Lexeo Therapeutics Inc. has announced that its IND for LX-2020 has been cleared by the FDA. LX-2020 is an AAVrh10-based gene therapy candidate designed to intravenously deliver a functional PKP2 gene to cardiac muscle for the treatment of arrhythmogenic cardiomyopathy (ACM) caused by variants in PKP2 (PKP2-ACM).
The U.S. FDA unveiled a proposal to once again reshuffle its operations, this time with a greater degree of emphasis on the function of the Office of Regulatory Affairs (ORA). Tim Philips, a consultant with Gardner Law and a former member of the FDA, told BioWorld that while these changes will likely yield some useful efficiencies, they might also dilute some of the more useful interaction between industry and FDA, a loss that may be keenly felt when it comes to matters such as FDA inspections.
About two months ahead of the planned PDUFA date, the U.S. FDA has granted expanded approval of PD-1 antibody Jemperli (dostarlimab) from partners Anaptysbio Inc. and GSK plc for use in combination with standard-of-care chemotherapy. GSK said the drug would be the only frontline immuno-oncology treatment for endometrial cancer available in combination with carboplatin and paclitaxel. The supporting supplemental BLA for the expanded indication previously received priority review.
Citius Pharmaceuticals Inc.’s retooled version of withdrawn cancer drug Ontak (denileukin diftitox) is not quite ready for prime time, according to the U.S. FDA, which issued a complete response letter (CRL) for the interleukin-2-diphtheria toxin fusion protein known as Lymphir. The agency is asking for Citius to incorporate enhanced product testing and additional controls but did not raise any concerns regarding safety and efficacy, the company said.
During the week of July 24, 2023, the FDA published several notifications of potentially elevated risks associated with medical devices, including a recall of a delivery sheath for the Amplatzer device by Abbott Laboratories. The agency also announced that Abiomed Inc. will provide a correction for the instructions for use (IFUs) for the Impella because of an issue seen when implanting the left ventricular assist device in patients with transcatheter aortic valve replacement (TAVR) devices.
The U.S. FDA has issued a draft guidance for devices intended to address opioid use disorder (OUD), a problem with a massive public health footprint that has defied the efforts of public health programs. The draft guidance highlights some of the difficulties in executing pivotal studies for these devices, but the FDA’s July 27 press omits any mention of a 2018 innovation challenge for this category of devices, a programmatic effort that seems to have yielded little in the way of tangible results.
Krystal Biotech Inc. has received FDA acceptance of its IND application for lead oncology drug candidate KB-707 for the treatment of locally advanced or metastatic solid tumor malignancies. KB-707 is a modified herpes simplex virus 1 (HSV-1) vector designed to deliver genes encoding both human IL-12 and IL-2 to the tumor microenvironment and promote systemic immune-mediated tumor clearance.
Cessation Therapeutics Inc. has obtained IND clearance from the FDA to initiate a first-in-human trial of CSX-1004 for the prevention of fentanyl overdose. The trial in healthy volunteers is expected to begin next month.
Nearly five years after submitting its first NDA seeking U.S. approval for Vanflyta (quizartinib) for treating a subset of patients with acute myeloid leukemia (AML), Daiichi Sankyo Co. Ltd. finally cleared the last hurdle. The FDA on July 20 approved the FLT3 inhibitor for use in combination with cytarabine and anthracycline induction and cytarabine consolidation, and as maintenance monotherapy following consolidation chemotherapy, for the treatment of adult patients with newly diagnosed disease that is FLT3 ITD-positive.
Epibone Inc. has received IND clearance from the FDA to begin testing its engineered allogenic osteochondral graft in humans. Clinical trials are set to open patient recruitment as soon as early next year.
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