FDA Archives

News and Trends 28 Jul 2023

FDA grants breakthrough therapy designation to Duchenne muscular dystrophy treatment

The U.S. Food & Drug Administration (FDA) has granted Breakthrough Therapy Designation to NS Pharma Inc.’s NS-089/NCNP-02, an investigational candidate for patients with Duchenne muscular dystrophy amenable to exon 44 skipping therapy. The breakthrough designation is based on results from a first-in-human, investigator-initiated clinical trial conducted in Japan. In June 2023, NS-089/NCNP-02 was granted Rare […]

July 28, 2023 - 2 minutesmins - By Jim Cornall

News and Trends 25 Jul 2023

Molecure raises $12M and gets FDA clearance for pulmonary sarcoidosis trial

Molecure S.A. has received clearance from the U.S. Food and Drug Administration (FDA) on its Investigational New Drug (IND) application to conduct phase II clinical testing of OATD-01. The announcement follows the company closing a secondary public offering and entering into subscription agreements for all 2,776,000 H-shares offered by way of private placement, within the […]

July 25, 2023 - 4 minutesmins - By Jim Cornall

News and Trends 18 Jul 2023

FDA approves Beyfortus ahead of 2023-24 RSV season

The U.S. Food and Drug Administration (FDA) has approved Sanofi and AstraZeneca’s Beyfortus (nirsevimab-alip) for the prevention of respiratory syncytial virus (RSV) lower respiratory tract disease (LRTD) in newborns and infants born during or entering their first RSV season. The approval also covers children up to 24 months of age who remain vulnerable to severe […]

July 18, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 30 Jun 2023

FDA approves BioMarin gene therapy for hemophilia A

BioMarin Pharmaceutical Inc. has announced that the United States Food and Drug Administration (FDA) has approved ROCTAVIAN (valoctocogene roxaparvovec-rvox) gene therapy for the treatment of adults with severe hemophilia A (congenital factor VIII (FVIII) deficiency with FVIII activity < 1 IU/dL) without antibodies to adeno-associated virus serotype 5 (AAV5) detected by an FDA-approved test. The […]

June 30, 2023 - 5 minutesmins - By Jim Cornall

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

Neisseria gonorrhoeae gonorrhoea gonorrhea

News and Trends 27 Jun 2023

GSK gonorrhea vaccine fast tracked

The US Food and Drug Administration (FDA) has granted a Fast Track designation for GSK plc’s Neisseria gonorrhoeae investigational vaccine (NgG). The vaccine candidate is currently in an ongoing phase II trial and aims to demonstrate proof of concept by assessing the efficacy of the NgG vaccine in healthy adults, 18 to 50 years of […]

June 27, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 20 Jun 2023

FDA puts hold on Arcellx myeloma program following patient death

Arcellx, Inc. has received notification from the U.S. Food and Drug Administration (FDA) that a clinical hold has been placed on its CART-ddBCMA investigational new drug (IND) for the treatment of patients with relapsed or refractory multiple myeloma (rrMM). The clinical hold was received on June 16, following a recent patient death. The company said […]

June 20, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 15 Jun 2023

Nordic Bioscience cancer biomarker gets FDA go ahead

Danish biomarker company Nordic Bioscience says its PRO-C3 biomarker assay has received a Letter of Support (LoS) from the U.S. Food and Drug Administration (FDA). The letter acknowledges and supports further study of the PRO-C3 biomarker assay as a prognostic biomarker in tumor fibrosis studies. Nordic Bioscience has a successful track record of developing and […]

June 15, 2023 - 3 minutesmins - By Jim Cornall

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

Knowledge hub

Categories

Illuminating biotech in the City of Light: Inside Paris’s thriving ecosystem

ASCO 2025: key highlights in cancer therapeutics

Approaches

Therapeutic areas

New report: Artificial intelligence and the future of antibody discovery

Stem cell therapy: a potential cure for hearing loss?

R&D

BD

World biotech leaders

Connect with decision-makers and researchers worldwide for collaborative opportunities

Company

Join Us

Subscribe to our newsletter

Advertise with us

Illuminating biotech in the City of Light: Inside Paris’s thriving ecosystem

ASCO 2025: key highlights in cancer therapeutics

New report: Artificial intelligence and the future of antibody discovery

Stem cell therapy: a potential cure for hearing loss?

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

FDA

FDA grants breakthrough therapy designation to Duchenne muscular dystrophy treatment

Molecure raises $12M and gets FDA clearance for pulmonary sarcoidosis trial

FDA approves Beyfortus ahead of 2023-24 RSV season

FDA approves BioMarin gene therapy for hemophilia A

FDA gives Orphan Drug Designation to CellCentric multiple myeloma drug

GSK gonorrhea vaccine fast tracked

FDA puts hold on Arcellx myeloma program following patient death

Nordic Bioscience cancer biomarker gets FDA go ahead

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

Subscribe to our newsletter to get the latest biotech news!

Illuminating biotech in the City of Light: Inside Paris’s thriving ecosystem

ASCO 2025: key highlights in cancer therapeutics

New report: Artificial intelligence and the future of antibody discovery

Stem cell therapy: a potential cure for hearing loss?

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

Illuminating biotech in the City of Light: Inside Paris’s thriving ecosystem

ASCO 2025: key highlights in cancer therapeutics

New report: Artificial intelligence and the future of antibody discovery

Stem cell therapy: a potential cure for hearing loss?

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

Subscribe to our newsletter

Get the latest biotech news directly to your inbox!

Newsletter Signup - Footer

Subscribe to our newsletter to get the latest biotech news!