Uniqure NV’s Hemgenix (etranacogene dezaparvovec-drlb), a one-time gene therapy – the first for the treatment of adults 18 and older living with hemophilia B – won approval from the U.S. FDA. Previously known as Etranadez, Hemgenix is indicated for patients who use factor IX prophylaxis therapy or have current or historical life-threatening hemorrhage. Those with repeated, serious spontaneous bleeding episodes are eligible, too. CSL Ltd. licensed the exclusive global rights to Hemgenix from Uniqure in May 2021 and is handling further development, registration, and commercialization of the therapy.

As clinical data drops from last year, COVID-19 reports are down 48%

Nearing the end of 2022, the COVID-19 pandemic clearly no longer dominates the clinical activity reported by biopharma companies. Overall clinical data tracked by BioWorld is down significantly, as is the proportion focused on the SARS-Cov-2 virus. Therapeutics and vaccines targeting cancer, infectious diseases and neurological disorders, however, remain a stronghold.

GABAA efforts march on; Avenue, Sage, others busy in space 

Avenue Therapeutics Inc.’s recent takeover of Baergic Bio Inc. highlighted the potential of targeting GABAA, an approach under investigation in various quarters. A standout is Sage Therapeutics Inc., with zuranolone, and plans to finish its rolling NDA in major depressive disorder and postpartum depression in December of this year. Others are in play, too.

FDA divisions to develop pilot for companion diagnostic performance criteria

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 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 for these studies and thus undercut any incentive to develop a patented in vitro diagnostic for that purpose.

Innate immunity can drive radiation-induced abscopal effect

A combination of radiation therapy and CD47 blockade induced an abscopal effect in animal studies even in animals that lacked T cells, researchers reported in the Nov. 21, 2022, online issue of Nature Cancer. The findings are “the first demonstration of T-cell-independent abscopal response,” co-corresponding author Edward Graves told BioWorld.

Holiday notice

BioWorld's offices will be closed in observance of Thanksgiving in the U.S. No issues will be published Thursday, Nov. 24, or Friday, Nov. 25.

Also in the news

AB Science, Acticor, Argenx, Arvinas, Biosight, Cassava, Embarkneuro, First Wave, G1, Gamida Cell, GSK, Innocare, Intelgenx, Intrabio, Junshi, Khondrion, Marker, Merck, Morphic, Oculis, Palisade, Regeneron, Revive, Takeda, TC, Telix, VBI Vaccines