multiple clinical milestones are expected in 2025, including a PDUFA decision for Sanfilippo syndrome gene therapy and Phase 3 results in osteogenesis imperfecta.