The US Food and Drug Administration (FDA) has granted the Gilead Sciences subsidiary Kite a Regenerative Medicine Advanced Therapy Designation (RMAT) for Yescarta (axicabtagene ciloleucel) for adults with newly-diagnosed, high-risk large B-cell lymphoma (LBCL). 3 October 2024
AstraZeneca’s supplemental New Drug Application (sNDA) for Calquence (acalabrutinib) has been accepted and granted Priority Review in the USA for the treatment of adults with previously untreated mantle cell lymphoma (MCL). 3 October 2024
Shares of UK drug developer Hemogenyx Pharmaceuticals rose as much as 9% and closed up 5.9% at 1.70 pence yesterday on the news of a further investment from Prevail Partners. 3 October 2024
CSL Behring has been granted a positive recommendation by the Spanish Interministerial Commission on the Pricing of Medicines (CIPM), resulting in national reimbursement for Hemgenix (etranacogene dezaparvovec) for eligible haemophilia B patients in Spain. 1 October 2024
Johnson & Johnson has announced long-term results from the Phase III CARTITUDE-4 study showing that a single infusion of Carvykti (ciltacabtagene autoleucel; cilta-cel) significantly extended overall survival (OS) in those with relapsed or lenalidomide-refractory multiple myeloma who have received at least one prior line of therapy, including a proteasome inhibitor (PI). 30 September 2024
Pfizer (NYSE: PFE) has announced the withdrawal of Oxbryta (voxelotor), a treatment for sickle cell disease (SCD), from all global markets. 26 September 2024
US biotech 2seventy bio and partner Bristol Myers Squibb today revealed that they will discontinue enrollment in its ongoing Phase III KarMMa-9 study. 25 September 2024
CSL Vifor today announced that Japan’s Ministry of Health and Labor Welfare (MHLW) has granted its partner, Zeria Pharmaceutical marketing authorization approval for Veltassa (patiromer). 24 September 2024
Aptadir Therapeutics, a biotech company focused on RNA inhibitor-based therapies for cancer and genetic conditions, has launched with $1.6 million in pre-seed funding. 24 September 2024
The US Food and Drug Administration (FDA) has approved Sarclisa (isatuximab) in combination with bortezomib, lenalidomide, and dexamethasone (VRd) as a first line treatment option for adult patients with newly diagnosed multiple myeloma (NDMM). 23 September 2024
The European Medicines Agency’s (EMA) Committee for Human Medicinal Products (CHMP) recommended approval of five novel medicines at its September meeting 20 September 2024
Daiichi Sankyo’s today revealed that its Vanflyta (quizartinib) has been recommended by the National Institute for Health and Care Excellence (NICE) for routine National Health Service (NHS) commissioning in England and Wales. 19 September 2024
The upcoming European Society for Medical Oncology (ESMO) Congress 2024 should provide a reminder—if it were needed—of the pace of innovation in the development of new medicines for cancer. 10 September 2024
Epsilogen, a specialist in the development of immunoglobulin E (IgE) antibodies to treat cancer, has announced the completion of a £12.5 million ($16.4 million) Series B expansion financing. 9 September 2024
Vor Bio has announced positive data from its ongoing Phase I/II VBP101 study evaluating the combination of trem-cel and Mylotarg (gemtuzumab ozogamicin) in people with relapsed or refractory acute myeloid leukemia (AML). 9 September 2024
Last week’s research news included French pharma major Sanofi releasing mixed Phase III results for it tolebrutinib in progressive multiple sclerosis. Also of note, US vaccine developer Vaxcyte announced strong early-stage trial results for its VAX-31 in pneumococcal disease. On the regulatory front, US drugmaker Travere Therapeutics gained full approval from the US Food and Drug Administration (FDA) for its kidney disease drug Filspari. US cancer drug developer IN8bio announced a prioritization of its R&D pipeline to focus on INB-100 in acute myeloid leukemia, along with a workforce cutback. 8 September 2024
