FDA grants orphan drug designation to potential treatment of STXBP1 developmental and epileptic encephalopathy
Research that was carried out by Baylor College of Medicine and Texas Children's Hospital doctors has led to the U.S. Food ...
Caliway Announced EMA Orphan Drug Designation Granted to CBL-514 for Dercum's Disease Treatment
Caliway Biopharmaceuticals (Caliway) announced that the European Medicines Agency (EMA) granted Orphan Drug Designation (ODD) to its novel small molecule drug, CBL-514, for the treatment of Dercum's ...
Targeted Oncology7d
MB-108 Gains FDA Orphan Drug Status for Malignant Glioma Treatment
MB-108 shows activity and is well tolerated in recurrent glioblastoma, with preclinical data supporting its combination with ...
Understanding orphan drugs: what are they, how are they classified and what are their challenges in the Indian context
Orphan drugs are further classified as approved by regulatory agencies like the U.S. Food and Drug Administration (FDA) or ...
Targeted Oncology8d
FDA Grants Orphan Drug Designation to LBL-034 in Multiple Myeloma
The FDA has granted LBL-034 orphan drug designation for relapsed/refractory multiple myeloma treatment. LBL-034 has received ...
AskBio Receives FDA Rare Pediatric Disease and Orphan-Drug Designations for AB-1003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2I/R9
First patient dosed in Phase 1/Phase 2 LION-CS101 trial of AB-1003 in August 2023, with enrollment continuing Asklepios ...
Mustang Bio Granted Orphan Drug Designation by U.S. FDA for MB-108 (HSV-1 oncolytic virus) to Treat Malignant Glioma
MB-108 (HSV-1 oncolytic virus) is active and well tolerated in patients with recurrent glioblastoma in ongoing Phase 1 clinical trialPreclinical ...
The Pharma Letter4d
FDA’s Orphan Products Grants Program awards significant funding for rare diseases
The US Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases ...
Business Insider4d
Attralus Therapeutic AT-02 Receives Orphan Drug Designation from the U.S. FDA for the Treatment of ATTR Amyloidosis
“We are pleased to have been granted orphan drug designation by the U.S. FDA for AT-02 in ATTR amyloidosis,” said Gregory Bell, M.D., Chief Medical Officer at Attralus. “Current approved therapies for ...
Monthly Prescribing Reference3d
Pan-Amyloid Removal Therapeutic Gets Orphan Drug Status for Systemic Amyloidosis
The Food and Drug Administration (FDA) has granted Orphan Drug designation to AT-02 for the treatment of transthyretin-associated amyloidosis (ATTR). AT-02 is a fusion protein of humanized ...
Pharmabiz1d
Caliway’s drug molecule CBL-514 to treat Dercum’s disease receives EMA orphan drug designation
Caliway’s drug molecule CBL-514 to treat Dercum’s disease receives EMA orphan drug designation: New Taipei City Thursday, November 14, 2024, 16:00 Hrs [IST] Caliway Biopharmac ...