The scoop:In a span of three months, Chiesi gained FDA approval for two of its enzyme replacement therapies. The second came in May when the U.S. regulator signed off on Fabry disease drug Elfabrio. But in February, Lamzede wasapprovedas the first enzyme replacement therapy to...
. These sites also provide updates on cellular and gene therapy products (https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products), as well as vaccines and other products (https://www.fda.gov/vaccines-blood-biologics/development-...
Omaveloxolone是由Reata Pharmaceuticals开发的first-in-class口服药物,用于每天一次治疗16岁及以上的弗里德赖希共济失调(Friedreich’s ataxia)。Friedreich’s ataxia是由编码线粒体蛋白frataxin的基因突变引起的,功能性frataxin的丧失会破坏铁硫簇的生物合成,诱导线粒体功能障碍并增加对氧化应激的敏感性。该疾病会对脊髓...
The FDA has approved lisocabtagene maraleucel as a second-line treatment for patients with relapsed or refractory large B-cell lymphoma. The FDA has granted lisocabtagene maraleucel (Breyanzi; liso-cel) approval as a second-line treatment for patients with relapsed or refractory large B-...
SGT-212 is a recombinant AAV-based gene replacement therapy for Friedreich’s ataxia (FA) designed to deliver full-length human frataxin (FXN) via a dual route of administration: intradentate nucleus (IDN) infusion, usin...
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[1]https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-bosutinib-pediatric-...
In November 2023, the FDA approved repotrectinib for adult patients with locally advanced or metastatic ROS1-positive NSCLC, also based on data from TRIDENT-1. “We look forward to working closely with the FDA on the review of our application for [repotrectinib] for this tumor-agnos...
Bristol Myers Squibb announced that the US Food and Drug Administration (FDA) has approved Breyanzi (lisocabtagene maraleucel; liso-cel), a CD19-directed chimeric antigen receptor (CAR) T cell therapy for the treatment of adult patients with relapsed or refractory (R/R) large B-cell lymphom...