All Pharmacovigilance and safety oversight articles – Page 20
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NewsFDA approves first cell-based gene therapies for sickle cell disease
The US FDA has approved Vertex’s Casgevy and bluebird bio’s Lyfgenia, for one-time treatment of sickle cell disease in patients 12 years and older.
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NewsGSK immuno-oncology treatment achieves novel EU approval
The EC’s approval of the immuno-oncology treatment “will define a new standard of care for certain patients with advanced or recurrent endometrial cancer in the EU,” says principal investigator of the RUBY trial.
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ArticleAdvancing AOCs to transform the delivery of RNA therapeutics
In this article, Dr Michael Flanagan discusses how a new class of medicines called antibody oligonucleotide conjugates (AOCs) have the potential to overcome a major challenge encountered with many established RNA-based therapeutics: delivery to tissues outside the liver. Last year, AOCs demonstrated the first-ever successful targeted delivery of RNA into ...
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NewsASH 2023: microbiome-based therapy shows potential in graft-versus-host disease
Data presented at the 2023 American Society of Hematology (ASH) Annual Meeting suggest that the off-the-shelf microbiome therapeutic could offer a “potentially life-saving approach” in graft-versus-host disease (GvHD).
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NewsCommon drug could facilitate “huge step-change” in managing type 1 diabetes
A common rheumatoid arthritis drug can suppress the progression of type 1 diabetes, research shows, making it the first disease-modifying, oral treatment of its kind for the condition.
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NewsNovartis secures first-of-a-kind haematology approval
A novel Factor B inhibitor of the immune system's complement pathway has been approved for paroxysmal nocturnal haemoglobinuria (PNH).
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NewsPhase III data for novel telomerase inhibitor released
Currently, imetelstat is being reviewed by the FDA and EMA for the treatment of transfusion-dependent anaemia in adults with lower risk MDS.
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NewsPharmaceutical excipients market to witness expansion
According to research, increased in R&D investment for developing novel excipients and greater emphasis on patient-centric formulations are key drivers for the pharmaceutical excipients market.
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NewsNovel generalised myasthenia gravis treatment approved
A subcutaneous C5 complement inhibitor has been authorised by the European Commission (EC) as a treatment for generalised myasthenia gravis in Europe.
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NewsInnovative technologies facilitate novel contamination-detection method
A new 24-hour sterility testing method combining nanopore sequencing and machine learning could revolutionise sterility assurance in biopharmaceutical manufacturing of cell therapies.
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NewsFerring reveals long-term data for cancer gene therapy
New data from a Phase III gene therapy trial has demonstrated a 90 percent three-year overall survival rate for its participants with a high-risk bladder cancer.
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NewsFirst pill for desmoid tumours approved
Approval of the first treatment option for desmoid tumours beyond surgery and radiation has been granted by the US Food and Drug Administration (FDA).
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NewsCould stem cell therapy stabilise MS progression?
Positive findings from a first-in-human trial means that an advanced cell therapy for progressive multiple sclerosis will be evaluated in Phase II.
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Whitepaper
Application note: Role of in-house isolates in pharmaceutical quality control
This application note addresses the need to evaluate key sources of contamination to maintain an optimum level of pharmaceutical quality control.
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NewsFirst treatment recommended for infants with Wolman Disease
Alexion’s intravenous enzyme replacement therapy has been recommended for the ultra-rare disease by the National Institute for Health and Care Excellence (NICE).
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NewsLong COVID study expanded across Europe
If biotech Berlin Cures’ larger Phase III study of its Long COVID drug candidate goes ahead, the findings will support the treatment’s potential future regulatory approval.
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NewsVertex scores European cystic fibrosis medicine approval
Following a milestone approval by the European Commission in July 2023, Vertex Pharmaceuticals has been granted a label expansion for its cystic fibrosis medicine KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) with ivacaftor.
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NewsNew Amgen immunotherapy could treat common solid tumour
With demonstrated benefit in anti-tumour activity and overall survival in patients with small cell lung cancer, Tarlatamab could provide a new third-line option, a Phase II study suggests.
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NewsEC approves first-line ovarian cancer treatment
A Phase III trial evaluating rucaparib demonstrated that it significantly improved progression-free survival in women with advanced ovarian cancer, regardless of their BRCA mutation status.
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NewsBRUKINSA® granted significant EU approval in follicular lymphoma
The first and only Bruton’s tyrosine kinase (BTK) inhibitor approved for follicular lymphoma in the EU, now has the broadest label of any medicine in its class globally.