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News and Trends 29 Jun 2023

FDA gives Orphan Drug Designation to CellCentric multiple myeloma drug

CellCentric, a UK-based biotechnology company, has announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for inobrodib in the treatment of multiple myeloma. Delivered as an oral capsule, inobrodib can be used at home without requiring intensive monitoring. It is a first-in-class drug, with a new mechanism of action. It […]

June 29, 2023 - 3 minutesmins - By Jim Cornall

blood bleeding disorders von Willebrand disease

News and Trends 2 Jun 2023

von Willebrand disease treatment gets FDA orphan drug designation

The U.S. Food and Drug Administration (FDA) has granted Vega Therapeutics, Inc. orphan drug designation for its antibody therapy, VGA039, for the treatment of the rare bleeding disorder, von Willebrand disease (VWD). Vega Therapeutics is a clinical stage biotechnology company developing novel therapies for rare blood disorders. VGA039 is a first-in-class antibody therapy with a […]

June 2, 2023 - 2 minutesmins - By Jim Cornall

News and Trends 11 Apr 2023

Sumitomo Pharma Oncology receives orphan drug designation for Ewing sarcoma treatment

Sumitomo Pharma Oncology, Inc., a clinical-stage company focused on novel cancer therapeutics, said the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for TP-1287, an investigational oral CDK9 inhibitor, for the treatment of Ewing sarcoma. “We are delighted to have received this designation for TP-1287 which underscores the need for additional treatment […]

April 11, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 3 Mar 2023

Orphan drug designation for Laevoroc metabolic immune checkpoint inhibitor

Laevoroc Immunology, a privately-owned, Swiss oncology development company, has announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for immune checkpoint inhibitor LR 09. LR 09 is a novel metabolic immune checkpoint inhibitor, for the treatment of patients with hematological malignancies who are diagnosed with a relapse after allogeneic stem […]

March 3, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 3 Feb 2023

Genoscience Pharma receives orphan drug designation for hepatocellular carcinoma drug

Genoscience Pharma has announced that its lead candidate, ezurpimtrostat, a PPT-1 (Palmitoyl Protein Thioesterase-1) inhibitor, has been granted orphan drug designation (ODD) by the US Food and Drug Administration (FDA) for the treatment of hepatocellular carcinoma (HCC). ODD qualifies ezurpimtrostat for a potential seven years of market exclusivity after approval. The FDA’s ODD program provides […]

February 3, 2023 - 3 minutesmins - By Jim Cornall

More News! 30 Nov 2022

FDA grants Sensorion Orphan Drug Designation for hearing loss drug

Sensorion a clinical-stage biotechnology company has announced that the US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to OTOF-GT, its lead gene therapy program for hearing loss. The drug is intended for the treatment of otoferlin gene mediated hearing loss. Sensorion is on track to file a clinical trial application (CTA) […]

November 30, 2022 - 3 minutesmins - By Liza Laws

News and Trends 3 Oct 2022

Poxel granted orphan drug designation for kidney disease treatment

French biopharma company Poxel SA says the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to PXL770 for the treatment of patients with autosomal-dominant polycystic kidney disease (ADPKD). PXL770 is a novel, first-in-class direct adenosine monophosphate-activated protein kinase (AMPK) activator – and is also a phase 2 ready ADPKD asset, subject […]

October 3, 2022 - 2 minutesmins - By Jim Cornall

News and Trends 5 Sep 2022

Avacta’s drug targeting soft tissue sarcoma granted Orphan Drug Designation by FDA

A drug to treat soft tissue sarcoma has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA). Clinical stage oncology drug company Avacta Group plc has produced a form of the generic chemotherapy, doxorubicin AVA6000. About Avacta’s drug The drug has been modified using the company’s pre|CISION technology and Affirmer […]

September 5, 2022 - 2 minutesmins - By Liza Laws

News and Trends 23 Aug 2022

Ocelot Bio receives FDA Orphan Drug Designation for lead candidate for treatment of hepatorenal syndrome

Ocelot Bio, Inc., a clinical-stage biopharmaceutical company, has announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to its lead candidate OCE-205 for the treatment of hepatorenal syndrome. Ocelot Bio is currently enrolling patients in a phase 2 clinical trial of OCE-205 in hepatorenal syndrome with acute kidney injury (HRS-AKI), […]

August 23, 2022 - 3 minutesmins - By Jim Cornall

News and Trends 10 Aug 2022

Aro Biotherapeutics gets FDA orphan drug designation for treatment of Pompe Disease

Aro Biotherapeutics says the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to ABX1100, an investigational Centyrin-siRNA conjugate that targets the Gys1 gene in the muscle, for the treatment of Pompe disease. “We are pleased to have received this designation and are gratified by the FDA’s recognition of the potential of ABX1100 […]

August 10, 2022 - 2 minutesmins - By Jim Cornall

News and Trends 25 Oct 2017

Shire’s Hemophilia Gene Therapy Boosted by Orphan Drug Designation

The FDA has granted orphan drug designation to Shire’s hemophilia A gene therapy, giving it the boost it needs to catch up with others in the field. Shire specializes in the development of drugs for rare diseases, including hemophilia A – a bleeding disorder caused by a lack of clotting factor VIII activity in the […]

October 25, 2017 - 1 minutemin - By Alex Dale

More News! 29 Mar 2017

More “FDA Love” for Cannabis Company with Orphan Drug Designation

Last year was a doozy for biotech, but it was spectacular for GW Pharmaceuticals. Its streak continues with more special treatment. GW Pharmaceuticals, which is developing epilepsy treatments from marijuana, is making a name for itself not just as “that company” turning a recreational drug into medical ones; the company was also one of 2016’s surprise successes — its […]

March 29, 2017 - 2 minutesmins - By Evelyn Warner

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