Shares of US gamma-delta T cell therapies for cancer developer IN8bio closed down more than 7% yesterday and fell a further 3.2% to $0.465 pre-market, after it announced a major reshuffle. 5 September 2024
Russian drugmaker Polysan will invest about 4 billion roubles (~$60 million) in the establishment of full-cycle production of a drug from German pharma major Bayer, according to the company, reports The Pharma Letter’s local correspondent. 13 December 2018
Australia has become the first country anywhere to grant regulatory approval to a cancer drug originally found in a marine microorganism. 11 December 2018
Following its acceptance in the UK’s Cancer Drug Fund in October, the medicines cost-effectiveness watchdog has now agreed to the regular NHS England funding of Yescarta (axicabtagene ciloleucel). 7 December 2018
Swedish Orphan Biovitrum and Novimmune have presented positive data from a pivotal Phase II/III study of Gamifant (emapalumab-lzsg) at the annual meeting of the American Society of Hematology. 5 December 2018
Janssen Pharmaceutical, the pharma business of US healthcare company Johnson & Johnson, has announced positive Phase III data for Darzalex (daratumumab) at the annual meeting of the American Society of Hematology. 5 December 2018
Belgo-Dutch biotech argenx yesterday announced an exclusive, global collaboration and license agreement for cusatuzumab (ARGX-110), adding to its lucrative deal in the summer with AbbVie (NYSE: ABBV), with the news sending its share leaping 13% to 10.90 euros by close of trading on Monday. 4 December 2018
Swiss pharma giant Roche has presented data from the largest pivotal study to date in children with hemophilia A at the American Society of Hematology Annual Meeting. 4 December 2018
BeiGene clinical data from the pivotal Phase II trial of its investigational anti-PD-1 antibody, tislelizumab, in Chinese patients with relapsed/refractory (R/R) classical Hodgkin’s lymphoma (cHL), at the Annual Meeting of the American Society of Hematology (ASH) yesterday. 4 December 2018
Daiichi Sankyo yesterday announced positive comprehensive analyses of overall survival from the pivotal QuANTUM-R Phase III study of single agent quizartinib compared to salvage chemotherapy in patients with FLT3-ITD acute myeloid leukemia (AML). 4 December 2018
Basel, Switzerland-based Roche has announced new Phase III data for Venclyxto (venetoclax) at the annual meeting of the American Society of Hematology. 4 December 2018
AstraZeneca and its hematology subsidiary Acerta Pharma have presented new, long-term follow-up results for Calquence (acalabrutinib) at the annual meeting of the American Society of Hematology. 4 December 2018
The Novartis drug Promacta (eltrombopag) improved outcomes for immune thrombocytopenia (ITP) patients compared to other second-line therapies. 3 December 2018
US biotech firm Seattle Genetics’ shares gained 2.75% to $64.3-in pre-market trading today, after it highlighted data from the ECHELON-1 Phase III clinical trial evaluating Adcetris (brentuximab vedotin) at the ASH meeting. 3 December 2018
China’s BeiGene on Saturday announced clinical data from two ongoing trials of its investigational Bruton’s tyrosine kinase (BTK) inhibitor, zanubrutinib, in patients with mantle cell lymphoma (MCL), at the 60th Annual Meeting of the American Society of Hematology (ASH), taking place December 1-4, 2018 in San Diego, USA. 3 December 2018
Gilead Sciences company Kite Pharma released positive two-year efficacy and safety data from the pivotal ZUMA-1 trial of Yescarta (axicabtagene ciloleucel) in patients with refractory large B-cell lymphoma. 3 December 2018
The US Food and Drug Administration on Wednesday approved Xospata (gilteritinib) tablets for the treatment of adult patients who have relapsed or refractory acute myeloid leukemia (AML) with a FLT3 mutation as detected by an FDA-approved test. 29 November 2018
US gene therapy company Rocket Pharmaceuticals yesterday announced that the US Food and Drug Administration has granted Regenerative Medicine Advanced Therapy (RMAT) and fast track designations to RP-L102, the company’s lentiviral vector (LVV)-based gene therapy for the treatment of Fanconi anemia (FA). 28 November 2018