The FDA has awarded orphan drug designation to Sensorion SA's OTOF-GT, a dual vector AAV gene therapy, for the treatment of otoferlin gene-mediated hearing loss.
The FDA has awarded orphan drug designation to Tenaya Therapeutics Inc.'s gene therapy product candidate, TN-401, for the treatment of arrhythmogenic right ventricular cardiomyopathy (ARVC). TN-401 is an adeno-associated virus (AAV)-based gene therapy being developed for the treatment of genetic ARVC caused by plakophilin-2 (PKP2) gene mutations.
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.
The FDA has awarded orphan drug designation to Eikonoklastes Therapeutics Inc.'s ET-101 program for the treatment of amyotrophic lateral sclerosis (ALS).
The FDA has awarded orphan drug designation to Omega Therapeutics Inc.'s OTX-2002, a first-in-class epigenomic controller engineered to downregulate c-Myc (MYC), for the treatment of hepatocellular carcinoma (HCC).
Novaccess Global Inc. has announced the approval of its application with the FDA for orphan drug designation for TLR-AD1, a vaccine immunotherapy for the treatment of aggressive brain cancers, including glioblastoma and other high-grade gliomas. The company’s therapeutic path involves a unique transformational process, which involves the addition of proprietary substances to create a cocktail for more personalized treatment that substantially increases clinical benefits for patients.
South Korea’s drug regulator should consider revising orphan drug definitions or allow more new innovative drugs to be excluded from pharmacoeconomic assessments, which are required for new drugs to be reimbursed under Korea’s single-payer system, the European Chamber of Commerce in Korea said.
Verismo Therapeutics Inc. has received U.S. orphan drug designation from the FDA for Synkir-110 for the treatment of patients with mesothelin-expressing mesotheliomas.
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