FDA Archives

News and Trends 29 Mar 2023

Pharming immunodeficiency drug given FDA approval

Pharming Group N.V. has announced that the US Food and Drug Administration (FDA) has approved Joenja (leniolisib) for the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older. Pharming’s Joenja, an oral, selective PI3Kδ inhibitor, is the first and only treatment approved in the […]

March 29, 2023 - 4 minutesmins - By Jim Cornall

Best in Biotech 9 Mar 2023

5 advancements in kidney disease research over the past year

Prevalent in millions of people across the globe, kidney diseases manifest through various symptoms like blood and excess protein in urine, fluid retention causing swollen ankles, poor appetite and even the inability of the body to filter waste, resulting in acute kidney failure. To boost the research and development of treatments for kidney diseases, Kidney […]

March 9, 2023 - 7 minutesmins - By Roohi Mariam Peter

Interview 3 Mar 2023

Interview: how has the first gene therapy for hemophilia B transformed research?

Hemophilia B, a rare disease that affects more than 230,000 people worldwide is caused by the lack of or defective clotting protein factor IX often due to a spontaneous mutation in the F9 gene, leading to excessive bleeding. Until recently, routine prophylactic infusions of factor IX replacement therapy to maintain enough clotting factor to prevent […]

March 3, 2023 - 8 minutesmins - By Roohi Mariam Peter

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 24 Feb 2023

Sanofi hemophilia drug gets FDA approval

The U.S. Food and Drug Administration (FDA) has approved Sanofi’s ALTUVIIIO [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl], previously referred to as efanesoctocog alfa, a first-in-class, high-sustained factor VIII replacement therapy. ALTUVIIIO is indicated for routine prophylaxis and on-demand treatment to control bleeding episodes, as well as perioperative management (surgery) for adults and children with hemophilia […]

February 24, 2023 - 4 minutesmins - By Jim Cornall

News and Trends 23 Feb 2023

Depression trial gets green light

Beckley Psytech Ltd has received investigational new drug (IND) approval from the U.S. Food and Drug Administration (FDA) for a global multi-site phase IIb study exploring the safety, efficacy and tolerability of two doses of its lead compound, BPL–003, in patients with treatment resistant depression (TRD). BPL-003 is the Beckley Psytech’s novel synthetic formulation of […]

February 23, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 21 Feb 2023

FDA approves alpha-mannosidosis drug

Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici S.p.A., an international research-focused healthcare Group (Chiesi Group), says the U.S. Food and Drug Administration (FDA) has approved Lamzede (velmanase alfa-tycv) for the treatment of non-central nervous system manifestations of alpha-mannosidosis (AM) in adult and pediatric patients. AM is an ultra-rare, progressive lysosomal storage disorder […]

February 21, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 10 Feb 2023

FDA approves GSK drug for recurrent or advanced mismatch repair-deficient endometrial cancer

GSK plc says the US Food and Drug Administration (FDA) has given full approval for Jemperli (dostarlimab-gxly) for the treatment of adult patients with mismatch repair-deficient (dMMR) recurrent or advanced endometrial cancer, as determined by a US FDA-approved test, that has progressed on or following a prior platinum-containing regimen in any setting and are not […]

February 10, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 10 Feb 2023

Rocket Pharmaceuticals receives FDA RMAT designation for Danon disease gene therapy treatment

Rocket Pharmaceuticals, Inc says the U.S. Food and Drug Administration (FDA) has granted regenerative medicine advanced therapy (RMAT) designation to RP-A501. RP-A501 is Rocket Pharmaceuticals’ investigational adeno-associated virus (AAV)-based gene therapy for the treatment of Danon disease, a devastating and fatal genetic cardiac disease for which there are no disease-altering therapies available. RMAT designation was […]

February 10, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 7 Feb 2023

Endogena Therapeutics retinitis pigmentosa treatment gets FDA fast track designation

The US Food and Drug Administration (FDA) has designated the investigation of Endogena Therapeutics’ EA-2353 for the treatment of retinitis pigmentosa (RP) as a fast track development program. Fast track is a process designed to enable patients to benefit earlier from new drugs for serious conditions. Endogena Therapeutics’ EA-2353 takes a novel, small-molecule approach and […]

February 7, 2023 - 2 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

News and Trends 31 Jan 2023

Stemline Therapeutics’ breast cancer treatment approved

The Menarini Group, an Italian pharmaceutical and diagnostics company, says that the U.S. Food and Drug Administration (FDA) has approved ORSERDU for the treatment of postmenopausal women or adult men, with ER+, HER2-, ESR1-mutated advanced or metastatic breast cancer with disease progression following at least one line of endocrine therapy. Stemline Therapeutics, a wholly-owned subsidiary […]

January 31, 2023 - 3 minutesmins - By Jim Cornall

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FDA

Pharming immunodeficiency drug given FDA approval

5 advancements in kidney disease research over the past year

Interview: how has the first gene therapy for hemophilia B transformed research?

Orphan drug designation for Laevoroc metabolic immune checkpoint inhibitor

Sanofi hemophilia drug gets FDA approval

Depression trial gets green light

FDA approves alpha-mannosidosis drug

FDA approves GSK drug for recurrent or advanced mismatch repair-deficient endometrial cancer

Rocket Pharmaceuticals receives FDA RMAT designation for Danon disease gene therapy treatment

Endogena Therapeutics retinitis pigmentosa treatment gets FDA fast track designation

Genoscience Pharma receives orphan drug designation for hepatocellular carcinoma drug

Stemline Therapeutics’ breast cancer treatment approved

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10 oncology deals in 2025 spotlight where industry leaders are betting big

10 biotech companies leading the way in Denmark

Webinar: Advancements in antimalarial drug discovery and development

Six psychedelic drug companies aiming to revolutionize mental health treatment

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

10 oncology deals in 2025 spotlight where industry leaders are betting big

10 biotech companies leading the way in Denmark

Webinar: Advancements in antimalarial drug discovery and development

Six psychedelic drug companies aiming to revolutionize mental health treatment

Connect with decision-makers and researchers worldwide for collaborative opportunities

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Advertise with us

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