The U.S. FDA gave its go-ahead for Hemgenix (etranacogene dezaparvovec-drlb), Uniqure NV’s one-time gene therapy – the first for the treatment of adults 18 and older living with hemophilia B. Patients have been waiting “maybe beyond two decades” for a new therapy, Uniqure CEO Matthew Kapusta said. Hemgenix emerged from pioneering work by St. Jude Children’s Research Hospital and the University College London.
The companion diagnostic (CDx) has been a mainstay of oncology care for several years, but Richard Pazdur, director of the U.S. FDA’s Oncology Center of Excellence (OCE), said recently in a public forum recently that the notion of a single CDx for an investigational drug has not served patients well. Pazdur said OCE and the FDA’s Center for Devices and Radiological Health are working on a pilot program that would entail the publication of performance standards for the tests used in these trials, a development that would lead to more routine development of lab-developed tests (LDTs) for these studies and thus undercut any incentive to develop a patented in vitro diagnostic for that purpose.
Provention Bio Inc. scored approval from the U.S. FDA of the BLA for Tzield (teplizumab-mzwv), an intravenously given, anti-CD3-directed antibody, as the first and only immunomodulatory treatment to delay the onset of stage 3 type 1 diabetes (T1D) in adult and pediatric patients ages 8 and older with stage 2 T1D.
With a 9-4 vote, the U.S. FDA’s Cardiovascular and Renal Drugs Advisory Committee bucked FDA reviewers who delayed PDUFA dates, issued a complete response letter and two formal dispute resolution requests for Ardelyx Inc.’s tenapanor as a hyperphosphatemia therapy for adults on dialysis with chronic kidney disease (CKD).
The FDA has awarded orphan drug designation to Canbridge Pharmaceuticals Inc.'s CAN-106, a clinical-stage long-acting recombinant humanized monoclonal antibody targeting C5, for the treatment of myasthenia gravis.
Gmax Biopharm LLC has received FDA clearance of its IND filing for GMA-131 injection, an ETa receptor-specific monoclonal antibody, for a phase Ib study in diabetic kidney disease (DKD).
Ardelyx Inc. could have a rocky row to hoe when it makes its case for tenapanor, as a hyperphosphatemia therapy in adults with chronic kidney disease, before the U.S. FDA’s Cardiovascular and Renal Drugs Advisory Committee Nov. 16. The big question facing the adcom is whether the change in baseline serum phosphorous levels achieved by the drug is clinically meaningful. Clearly, FDA reviewers don’t think so, as that question already has resulted in delayed PDUFA dates, a complete response letter and two formal dispute resolution requests.
Asieris Pharmaceuticals Co. Ltd. has received FDA clearance of its IND for APL-1401 for the treatment of moderate to severely active ulcerative colitis.
The U.S. FDA hit a Phoenix-based Abraxis Biosciences LLC facility with a warning letter citing out-of-control aseptic manufacturing processes for Abraxane (paclitaxel), a key chemotherapy drug. The letter, posted Nov. 8, noted that multiple media fill failures occurred last year during simulated aseptic processing operations on the Abraxane filling line.
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