Bloomsbury Genetic Therapies Ltd. announced that the FDA granted orphan drug designation for its investigational liver-targeted gene therapy, BGT-OTCD, for the treatment of ornithine transcarbamylase deficiency (OTCD).
The U.S. FDA has approved Daxxify (daxibotulinumtoxinA-lanm) for treating cervical dystonia in adults nearly a week ahead of its Aug. 19 PDUFA date. Approval of the sBLA for the injectable, peptide-formulated neuromodulator went to Revance Therapeutics Inc. The drug’s original approval was for treating glabellar lines, better known as frown lines, came in September 2022. Daxxify is the first neuromodulator stabilized with Revance’s peptide exchange technology. It is created without human serum albumin or animal-based components.
Venostent Inc. has completed $16 million in series A financing to support a U.S. pivotal trial for bioabsorbable wraps designed to improve outcomes for hemodialysis patients suffering from end-stage renal disease (ESRD). This comes after Venostent was granted an FDA investigational device exemption (IDE) to begin its U.S. Selfwrap-assisted arteriovenous fistula study and breakthrough device designation of its technology awarded last year.
Genprex Inc. announced that the FDA has granted orphan drug designation to the company's lead drug candidate, Reqorsa immunogene therapy (quaratusugene ozeplasmid, GPX-001), for the treatment of small cell lung cancer (SCLC). Reqorsa contains a plasmid that expresses the tumor suppressor gene TUSC2, the expression of which is reduced or not present in SCLC.
A complete response letter (CRL) from the U.S. FDA for GC-4419 (avasopasem) is causing Galera Therapeutics Inc. to dramatically change course to survive, with the company laying off about 70% of its workforce. That layoff, which will reach across several company departments, is designed to extend the cash runway into the second quarter of 2024. The adjustment comes after Galera expanded its commercial leadership in May to prep for a potential launch of avasopasem before the end of 2023.
Accuray Inc. offered a classic good news-bad news set-up for investors on Wednesday, with a notable FDA 510(k) clearance balanced by a miss on fourth quarter revenue and projections for fiscal year 2024 significantly below consensus expectations. Still, the takeaway is generally positive, with several strong catalysts expected to build momentum for the company in the coming year and much of the underperformance attributable to foreign exchange headwinds that have plagued many med-tech companies.
The accelerated clearance won by Johnson & Johnson (J&J) from the U.S. FDA for Talvey (talquetamab-tgvs) for relapsed or refractory multiple myeloma (r/r MM) in adults brings to the market the bispecific antibody that takes aim at G protein-coupled receptor class C group 5 member D, a target of interest to other developers as well.
Georgiamune Inc. announced that the FDA cleared its IND application for GIM-122, a first-in-class dual-functioning monoclonal antibody designed to target a highly novel mechanism to overcome immunotherapy resistance.
The U.S. FDA’s final guidance for premarket submissions for device software functions serves as a much needed overwrite of a badly outdated policy but includes some significant changes over the legacy guidance. There are also a few changes between the 2021 draft and the 2023 final guidance, however, such as a call for more details about how software anomalies were discovered and what a root cause analysis would suggest about the origin of the anomaly.
Trinity Biotech plc. received U.S. FDA 510(k) clearance for its lab-based hemoglobin diagnostic system, the Premier Resolution system, which the company hopes will allow it to regain its market leading position in hemoglobin variant detection. The Premier Resolution system is an automated analyzer which quantifies fetal hemoglobin and hemoglobin A2 and detects more than 200 hemoglobin variants. The device is a modern successor to the company’s Ultra system which once held a leading position in the U.S. hemoglobin variant diagnostic market.
RSS
