Tikun Therapeutics Inc. has obtained U.S. orphan drug and rare pediatric disease designations for its programs in familial dysautonomia, namely its rAAV2-U1a-hELP1 gene replacement therapy for the treatment of optic neuropathy in familial dysautonomia and BPN-36964 for systemic treatment of familial dysautonomia.
Medexus Pharmaceuticals Inc. looks set for its U.S. launch of bifunctional alkylating agent treosulfan in the first half of 2025 following a long-awaited FDA approval of the drug, branded Grafapex, for use in combination with fludarabine as a preparative regimen for allogeneic hematopoietic stem cell transplantation (allo-HSCT) in adults and pediatric patients, 1 and older, with acute myeloid leukemia or myelodysplastic syndrome.
Roche AG secured U.S. FDA 510(k) clearance for its cobas liat sexually transmitted infection (STI) multiplex assay panels. The tests, which also received a Clinical Laboratory Improvement Amendments waiver, will enable clinicians to detect several STIs including chlamydia, gonorrhea and Mycoplasma genitalium, in as little as 20 minutes.
Standing for live visualization of brain circuits, LVIS Corp launched the cloud-based Neuromatch Access platform as a software as a service to help neurologists and medical professionals review, interpret and analyze electroencephalogram (EEG) data.
Repair Biotechnologies Inc. has received encouraging feedback from a pre-IND meeting with the FDA as it works toward an IND filing to conduct a phase Ib study of its REP-0003 mRNA therapy in patients with homozygous familial hypercholesterolemia (HoFH).
It’s one thing for the scientific community to propose a fundamental change to the way obesity is defined and diagnosed. But it’s another for that proposal to be adopted by regulators, especially when the current definition that relies primarily on the body mass index is entrenched in guidance and obesity drug development and approval.
Marking the second global approval after Japan, the U.S. FDA has approved Datroway (datopotamab deruxtecan), a trophoblast cell surface antigen 2-directed antibody-drug conjugate (ADC) from Daiichi Sankyo Co. Ltd. and Astrazeneca plc, for treating adults with hormone receptor-positive, HER2-negative unresectable or recurrent breast cancer after prior chemotherapy.
Just a few weeks later than expected, Inflammatix Inc. secured U.S. FDA clearance of the Triverity test system for use in emergency triage of patients with suspected acute infection or sepsis. The molecular blood test is the first to identify bacterial and viral infections and provide an all-cause risk evaluation of the likelihood of developing severe illness.
It’s one thing for the scientific community to propose a fundamental change to the way obesity is defined and diagnosed. But it’s another for that proposal to be adopted by regulators, especially when the current definition that relies primarily on the body mass index is entrenched in guidance and obesity drug development and approval.
Atara Biotherapeutics Inc. received a complete response letter (CRL) from the U.S. FDA for its lead product, Ebvallo (tabelecleucel), a breakthrough therapy for Epstein-Barr virus positive post-transplant lymphoproliferative disease that is already approved in the EU, the U.K. and Switzerland.
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