Approvals
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.
Ono picked up Romvimza—previously known by its active ingredient vimseltinib—from its $2.4 billion acquisition of Deciphera Pharmaceuticals in April 2024.
Merilog’s approval comes as the insulin space has over the past year suffered several setbacks, including strong calls for price caps and, potentially, the rise of the mammoth GLP-1 market.
Evrysdi is the first, and so far only, noninvasive disease-modifying treatment for spinal muscular atrophy.
Leaked data showed that Pfizer’s mevrometostat has strong therapeutic potential in metastatic castration-resistant prostate cancer, while recent readouts also position the pharma as a strong contender in colorectal cancer and bladder cancer.
The approval comes days after Germany’s Merck KGaA confirmed it was in advanced talks to acquire SpringWorks.
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