The approval is supported by strong preclinical data demonstrating MP1032's capacity to sustain muscle function.

The FDA awarded orphan drug designation to VT3989 for mesothelioma, supporting the experimental cancer treatment’s ...

The FDA's Orphan Drug Designation is granted to investigational therapies intended for the treatment of rare diseases or conditions that affect fewer than 200,000 people in the United States.

Avadel Pharmaceuticals announces Orphan Drug Designation for LUMRYZ to treat Idiopathic Hypersomnia, emphasizing its once-nightly dosing benefit. Quiver AI Summary Avadel Pharmaceuticals announced ...

Currently, the Korean MFDS adopts a dual standard for orphan drug designation: 1. Drugs used for diseases with a domestic patient population (prevalence) of 20,000 or fewer; 2.

This new azeliragon designation adds to azeliragon's two other Orphan Drug Designations for the treatment of pancreatic cancer and glioblastoma, received in mid-2024 and early 2023, respectively.

The FDA has granted Cour Pharmaceuticals orphan drug designation for CNP-104, a biodegradable nanoparticle in development for the treatment of primary biliary cholangitis, the company announced in ...

ATSN-201 received orphan drug designation from the FDA for the treatment of X-linked retinoschisis, according to a press release from Atsena Therapeutics. “There are currently no approved ...

Palatin's Oral MC4R Agonist PL7737 Receives FDA Orphan Drug Designation for Obesity Due to Leptin Receptor Deficiency Provided by PR Newswire Mar 25, 2025, 4:30:00 AM ...

Ms. Jinhua Zhang, Founder, Chairperson, and CEO of IASO Biotherapeutics, stated: "We are delighted that equecabtagene autoleucel has been grated the Orphan Drug Designation by the SFDA.

Currently, the Korean MFDS adopts a dual standard for orphan drug designation: 1. Drugs used for diseases with a domestic patient population (prevalence) of 20,000 or fewer; 2.