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Biotechnology
US biopharma company Vertex Pharmaceuticals showcased a year of robust growth and significant progress in its latest earnings call. 6 February 2024
Pharmaceutical
US health technology assessor the Institute for Clinical and Economic Review (ICER) has posted its revised Evidence Report assessing the comparative clinical effectiveness and value of iptacopan, from Novartis and danicopan from AstraZeneca’s rare diseases unit Alexion, for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). 2 February 2024
Pharmaceutical
Positive high-level results from the Japan Phase III trial of acoramidis in transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) showed consistency to those in the global BridgeBio Pharma ATTRibute-CM Phase III trial, including survival, cardiac-related hospitalizations and other measures of improved functions and quality of life, at 30 months. 2 February 2024
Pharmaceutical
Takeda logged a sales rise of 4.6% in April-December 2023 to 3,212.9 billion yen ($21.7 billion). 2 February 2024
Pharmaceutical
French pharma major Sanofi was trading 4% lower late Thursday after presenting its fourth quarter and full year 2023 financial results. 1 February 2024
Biotechnology
Shares of Swiss pharma giant Roche were down nearly 4% at 370.55 francs today, after it released financial results for full-year 2023. 1 February 2024
Biosimilars
Sandoz, the recent generic and biosimilar medicines spin out from Novartis , today announced the launch of Tyruko (natalizumab) in Germany from February 1. 31 January 2024
Biotechnology
Chicago, USA-based biotech COUR Pharmaceuticals has raised $105 million in a series A financing round, co-led by Lumira Ventures and Alpha Wave Ventures. 31 January 2024
Biotechnology
US gene therapy company Avrobio and privately-held biotech Tectonic Therapeutic today announced that the companies have entered into a definitive merger agreement to combine in an all-stock transaction. 31 January 2024
Biotechnology
Japan has recently approved Dutch immunology specialist argenx’ (Euronext: ARGX) Vyvdura (efgartigimod alfa and hyaluronidase-qvfc) subcutaneous (SC) injection for the treatment of adult patients with generalized myasthenia gravis (gMG), who do not have sufficient response to steroids or non-steroidal immunosuppressive therapies (ISTs). 31 January 2024
Biotechnology
US precision genetic medicines firm Sarepta Therapeutics has announced positive data from Part B of the MOMENTUM study (Study SRP-5051-201), a global, Phase II, multi-ascending dose clinical trial of SRP-5051 (vesleteplirsen) that enrolled patients aged eight to 21 years. 30 January 2024
article
Following on from its recent approval of Hyqvia (immune globulin) in Europe, Japanese pharma major Takeda has picked up a US approval for another CIDP product. 30 January 2024
Pharmaceutical
Takeda has secured European approval for Hyqvia (immune globulin) as maintenance therapy for people with chronic inflammatory demyelinating polyneuropathy (CIDP). 29 January 2024
Biotechnology
Shares of US clinical-stage biotech firm Vera Therapeutics leapt more than 19% to $30.18, after the company announced positive 72-week data from the open label extension (OLE) period of its Phase IIb ORIGIN clinical trial of atacicept in participants with IgA nephropathy (IgAN). 29 January 2024
Pharmaceutical
The European Medicines Agency’s (EMA) human medicines committee (CHMP) recommended two novel medicines for approval at its January 2024 meeting, but rejected two others. 26 January 2024
Biotechnology
French pharma major Sanofi has entered into a definitive agreement to acquire US biotech Inhibrx following the spin-off of non-INBRX-101 assets into ‘New Inhibrx’. 23 January 2024
Pharmaceutical
Ionis Pharmaceuticals yesterday announced positive top-line results for the Phase III OASIS-HAE study of donidalorsen in people with hereditary angioedema (HAE), with the news sending the US RNA-targeted drug developer’s shares up more than 5% to $53.58. 23 January 2024
Biotechnology
Last Tuesday, US biotech Aclaris Therapeutics announced that its chief executive would be stepping down and that the company is undertaking a review of its business and development of its dermatology candidates ATI-1777 and ATI-2138. US biotech Allakos suffered a further set back with its lirentelimab in trials for dermatitis and urticaria, leading to a decision to discontinue development. US biotech BridgeBio Pharma has agreed a deal to add up to $1.25 billion to its cash pile in a bid to progress its cardiovascular candidate acoramidis. Dutch biotech argenx gained Japanese approval for its myasthenia gravis drug Vyvdura/Vyvgart. 21 January 2024
Pharmaceutical
Medullary thyroid carcinoma (MTC) is a rare neuroendocrine condition that develops in the parafollicular C cells of the thyroid gland.1 It makes up 3%-5% of all thyroid cancers, with a 10-year mortality rate of about 50%.2 20 January 2024
Pharmaceutical
