Acid Sphingomyelinase Deficiency
Jul 05, 2022
AstraZeneca’s Imfinzi Shows Positive Results; Novartis Announces Results of Tislelizumab; FDA Grants Orphan Drug Designation to Evorpacept; EU Approves Sanofi’s Xenpozyme; Bayer’s Kerendia Approves in China; FDA Breakthrough Therapy Designation to Talquetamab; FDA Puts Clinical Hold on Sanofi’s Tolebrutinib; FDA Rejects Spero’s Tebipenem
FDA Grants Orphan Drug Designation to Evorpacept for AML ALX Oncology Holdings announced that the U.S. Food and Drug Administration had granted orphan drug designation to Evorpacept, a next-generation CD47 blocker, for treating patients with acute myeloid leukemia (AML). Acute Myeloid Leukemia (AML) is an agg...
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Mar 29, 2022
Sanofi’s Rare Disease Drug Xenpozyme’ Approval; FDA Approves Novartis’ Pluvicto; AstraZeneca’s Imfinzi Trial Result; FDA’s Green Signal to Argenx’ Vyvgart; ViiV Healthcare and J&J’s HIV Treatment Cabenuva; UCB’s FINTEPLA Approval; Cullinan’s Update for CLN-081; MEI Pharma & Kyowa’s Zandelisib Approval
Sanofi’s Rare Disease Drug Xenpozyme Wins World’s First Approval in Japan Sanofi’s Xenpozyme, also known as olipudase alfa, the world's first therapy for the rare disease Niemann-Pick type A/B and B, has been approved for the treatment of children and adults with non-CNS manifestations of Acid Sphingomyelinase D...
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