Biodexa, which has already received U.S. FDA Orphan Drug designation for eRapa in FAP, plans to seek a similar designation in Europe. eRapa is a proprietary oral tablet formulation of rapamycin ...

takes to rare disease treatments that have secured an orphan drug designation. That includes when orphan drugs go through the Single Technology Appraisal (STA) route; the route that most take.

For many years, orphan drugs for rare diseases have been a draw for pharma companies, offering high prices, swift regulatory reviews, and often extended marketing exclusivity – but there are ...

Genprex, Inc. ("Genprex" or the "Company") , a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, today announced the advancement ...

Anticancer drug utilizing Trans-splicing Ribozyme technology Second Fast Track Designation following brain cancer approval SEONGNAM, South Korea, Feb. 18, 2025 /PRNewswire/ -- Rznomics announced ...

The FDA granted orphan drug designation to OPN-6602 for the potential treatment of relapsed/refractory multiple myeloma. The FDA granted orphan drug designation (ODD) to OPN-6602 for the treatment of ...

The evolution of cell and gene therapy is reshaping drug discovery, providing new hope for rare diseases and cancer through innovative gene editing approaches.

The idea that drug problems only exist in big cities is a myth. Every community, regardless of size, is facing these same issues.” His remarks followed his High in Plain Sight training ...

Drug discovery is the process through which potential new medicines are identified. It involves a wide range of scientific disciplines, including biology, chemistry and pharmacology. Many ...

US District Judge Amit P. Mehta’s memorandum opinion ruled the Food and Drug Administration violated the Orphan Drug Act in 2024 when it approved Aquestive Therapeutics Inc.’s drug, Libervant, to ...

The FDA granted 510(k) clearance for the VIA360 surgical system, which is designed to deliver viscoelastic fluid during ophthalmic surgeries, according to a press release from New World Medical ...