“Receiving an FDA orphan drug designation for EP0042 validates this compound’s potential in a currently underserved area of medicine,” Rajan Jethwa, MA, MRCS, chief executive officer and co-founder of Ellipses Pharma, stated in a press release.1“The designation is an important milestone in ...
The US Food and Drug Administration (FDA) has granted orphan drug designation for Arcturus Therapeutics’ ARCT-032 for cystic fibrosis.
FDA Grants Orphan Drug Status to Plague VaccineLea EslavaKimPharmD
Friedreich's Ataxia Research Alliance (FARA) has announced in a press release that the FDA has granted orphan drug status to Edison Pharmaceutical’s EPI-743 (vatiquinone). The drug treats Friedreich’s ataxia, a rare nervous system disorder. Guy Miller, MD, PhD, Chairman & CEO, Edison ...
The FDA has granted orphan drug designation to toripalimab for the treatment of patients with small cell lung cancer. The FDA has granted an orphan drug designation to toripalimab, a PD-1 inhibitor, for patients with small cell lung cancer (SCLC), according to an announcement from Coher...
FDA Grants Orphan Drug Status to OV101 for Rare, Genetic Neurologic DisorderLea EslavaKimPharmD
Immunomedics, a leading biopharmaceutical company, announced that the Office of Orphan Products Development of the US Food and Drug Administration (FDA) has granted Trodelvy (sacituzumab govitecan-hziy) orphan status for the treatment of adult and pediatric patients with glioblastoma. ...
Orphan Drug status allows for recognition of the potential viability of a drug therapy while providing a variety of benefits during the drug approval process. These benefits include waivers of certain FDA fees, the availability of government grants, and FDA attention and assistance during the...
An orphan drug designation has been granted to the CD40 agonistic monoclonal antibody, sotigalimab, for the treatment of patients with soft tissue sarcoma. An orphan drug designation has been granted to the CD40 agonistic monoclonal antibody, sotigalimab (APX005M), for the treatment ...
US FDA grants orphan drug status to Ascentage’s MDM2-p53 inhibitor, APG-115 to treat gastric cancer