Ultragenyx Wins FDA Approval for FAYUVI, First Sanfilippo Type A Treatment

Agios Pharmaceuticals is leaning heavily on its lead therapy mitapivat for near- to medium-term growth after pipeline setbacks and rising competition in sickle cell disease. Pyrukynd and Aqvesme together generated $44.7 million in worldwide net revenues in the second quarter of 2026, up 259.3% year over year, following the January 2026 U.S. launch of Aqvesme for adults with alpha- or beta-thalassemia. Aqvesme recorded 442 cumulative prescriptions from REMS-certified U.S. physicians as of June 30, 2026, up from 242 at the end of the first quarter, while the European Commission approved Pyrukynd for thalassemia in May 2026. Agios has submitted a supplemental new drug application for mitapivat in sickle cell disease under the accelerated approval pathway, and the FDA has granted priority review with a final decision expected by Nov. 1, 2026. The company discontinued its next-generation PK activator tebapivat in mid-2026 after setbacks in lower-risk myelodysplastic syndromes and sickle cell disease, and it faces competition from Novo Nordisk's etavopivat, which Novo Nordisk plans to file for approval in the fourth quarter of 2026, as well as from Bristol Myers Squibb's Reblozyl and Novartis' Adakveo.

United Therapeutics shares ended the last trading session 12.5% higher at $541.89 after a Delaware court ruled that Liquidia's Yutrepia infringes two claims of UTHR's patent covering inhaled treprostinil for PH-ILD. The ruling could lead to restrictions on Yutrepia's PH-ILD use, potentially reducing competition for United Therapeutics' Tyvaso and Tyvaso DPI products, and the company could further benefit from potential monetary damages related to Liquidia's past infringement, although the final remedies remain pending. The drugmaker is expected to post quarterly earnings of $6.38 per share in its upcoming report, a year-over-year change of -10.9%, on revenues of $792.64 million, down 0.9% from the year-ago quarter. The consensus EPS estimate for the quarter has been revised 1.4% lower over the last 30 days, and the stock currently carries a Zacks Rank #3 (Hold).