Release Date: 07 October 2021
The European Commission (EC) has given orphan designation to AOC 1001 for the treatment of myotonic dystrophy type 1 (DM1). The decision by the EC follows the U.S. Food and Drug Administration (FDA) also granting orphan drug design...
Release Date: 06 October 2021
European Commission has granted Orphan Drug Designation to ZYNLONTAandreg;, a CD19-targeted ADC, for the treatment of diffuse large B-cell lymphoma (DLBCL). ZYNLONTAandreg; is a CD19-directed antibody drug conjugate (ADC). Once bound to ...
Release Date: 06 October 2021
US FDA has granted Orphan Drug Designation to Nadunolimab (CAN04) to cure the pancreatic cancer. Nadunolimab is currently evaluated in ongoing clinical trials for treatment of PDAC. A phase I study, CAPAFOUR, was started in H1’2021...
Release Date: 05 October 2021
US FDA has granted the orphan-drug designation to gavo-cel for the treatment of cholangiocarcinoma, a group of cancers that begin in the bile ducts. TCR Therapeutics is studying gavo-cel in a Phase 1/2 clinical trial to treat patients wi...
Release Date: 05 October 2021
US FDA has granted orphan drug designation (ODD) for the company's investigational medicinal product, octreotide subcutaneous depot (CAM2029), for the treatment of autosomal dominant polycystic liver disease.
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Release Date: 04 October 2021
PLX-200 Drug granted Orphan Drug Designation from FDA for the treatment of Krabbe Disease Therapy. The FDA’s Orphan Drug designation is granted to medicines intended to treat or prevent rare diseases or disorders that affect fewer ...
Release Date: 04 October 2021
FDA has granted LP-184 Orphan Drug Designation as a new treatment of glioblastoma multiforme (GBM) and other malignant gliomas, which follows the recent announcement of the approval of the drug to be used for pancreatic cancer treatment....
Release Date: 03 October 2021
U.S FDA has granted Orphan Drug, Fast Track and Qualified Infectious Disease Product designations for opelconazole (PC945) for the treatment of invasive pulmonary aspergillosis (IPA).
Opelconazole (PC945),...
Release Date: 03 October 2021
U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to lead product candidate VS-01 for the treatment of hyperammonemia (HA) in inborn errors of metabolism (IEM).
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Release Date: 02 October 2021
US FDA has granted both Orphan Drug Designation and Rare Pediatric Disease Designation for the company’s lead gene therapy product candidate, DB-OTO, for the treatment of otoferlin-related congenital hearing loss. DB-OTO is a dual-...
Release Date: 02 October 2021
Both the European Commission (EC) and FDA have granted Orphan Drug Designation for FLT201 for the treatment of Gaucher disease. FLT201 is an investigational liver-directed AAV gene therapy for the treatment of Gaucher disease Type 1. FLT...
Release Date: 01 October 2021
U.S. Food and Drug Administration (FDA) Office of Orphan Products Development (OOPD) has awarded seven years of marketing exclusivity for Octagamandreg; 10% [Immune Globulin Intravenous (Human)], the first and only intravenous immunoglob...
Release Date: 01 October 2021
PLX-200 drug to treat GM2 gangliosidoses has been granted by the U.S. Food and Drug Administration (FDA). This disease is also known as Tay-Sachs and Sandhoff diseases that are ultra-rare and fatal pediatric neurodegenerative disorders. ...
Release Date: 30 September 2021
European Commission has granted orphan drug designation for DAY101, for the treatment of glioma. The most common brain tumor diagnosed in children is Pediatric low-grade glioma (pLGG), accounting for 30% – 50% of all central nervou...
Release Date: 30 September 2021
US FDA has granted orphan drug designation to Tesomet for the treatment of hypothalamic obesity (HO). Hypothalamic obesity (HO) is a rare disorder caused by injury to a region of the brain known as the hypothalamus. It is caused by intra...