Pharmaceutical Executive Daily: FDA Approves Fayuvi

Welcome to Pharmaceutical Executive Daily, your quick briefing on the top news shaping the pharmaceutical and life sciences industry.

In today's Pharmaceutical Executive Daily, FDA approves Ultragenyx's Fayuvi as the first treatment for Sanfilippo syndrome type A in pediatric patients ages two to five, an M&A roundup covers Lisata Therapeutics' acquisition of Marea Therapeutics and mAbxience's biosimilar collaboration with Sandoz, and Pharmaceutical Executive speaks with Joe Cunningham on why most-favored-nation pricing deals alone aren't enough to lower drug costs.

FDA approves Ultragenyx's Fayuvi (rebisufligene etisparvovec-hopf) as the first treatment for mucopolysaccharidosis type IIIA, or Sanfilippo syndrome type A, in pediatric patients ages two to five. The one-time gene therapy uses an AAV9 vector to deliver a functional SGSH gene, restoring the enzyme needed to break down toxic heparan sulfate buildup in the body and brain. In an open-label study, treated patients maintain or improve cognitive function compared with an untreated historical control cohort, a marked departure from the disease's expected early decline.

Two deals reshape pipelines this week, one through acquisition and one through biosimilar licensing. Lisata Therapeutics acquires Marea Therapeutics in an all-stock transaction paired with a $225 million private placement, combining Marea's genetics-based cardioendocrine platform with fresh capital to advance two first-in-class antibody candidates: Mar001/005 for severe hypertriglyceridemia, in Phase IIb, and Mar002 for acromegaly, in Phase II, with topline data expected for both in the fourth quarter of 2027. Separately, mAbxience partners with Sandoz on an emicizumab biosimilar for hemophilia A, with Sandoz taking exclusive global commercialization rights outside Argentina, Uruguay, and Paraguay in a market estimated at $5.7 billion.

Finally, Pharmaceutical Executive speaks with Joe Cunningham, a former U.S. representative from South Carolina and spokesperson for the Pharmaceutical Reform Alliance, on why executive-order drug pricing deals aren't enough. Cunningham argues that most-favored-nation orders lack durability and, since they apply only to Medicaid, leave out commercial insurance covering roughly 80% of Americans, pointing to survey data showing 90% of voters blame pharmaceutical companies for high healthcare costs and 85% support shorter patent protections to speed generic competition.

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