Samsung Bioepis Co. Ltd., of Incheon, South Korea, gained U.S. FDA approval of Epysqli (eculizumab-aagh) as the second biosimilar product to Alexion Pharmaceuticals Inc.’s Soliris (eculizumab) to treat two rare diseases. The regulatory clearance July 22 grants use of Epysqli to treat paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome – two rare hematologic- and kidney-related disorders known to affect about 50,000 and 5,000 patients in the U.S., respectively.
Orum Therapeutics Inc. struck a potential $945 million (₩1.3 trillion) deal with Vertex Pharmaceuticals Inc. to discover novel degrader antibody conjugates (DAC) as targeted conditioning agents for use with gene editing, including Vertex’s gene therapy, Casgevy (exagamglogene autotemcel).
Takeda Pharmaceutical Co. Ltd. is progressing mezagitamab to phase III trials after the CD38 monoclonal antibody showed rapid and sustained increases in platelet counts in patients with persistent or chronic primary immune thrombocytopenia (ITP) in a phase IIb trial.
Takeda Pharmaceutical Co. Ltd. has signed an option agreement to in-license Ascentage Pharma Group Inc.’s olverembatinib, an oral third-generation BCR-ABL tyrosine kinase inhibitor (TKI). If exercised, the option would allow Takeda to license exclusive global rights to develop and commercialize olverembatinib in all territories outside of mainland China, Hong Kong, Macau, Taiwan and Russia.
From glaucoma to Stargardt disease, age-related macular degeneration (AMD) to retinitis pigmentosa, or a corneal transplant to Bietti’s crystalline dystrophy, the 27th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) is working to bring some light to patients with age and congenital diseases that affect vision. From May 7-11, 2024, thousands of scientists are gathering in Baltimore to show their advances against the challenges of delivering genes and cells to the correct place, avoiding immunogenicity and improving diseases.
Amidst a slew of end-of-week, positive EMA Committee for Medicinal Products for Human Use (CHMP) opinions is Takeda Pharmaceutical Co. Ltd.’s Fruzaqla (fruquintinib). The selective inhibitor of vascular endothelial growth factor receptors-1, -2 and -3 is for adults with previously treated metastatic colorectal cancer.
The EMA’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending approval of Pfizer Inc.’s Emblaveo (aztreonam-avibactam), an antibiotic combination that would offer a new option to patients with serious bacterial infections caused by multidrug-resistant gram-negative bacteria.
Takeda Pharmaceutical Co. Ltd. and Protagonist Therapeutics Inc. have inked a global development and commercialization deal worth up to $1.7 billion for Protagonist’s rusfertide for treatment of polycythemia vera (PV), a rare and chronic blood disorder affecting bone marrow.
China’s National Medical Products Administration (NMPA) has accepted for review Hutchmed’s NDA for sovleplenib (HMPL-523) for treatment of primary immune thrombocytopenia.
Spirits were high at the 2023 annual meeting of the American Society of Hematology (ASH), buoyed by the U.S. FDA approval of the first two gene therapies for sickle cell disease (SCD) the day before the conference kicked off in San Diego. The addition of gene therapy to the therapeutic arsenal for SCD is “phenomenal,” Adetola Kassim, director of the Adult Sickle Cell Disease Program and professor of medicine at the Vanderbilt-Ingram Cancer Center, told BioWorld. Nevertheless, at a Saturday, Dec. 9, session titled, “Improving Outcomes for Individuals with Sickle Cell Disease: Are We Moving the Needle?,” which Kassim chaired, the answer remained “maybe.”
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