by Madaline Spencer | Jan 5, 2026
The U.S. Food and Drug Administration (FDA) has approved Myqorzo (aficamten) for the treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM). oHCM is a rare genetic heart condition characterized by excessive growth of heart muscle cells....
by Madaline Spencer | Jan 5, 2026
Binod Dhakal, MD, Associate Professor of Medicine at Medical College of Wisconsin, discusses results from the CARTITUDE-4 clinical trial in patients with multiple myeloma (MM). MM is a form of cancer that occurs due to abnormal and uncontrolled growth of...
by Madaline Spencer | Jan 2, 2026
Jean-Marie Michot, MD, Institut Gustave Roussy, France, discusses results from Part 1A (dose escalation) of the OLYMPIA-3 study of odronextamab plus chemotherapy in patients with diffuse large B-cell lymphoma (DLBCL). DLBCL is a form of non-Hodgkin...
by Peter Ciszewski and Madaline Spencer | Jan 1, 2026
Below is the list of important regulatory dates for all orphan drugs for 2026. Prescription Drug User Fee Act (PDUFA) dates refer to deadlines for the FDA to review new drugs. PDUFA DateOrphan DrugIndicationCompanyStatus 1.5.2026Cablivi (caplacizumab)Acquired...
by Madaline Spencer | Dec 30, 2025
Paolo Ghia, MD, PhD, Professor of Medical Oncology at the Università Vita-Salute San Raffaele in Milan, Italy, discusses data comparing ibrutinib plus venetoclax combination therapy with chlorambucil + obinutuzumab combination therapy in patients with chronic...
