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&D and SG&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.

EU approves Eli Lilly's Kisunla, Sanofi expands $35 insulin program, Regeneron's Evkeeza approved for younger HoFH patients, Cure Alzheimer's Fund gets $50M donation

Reported Friday, Sep 26 - 7:08am EDT (TheFly subscription required to read more.)

Reported Monday, Jul 14 - 8:02am EDT (TheFly subscription required to read more.)

Medical Letter on Drugs and Therapeutics 63(1623): 66-68

Medical Letter on Drugs and Therapeutics 63(1623): 66-68

Here is your Rare Diseases update. FDA granted Orphan Drug Designation to CRD-003 for LGMDR9. Japan named JR-446 for MPS IIIB and DYNE-251 for exon 51 DMD as orphan drugs. Evkeeza now approved in the United States for HoFH in children 1 to under 5. GEMMA Biotherapeutics launched Rare Therapeutics for ultra orphan gene therapies. Rocket withdrew its RP-L102 BLA in Fanconi anemia. AAVantgarde secured US ODD and a UK CTA for AAVB-039 in Stargardt. Chiesi and Arbor signed a PH1 gene editing pact globally.

From the United States to Japan and Italy, this concise roundup spotlights pivotal regulatory milestones, portfolio moves, and modality signals shaping the rare pipeline over the past two weeks. 🔬 Key highlights: 🧬 Cure Rare Disease: FDA Orphan Drug Designation for CRD-003 in LGMDR9 🧪 JCR/MEDIPAL: Japan ODD for JR-446 in MPS IIIB, adding to US/EU ODD 🧓 Dyne Therapeutics: Japan ODD for DYNE-251 in DMD exon-51, complements US/EU 🧒 Regeneron: Evkeeza label extended in US to ages 1 to under 5 for HoFH 🚀 GEMMA Biotherapeutics: Launched Rare Therapeutics to advance ultra-orphan gene therapies 🛰️ Rocket Pharmaceuticals: Withdrew FDA BLA for RP-L102 in Fanconi anemia, citing strategy 👁️ AAVantgarde Bio: FDA ODD and UK CTA for AAVB-039 in Stargardt disease 🤝 Chiesi Group & Arbor Biotechnologies: Global deal on ABO-101 for PH1 plus liver gene-editing options 📢 Stay Ahead in Rare diseases Research! ✅ Like, share, and subscribe for future updates on Rare diseases and specific conditions like DMD, HoFH, Stargardt disease, MPS IIIB, PH1, and LGMDR9. ✅ Visit www.lqventures.com for expert healthcare insights and consulting services. ✅ Contact LucidQuest at info@lqventures.com for strategic guidance on Rare diseases innovations and clinical research. 🔔 Turn on notifications so you never miss an update! #RareDiseases #OrphanDrugDesignation #GeneTherapy #DMD #HoFH #Stargardt #MPSIIIB #PH1 #LGMDR9 #Biotech #ClinicalTrials #RegulatoryUpdates #DrugDevelopment #HealthcareInnovation #US #Japan #Italy #LucidQuest

Plus, news about Regeneron's Evkeeza and Cure Alzheimer's: ?? EU approves Eli Lilly’s Kisunla: The Alzheimer’s drug originally earned a negative CHMP opinion in March but was re-evaluated in June. Lilly