Rare diseases
BridgeBio beat investor expectations with 1,028 unique prescriptions for ATTR-CM therapy Attruby, setting the company up to beat a 2025 sales consensus of $86 million.
The partnership splits the rights to Stoke’s epilepsy antisense oligonucleotide, with up to $385 million in potential payments due to Stoke.
Ono picked up Romvimza—previously known by its active ingredient vimseltinib—from its $2.4 billion acquisition of Deciphera Pharmaceuticals in April 2024.
With its recent data drop for an oligonucleotide candidate, Dyne Therapeutics signals it may become a frontrunner in this disease space alongside Avidity Biosciences, Lupin and AMO Pharma.
Evrysdi is the first, and so far only, noninvasive disease-modifying treatment for spinal muscular atrophy.
The Boston-area company’s previous raises were also aimed at getting its investigational antibody treatment for inclusion body myositis through clinical trials. Now, Abcuro is eyeing a regulatory submission and potential launch.
Vertex expects to make the newly approved non-opioid pain medicine Journavx available by the end of February.
AceLink is advancing a small-molecule treatment option for Fabry disease that could provide a more convenient alternative to the current enzyme replacement therapy standard.
Novo Nordisk’s bispecific antibody Mim8 prevented bleeding events and caused no adverse safety events in the Phase III FRONTIER3 trial. The company plans to file for approval this year, hoping to compete in the hemophilia A space with Roche’s blockbuster Hemlibra.
Despite the discontinuations, CEO Pascal Soriot said AstraZeneca’s December 2020 acquisition of Alexion was a “fantastic” deal for the pharma.
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