Voydeya (danicopan) has been approved in Japan for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). 19 January 2024
Pharmaceutical
Swiss pharma giant Novartis today announced an upgrade to its mid-term guidance, in advance of its Meet Novartis Management event for investors and analysts in London. 21 November 2024
Biotechnology
US biotech major Amgen has announced that Dr Howard Chang will join the company as senior vice president of research, effective December 16, 2024. 21 November 2024
Biotechnology
Australia’s CSL Limited plans to shut down its Californian R&D facility focused on cell and gene therapies by January 2025, signaling a shift away from ex vivo lentiviral-based technology. 19 November 2024
Biotechnology
Cellular immunotherapy specialist Fate Therapeutics soared as much as 73%, and closed up 12.45 at $2.27 yesterday, after it released early-stage data on its FT819 in lupus. 19 November 2024
Pharmaceutical
US antiviral giant Gilead Sciences on Friday unveiled two-and-a-half-year interim analysis from the ongoing Phase III ASSURE study of its Livdelzi (seladelpar). 16 November 2024
Pharmaceutical
French drugmaker Ipsen announced late-breaking data for Iqirvo (elafibranor 80mg tablets) from an interim analysis of the ongoing open-label extension of the Phase III ELATIVE study at the American Association for the Study of Liver Disease (AASLD) congress. 16 November 2024
Biotechnology
Oxford, UK-based rare respiratory diseases focused AlveoGene today revealed it has been granted a Rare Pediatric Disease designation (RPDD) by the US Food and Drug Administration (FDA) for AVG-002, its novel, inhaled gene therapy for lethal neonatal surfactant protein B (SP-B) deficiency. 15 November 2024
Biotechnology
Shares of US genetic meds company MeiraGTx Holdings were up 7% at $7.12 in early trading after it announced important developments along with its third-quarter 2024 financials, reporting a $0.54 per share loss. 13 November 2024
Biotechnology
Dutch firm Argenx and China’s Zai Lab have received an approval from the National Medical Products Administration (NMPA) for Vyvgart Hytrulo (efgartigimod alfa). 13 November 2024
Pharmaceutical
AstraZeneca and Merck & Co announced that their jointly developed drug Koselugo (selumetinib) has shown positive outcomes in the KOMET Phase III trial. 12 November 2024
Biotechnology
Geron Corporation, a biopharma company focused on telomerase inhibition therapies, has announced a $375 million financing agreement aimed at supporting the commercial rollout of Rytelo (imetelstat). 11 November 2024
Pharmaceutical
The US Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program, according to Sarah Wicks and James Valentine at US law firm Hyman, Phelps & McNamara. 11 November 2024
Biotechnology
Sarepta Therapeutics has announced it will cease development of SRP-5051, an experimental drug for Duchenne muscular dystrophy, citing safety issues, feedback from the Food and Drug Administration, and the shifting Duchenne treatment landscape. 11 November 2024
Pharmaceutical
Research results featured heavily in the news last week, first with Viking Therapeutics releasing promising early-stage data for its investigational medicine VK2738 at the ObesityWeek meeting. US biotech Beam Therapeutics released early-stage data on its sickle cell disease candidate BEAM-101. Also of note, Arcus Biosciences and Gilead Sciences announced promising Phase III results for their TIGIT inhibitor domvanalimab in combination with zimberelimab. Denmark’s Novo Nordisk released financial results that attracted interest. 10 November 2024
Pharmaceutical
Italian pharma major Recordati saw its shares dip 3.75% to 50.10 euros this morning, despite posting a set of strong financial results. 8 November 2024
Biotechnology
Asklepios BioPharmaceutical (AskBio) today announced that AB-1003 (also known as LION-101) has received rare pediatric disease designation and orphan-drug designation from the US Food and Drug Administration (FDA) for the treatment of limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). 7 November 2024
Biotechnology
The global market for Duchenne muscular dystrophy (DMD) therapeutics is projected to grow by $4.4 billion between 2024 and 2028, fueled by artificial intelligence and strong demand for new treatments. 7 November 2024
Pharmaceutical
US drugmaker Sarepta Therapeutic saw its shares gain 2.4% to $127.97 as it reported financial results for the third quarter 2024. 7 November 2024
Pharmaceutical
USA-based Acadia Pharmaceuticals has entered into a definitive asset purchase agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for $150 million upon the closing of the transaction. 6 November 2024
Biotechnology
UK health technology assessor the National Institute for Health and Care Excellence (NICE) issued final guidance recommending reimbursement of Ayvakyt (avapritinib) from USA-based Blueprint Medicines. 6 November 2024
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