FDA Archives

News and Trends 8 Jun 2023

Minoryx can start phase 3 trial for cerebral adrenoleukodystrophy patients

Spanish-headquartered Minoryx Therapeutics says the U.S. Food and Drug Administration (FDA) has approved its phase 3 clinical trial (CALYX) of lead candidate leriglitazone, to treat adult male X-linked adrenoleukodystrophy (X-ALD) patients with cerebral adrenoleukodystrophy (cALD). X-ALD is an orphan neurodegenerative disease. The global incidence of X-ALD is approximately six to eight per 100,000 live births. […]

June 8, 2023 - 4 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 26 May 2023

Rocket Pharmaceuticals receives FDA designation for pyruvate kinase deficiency gene therapy

Rocket Pharmaceuticals, Inc. has announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to RP-L301, the company’s investigational lentiviral-based gene therapy for pyruvate kinase deficiency (PKD). PKD is a rare blood disorder characterized by severe anemia and excessive red blood cell breakdown. RMAT designation was granted based […]

May 26, 2023 - 4 minutesmins - By Jim Cornall

News and Trends 23 May 2023

FDA approval for Rznomics’ glioblastoma treatment trial

Rznomics Inc., a South Korea based biopharmaceutical company specializing in the development of RNA-based gene therapeutics, has received phase 1/2a IND approval from the U.S. FDA for its glioblastoma multiforme (GBM) treatment. RZ-001 initially obtained IND approval for hepatocellular carcinoma (HCC), but Rznomics also found pre-clinical efficacy in GBM models and submitted the IND for […]

May 23, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 22 May 2023

FDA approves first topical gene therapy for dystrophic epidermolysis bullosa wound treatment

The U.S. Food and Drug Administration has approved Vyjuvek, a herpes-simplex virus type 1 (HSV-1) vector-based gene therapy, for the treatment of wounds in patients 6 months of age and older with dystrophic epidermolysis bullosa (DEB) with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene. The approval of Vyjuvek was granted to […]

May 22, 2023 - 3 minutesmins - By Jim Cornall

Osteopetrosis ADO2 radiopaedia case ID 8037

News and Trends 16 May 2023

SiSaf siRNA therapy gets FDA designations to treat autosomal dominant osteopetrosis

SiSaf Ltd, an RNA delivery and therapeutics company, has announced that SIS-101-ADO, its siRNA therapeutic for patients with autosomal dominant osteopetrosis Type 2 (ADO2), has been granted orphan drug designation by the U.S. FDA. Also, due to the serious manifestations of this rare skeletal disorder in children, SIS-101-ADO has been granted rare pediatric disease designation […]

May 16, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 4 May 2023

World’s first RSV vaccine for older adults gets FDA approval

GSK plc says the US Food and Drug Administration (FDA) has approved Arexvy (respiratory syncytial virus vaccine, adjuvanted) for the prevention of lower respiratory tract disease (LRTD) caused by respiratory syncytial virus (RSV) in individuals 60 years of age and older. It is the first RSV vaccine for older adults to be approved anywhere in […]

May 4, 2023 - 5 minutesmins - By Jim Cornall

News and Trends 2 May 2023

Cabaletta Bio’s lupus drug cleared for trial

Cabaletta Bio, Inc., a U.S. clinical-stage biotechnology company focused on developing and launching the first curative targeted cell therapies for patients with autoimmune diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted fast track designation to CABA-201. Cabaletta Bio’s CABA-201 is a 4-1BB-containing fully human CD19-CAR T cell investigational therapy, designed […]

May 2, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 2 May 2023

FDA fast track for Memo’s kidney transplant infection treatment

Memo Therapeutics AG, a biotech company developing therapeutic antibodies, says the U.S. Food and Drug Administration (FDA) has granted fast track designation to AntiBKV, Memo Therapeutics’ lead antibody therapeutic that targets BK polyomavirus (BKV) infection commonly seen in renal transplant patients. AntiBKV has successfully completed a phase I clinical study and following FDA clearance has […]

May 2, 2023 - 2 minutesmins - By Jim Cornall

News and Trends 2 May 2023

Schizophrenia injection gets FDA approval

Teva Pharmaceuticals, a U.S. affiliate of Teva Pharmaceutical Industries Ltd., and MedinCell have announced the U.S. Food and Drug Administration (FDA) has approved UZEDY (risperidone) extended-release injectable suspension for the treatment of schizophrenia in adults. UZEDY is the first subcutaneous, long-acting formulation of risperidone that utilizes SteadyTeq, a copolymer technology proprietary to MedinCell that controls […]

May 2, 2023 - 4 minutesmins - By Jim Cornall

News and Trends 27 Apr 2023

Biogen ALS drug gets FDA ok

The U.S. Food and Drug Administration (FDA) has approved Biogen’s QALSODY (tofersen) 100 mg/15mL injection for the treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene. This indication is approved under accelerated approval based on reduction in plasma neurofilament light chain (NfL) observed in patients […]

April 27, 2023 - 5 minutesmins - By Jim Cornall

News and Trends 20 Apr 2023

FDA approves Genentech’s lymphoma drug

Genentech, a member of the Roche Group, has announced the U.S. Food and Drug Administration (FDA) has approved Polivy (polatuzumab vedotin-piiq) in combination with Rituxan (rituximab), cyclophosphamide, doxorubicin and prednisone (R-CHP). The approval covers the treatment of adult patients who have previously untreated diffuse large B-cell lymphoma (DLBCL), not otherwise specified (NOS) or high-grade B-cell […]

April 20, 2023 - 4 minutesmins - By Jim Cornall

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FDA

Minoryx can start phase 3 trial for cerebral adrenoleukodystrophy patients

von Willebrand disease treatment gets FDA orphan drug designation

Rocket Pharmaceuticals receives FDA designation for pyruvate kinase deficiency gene therapy

FDA approval for Rznomics’ glioblastoma treatment trial

FDA approves first topical gene therapy for dystrophic epidermolysis bullosa wound treatment

SiSaf siRNA therapy gets FDA designations to treat autosomal dominant osteopetrosis

World’s first RSV vaccine for older adults gets FDA approval

Cabaletta Bio’s lupus drug cleared for trial

FDA fast track for Memo’s kidney transplant infection treatment

Schizophrenia injection gets FDA approval

Biogen ALS drug gets FDA ok

FDA approves Genentech’s lymphoma drug

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