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Biotechnology
Japanese drugmaker Chugai, which is majority owned by Roche, On Friday revealed that Zenyaku Kogyo has obtained regulatory approval from the Ministry of Health, Labor and Welfare (MHLW), for an additional dosage and administration of an anti-CD20 monoclonal antibody Rituxan (rituximab) intravenous injection 100mg and 500mg, which is co-marketed by both companies, for chronic idiopathic thrombocytopenic purpura (ITP) in children. 23 November 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
Biotechnology
US biotech Regenxbio announced a new exclusive, worldwide license agreement with Ultragenyx Pharmaceutical, extending the companies’ existing gene therapy partnership announced in October 2018. 1 April 2020
Biotechnology
The US Food and Drug Administration has dropped an investigation into Novartis company AveXis, related to the novel spinal muscular atrophy (SMA) Zolgensma (onasemnogene abeparvovec-xioi). 1 April 2020
Biotechnology
Swiss pharma giant Novartis recently announced new anticipated long-term positive data for its one-time infusion gene therapy, Zolgensma (onasemnogene abeparvovec-xioi), which is set to help the drug generate global sales of $2.5 billion by 2025 and lead the spinal muscular atrophy (SMA) market, says analyst. 30 March 2020
Pharmaceutical
Deal-making news last week featured Bayer’s announcement on Monday that it is teaming up with India’s Curadev to use the latter’s STING antagonists to develop therapies across a range of indications. Also, Silence Therapeutics has inked a deal with AstraZeneca worth up to $400 million on siRNA therapeutics, including SLB360. Among research news, Novavax presented new data on its flu vaccine candidate NanoFlu that compared it with Sanofi’s Fluzone. Regulatory news included Food and Drug Administration approval for Bristol-Myers Squibb’s multiple sclerosis drug Zeposia. Also, in an unusual move, Gilead Sciences asked the FDA to rescind Orphan Drug designation for its anti-viral candidate remdesivir, which is now been investigating as a possible COVID-19 treatment. 29 March 2020
Pharmaceutical
At its March 23-26 meeting, the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) recommended eight medicines for approval at its March 2020 meeting. 27 March 2020
Biotechnology
Japan’s JCR Pharmaceuticals has reached an agreement to acquire ArmaGen, a privately held US biopharmaceutical company, along with all its assets. 27 March 2020
Biotechnology
Oxford University biotech spinout Evox Therapeutics has entered into a rare disease-focused partnership with Japan’s largest drugmaker Takeda Pharmaceutical. 27 March 2020
Biotechnology
The New England Journal of Medicine (NEJM) has published results from BELIEVE, the pivotal Phase III study evaluating the safety and efficacy of Reblozyl (luspatercept-aamt) for the treatment of anemia in adults with beta thalassemia who require regular red blood cell (RBC) transfusions. 26 March 2020
Pharmaceutical
Italian drugmaker Recordati today announced the submission of the Japanese New Drug Application (JNDA) to the Ministry of Health, Labor and Welfare (MHLW) seeking marketing approval for osilodrostat, an oral inhibitor of 11-beta-hydroxylase for the treatment of patients with Cushing’s syndrome (CS). 26 March 2020
Biotechnology
Ipsen shares began trading around 2% higher on Thursday morning, after the French biotech announced today it would restart the global Phase III MOVE trial, in people over the age of 14. 26 March 2020
Biotechnology
In the USA, approximately 300,000 infants are born with sickle cell disease (SCD) each year.1 In this article, Dr Nicola Davies discusses how SCD is treated and what solutions are on the horizon. 13 March 2020
Biotechnology
UCB has announced the initiation of a Phase III clinical trial evaluating the efficacy and safety of dapirolizumab pegol (DZP) as an add-on treatment to standard of care (SoC) medication for lupus. 12 March 2020
Biotechnology
Shares of Israeli biotech firm Protalix BioTherapeutics, which are also traded in New York, were up 10.6% at $2.61 in morning activity, after the company said it has successfully secured securities purchase agreements to raise $43.7 million through a private equity investment (PIPE) in the company. 12 March 2020
Biotechnology
The US Food and Drug Administration on Monday approved Ofev (nintedanib) oral capsules to treat patients with chronic fibrosing (scarring) interstitial lung diseases (ILD) with a progressive phenotype (trait). 10 March 2020
Biotechnology
Alessia Deglincerti, Frank Borowsky and Rachel Laing from consulting firm Bionest Partners, explore in an Expert View how the development of spinal muscular atrophy (SMA) therapies could provide a blueprint for genetic diseases. 9 March 2020
Pharmaceutical
The US Food and Drug Administration on Friday approved Isturisa (osilodrostat) oral tablets for adults with Cushing’s disease who either cannot undergo pituitary gland surgery or have undergone the surgery but still have the disease. 7 March 2020
Pharmaceutical
Osaka, Japan-headquartered Shionogi has moved a step closer to taking full ownership of rare disease specialist and co-developer Tetra Therapeutics. 6 March 2020
Biotechnology
US biotech Stealth BioTherapeutics has revealed that the US Food and Drug Administration has granted Rare Pediatric Disease (RPD) designation for elamipretide for the treatment of Barth syndrome, an ultra-rare genetic condition. 4 March 2020
Biotechnology
Following a positive European Medicines advisory panel recommendation at the end of January, RNAi therapeutics company Alnylam Pharmaceuticals yesterday announced that the European Commission (EC) has granted marketing authorization for Givlaari (givosiran), an injection for subcutaneous use targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyria (AHP) in adults and adolescents aged 12 years and older. 4 March 2020
Biotechnology
The US Food and Drug Administration has granted Breakthrough Therapy designation to Esbriet (pirfenidone) for adults with unclassifiable interstitial lung disease (uILD) 3 March 2020
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