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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
Biotechnology
In a busy day for the Japanese regulator, the Japanese Ministry of Health, Labor and Welfare (MHLW) has cleared three new medicines from European drugmakers for marketing in the country. 27 September 2022
Biotechnology
In what is the biggest ‘whistleblower’ settlement, US major Biogen has agreed to pay $900 million to resolve allegations that it caused the submission of false claims to Medicare and Medicaid by paying kickbacks to physicians to induce them to prescribe its multiple sclerosis drugs. 27 September 2022
Biotechnology
Developed by UK pharma major AstraZeneca, Ultomiris (ravulizumab) has been approved in Europe for a new indication. 23 September 2022
Biotechnology
UK health technology assessor the National Institute for Health and Care Excellence (NICE) has issued a final appraisal document (FAD) recommending Brukinsa (zanubrutinib). 21 September 2022
Pharmaceutical
The US Court of Appeals for the Federal Circuit (CAFC) has denied its petition to rehear the negative decision regarding the validity of US Patent No 9,187,405, covering a dosing regimen for 0.5mg of its blockbuster multiple sclerosis drug Gilenya (fingolimod), Swiss pharma giant Novartis announced this morning. 21 September 2022
Biotechnology
In an all-stock deal valued at around $53 million, Rocket Pharmaceuticals is to acquire fellow US biotech firm Renovacor. 20 September 2022
Pharmaceutical
The European Commission (EC) has granted marketing authorization for Nulibry (fosdenopterin) for Injection as the first therapy for the treatment of patients with molybdenum cofactor deficiency (MoCD) Type A. 20 September 2022
Biotechnology
Danish diabetes care giant Novo Nordisk has entered into a definitive agreement to acquire Forma Therapeutics, a US clinical-stage biopharmaceutical company focused on transforming the lives of patients with sickle cell disease (SCD) and rare blood disorders, whose shares rocketed up 48% to $19.84 on the news. 19 September 2022
Biotechnology
US biotech bluebird bio has had a rocky ride in gaining US approval for its Skysona (elivaldogene autotemcel), but the US Food and Drug Administration (FDA), having earlier this year extended the review period for the product, on Friday granted authorization for the gene therapy. 19 September 2022
Biotechnology
Last month, Belgo-Dutch autoimmune diseases specialist argenx announced the European Commission (EC) approval of Vyvgart (efgartigimod alfa-fcab) as an add-on to standard therapy for adults with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive. 19 September 2022
Biotechnology
Intellia Therapeutics and Regeneron Pharmaceuticals presented positive interim results from a well-watched Phase I trial early Friday. 16 September 2022
Biotechnology
The European Medicines Agency’s (EMA) human medicines committee (CHMP) today released its list of recommended novel medicines and one biosimilar for approval following its September 2022 meeting. 16 September 2022
Biotechnology
Japanese medium-sized drugmaker Asahi Kasei Pharma yesterday revealed it has acquired the exclusive distribution rights in Japan for two products developed by Swedish Orphan Biovitrum. 16 September 2022
Biotechnology
Looking to cement its lead in the treatment of rare blood disorders, AstraZeneca has lifted the lid on positive results for a new add-on therapy for its blockbuster C5 inhibitor franchise. 16 September 2022
Biotechnology
Privately-held Swiss drug developer Neovii Pharmaceuticals has entered into an agreement with a unit of Shanghai Fosun Pharmaceutical, granting the latter an exclusive license to develop and commercialize Neovii's Grafalon (rabbit anti-human T-lymphocyte globulin, ATLG) in Chinese Mainland, Hong Kong SAR, Macau SAR and Taiwan region. 15 September 2022
Pharmaceutical
Coinciding with the 50th anniversary of its Lessines, Belgium, site, Japanese pharma major Takeda said it is investing close to 300 million euros ($304 million) in a new state-of-the-art production facility for the manufacturing of plasma-derived therapies for people with rare and complex chronic diseases. 15 September 2022
Biotechnology
Positive news from the medicines regulator yesterday saw US clinical-stage biotech Larimar Therapeutics’ shares close up 15.5% at $3.64. 15 September 2022
Biotechnology
Spanish CNS specialist Minoryx Therapeutics said today it has filed a Marketing Authorization Application (MAA) for its lead candidate leriglitazone to the European Medicines Agency (EMA) for the treatment of adult male patients with X-linked adrenoleukodystrophy (X-ALD). 14 September 2022
Pharmaceutical
Privately-held US developer of healthcare therapies Nacuity Pharmaceuticals and Icelandic firm Arctic Therapeutics International have entered into an exclusive licensing agreement and strategic partnership for the development and commercialization of NPI-001 in Iceland for the treatment of hereditary cystatin C amyloid angiopathy (HCCAA). 14 September 2022
Pharmaceutical
The USA’s medicines cost-effectiveness watchdog the Institute for Clinical and Economic Review (ICER) today released a Final Evidence Report assessing the comparative clinical effectiveness and value of treatment of amyotrophic lateral sclerosis (ALS). 13 September 2022
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