Regulatory approval Archives

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 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 10 Feb 2023

Europe approves childhood epilepsy treatment

UCB’s FINTEPLA (fenfluramine) oral solution has been approved in the European Union (EU) for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS) as an add-on therapy to other anti-epileptic medicines for patients two years of age and older. The approval by the European Commission (EC) was based on safety and efficacy data from a […]

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

UK approval for AbbVie Crohn’s disease drug

AbbVie has announced that the UK Medicines and Healthcare products Regulatory Agency (MHRA) has approved RINVOQ (upadacitinib), a janus kinase (JAK) inhibitor, for the treatment of adult patients with moderately to severely active Crohn’s disease (CD) who have had an inadequate response, lost response or were intolerant to either conventional therapy or a biological agent. […]

February 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

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

News and Trends 31 Jan 2023

Stargardt disease treatment gets FDA fast track

Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, has announced that the U.S. Food and Drug Administration (FDA) has granted fast track designation (FTD) for MCO-010. MCO-010 is an ambient-light activatable multi-characteristic opsin (MCO) optogenetic monotherapy to restore vision in blind patients, for the treatment of Stargardt disease to […]

January 31, 2023 - 2 minutesmins - By Jim Cornall

News and Trends 31 Jan 2023

Abbisko Therapeutics receives breakthrough therapy designation for CSF-1R inhibitor

Abbisko Therapeutics Co., Ltd. has announced that its CSF-1R inhibitor Pimicotinib has been granted the breakthrough therapy designation from the FDA for the treatment of tenosynovial giant cell tumor (TGCT) patients that are not amenable to surgery. The designation approval is based on results from the phase Ib clinical trial of TGCT cohort for Pimicotinib. […]

January 31, 2023 - 4 minutesmins - By Jim Cornall

News and Trends 27 Jan 2023

EMA accepts Marketing Authorization Application for lecanemab to treat early Alzheimer’s disease

BioArctic AB’s partner Eisai says the European Medicines Agency (EMA) has accepted a marketing authorization application (MAA) for lecanemab. Lecanemab, known as LEQEMBI in the U.S., is an investigational anti-amyloid beta (Aβ) protofibril antibody, for the treatment of early Alzheimer’s disease (mild cognitive impairment due to Alzheimer’s disease (AD) and mild AD dementia) with confirmed […]

January 27, 2023 - 2 minutesmins - By Jim Cornall

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Regulatory approval

Sanofi hemophilia drug gets FDA approval

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

Europe approves childhood epilepsy treatment

Endogena Therapeutics retinitis pigmentosa treatment gets FDA fast track designation

UK approval for AbbVie Crohn’s disease drug

Genoscience Pharma receives orphan drug designation for hepatocellular carcinoma drug

Stemline Therapeutics’ breast cancer treatment approved

Stargardt disease treatment gets FDA fast track

Abbisko Therapeutics receives breakthrough therapy designation for CSF-1R inhibitor

EMA accepts Marketing Authorization Application for lecanemab to treat early Alzheimer’s disease

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