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.
The EMA's Committee for Orphan Medicinal Products (COMP) has adopted a positive opinion on Sensorion SA's application seeking orphan drug designation for OTOF-GT, a gene therapy intended for the treatment of otoferlin gene-mediated hearing loss.
The FDA has granted orphan drug designation to Iecure Inc.'s lead product candidate GTP-506, an investigational product for the treatment of ornithine transcarbamylase (OTC) deficiency.
Although Pfizer Inc. has the only drugs approved in the U.S. to treat a rare, progressive heart disease, the U.S. Court of Appeals for the Second Circuit agreed this week with the Department of Health and Human Services, and a lower court, that Pfizer’s proposed copay assistance program for middle-income Americans covered by Medicare would violate the federal Anti-Kickback Statute – even if the company has no “corrupt” intent.
The U.S. FDA has granted Alpheus Medical Inc. orphan drug and fast track designations for a therapeutic platform that could improve outcomes for patients suffering from recurrent glioblastoma (GBM), the most common primary brain cancer and among the hardest to treat.
Following an investigation into Leadiant Group’s pricing of a rare disease drug, the Italian Competition Authority fined the privately held company about €3.5 million (US$3.76 million) May 31 for charging the Italian National Health Service excessive prices since 2017 for Chenodeoxycholic Acid Leadiant.
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