Approvals
Dupixent, which was rejected by the FDA for chronic spontaneous urticaria in October 2023, is now approved as the first new targeted therapy for the indication in more than 10 years.
Losing the FDA’s senior negotiators would slow the renewal of the user fee programs “considerably,” according to policy and regulatory expert Steven Grossman.
Industry representatives will still be allowed at these meetings, but they will no longer have a spot on the advisory committee.
Lined up for the FDA in the coming weeks are a cell-based gene therapy for a rare skin disease and two product expansions for Regeneron, one with partner Sanofi.
In the Phase III MITIGATE trial, Uplizna cut IgG4-related disease flares by 87% versus placebo.
Unlike other hemophilia therapies, Qfitlia is indicated for both hemophilia A and B, and can be given in patients regardless of the presence of neutralizing antibodies against clotting factor VIII or IX.
Neffy 1 mg is the “first significant innovation” for epinephrine delivery in small children aged 4 years and up in over 35 years, according to ARS Pharmaceuticals.
TNKase is the first stroke drug to win FDA approval in nearly three decades.
Ctexli’s approval further entrenches Mirum as a leader in rare liver diseases, alongside its cornerstone product Livmarli and upcoming drug volixibat.
The approvals come as Robert F. Kennedy Jr.—who has been critical of vaccines—takes leadership of the Department of Health and Human Services.
PRESS RELEASES