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Rare diseases are almost certain to remain one of the biggest areas for novel drug development in 2024, but the orphan drug pipeline will be making space for some novel therapies for common conditions
The month of September started out quietly in terms of approvals, but submission activity continued at a smart pace. Rare diseases are prominent among the recent applications, which include a first-li
FibroGen, Inc. ’s monoclonal antibody candidate pamrevlumab has disappointed in the Phase III ZEPHYRUS-1 trial in idiopathic pulmonary fibrosis (IPF), rendering the drug’s future prospects even more u
The US FDA’s Center for Drug Evaluation and Research cleared two new molecular entities in the last days of September, both for rare diseases – intrahepatic cholangiocarcinoma for Taiho Pharmaceutica