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Pharmaceutical
The Russian government is considering introducing a risk-sharing mechanism for public procurement of expensive innovative drugs. 13 July 2023
Biotechnology
US gene therapy company Avrobio today announced that it has completed a review of its business, including the status of its programs, resources, and capabilities, joining a growing list of biotechs that have recently announced similar actions. 12 July 2023
Biotechnology
Yesterday after market close, US genetic medicines company 4D Molecular Therapeutics announced a license agreement with Japanese drug major Astellas Pharma. 11 July 2023
Biotechnology
US gene editing company Precision BioSciences and Prevail Therapeutics, a wholly-owned subsidiary of Eli Lilly, have amended and restated the development and license agreement that they signed in November 2020. 10 July 2023
Pharmaceutical
The US Food and Drug Administration (FDA) has removed the partial clinical hold on the TakeAim Leukemia Phase I/II study of emavusertib for the treatment of hematologic malignancies, conducted by US biotech Curis. 7 July 2023
Pharmaceutical
Privately-held Italian drugmaker Chiesi Farmaceutici has appointed Alessandro Chiesi as chairman of the Chiesi Group. Additionally, it said that Maria Paola Chiesi becomes vice chair. 7 July 2023
Biotechnology
The European Commission has granted approval for the label extension of Orkambi (lumacaftor/ivacaftor), from US biopharma firm Vertex Pharmaceuticals, for the treatment of children with cystic fibrosis (CF) ages 1 to 5 July 2023
Biotechnology
For many years, Britain’s pharma largest company AstraZeneca has maintained a strong presence in China, and this approach looks set to pay off. 4 July 2023
Biotechnology
German family-owned pharma major Boehringer Ingelheim today presented new late-breaking data from the EFFISAYIL trial showing that patients receiving the highest dose of spesolimab experienced significantly reduced generalized pustular psoriasis (GPP) flares by 84% over 48 weeks compared to placebo (hazard ratio 0.157; 95% confidence interval 0.05–0.54; p=0.0005). 4 July 2023
Generics
Swiss generic drugmaker Dipharma today announced that its product Sapropterin Dipharma is now available in main European countries including in Switzerland. 3 July 2023
Biotechnology
News coming out of Cure SMA meeting on new 36-month extension data from US biotech Scholar Rock’s Phase II TOPAZ clinical trial reinforces long-term substantial and sustained improvement of motor function in patients with non-ambulatory spinal muscular atrophy (SMA) treated with apitegromab. 3 July 2023
Biotechnology
Gaining US Food and Drug Administration (FDA) approval for its news generalized myasthenia gravis (gMG) drug was positive news for Belgium’s largest drug maker UCB, but the company will face challenges 1 July 2023
Biotechnology
Dublin, Ireland-headquartered protein specialist Prothena has published results from the Phase III VITAL trial in a journal of the American Society of Hematology (ASH). 30 June 2023
Pharmaceutical
To ensure the effective implementation of India's National Rare Disease Policy-2021, the Delhi High Court has formed a five-member National Rare Diseases Committee to ensure that the policy benefits reach the ultimate patients. 30 June 2023
Pharmaceutical
Italy’s privately-held Italfarmaco Group has taken another pivotal step towards approval of its proprietary histone deacetylase (HDAC), givinostat, in Duchenne muscular dystrophy (DMD). 29 June 2023
Pharmaceutical
The US Food and Drug Administration’s (FDA) Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) voted in favor of investigational palovarotene as an effective treatment, with a positive risk-benefit profile, for people living with the ultra-rare bone disease, fibrodysplasia ossificans progressiva (FOP). 29 June 2023
Pharmaceutical
US pharma giant Pfizer says the US Food and Drug Administration (FDA) has approved Ngenla (somatrogon-ghla), a once-weekly, human growth hormone analog indicated for treatment of pediatric patients aged three years and older who have growth failure due to inadequate secretion of endogenous growth hormone. 28 June 2023
Pharmaceutical
Mid-size French drugmaker Ipsen is hoping to be lucky third time around in its bid to gain US Food and Drug Administration (FDA) approval for palovarotene for the prevention of heterotopic ossification in patients with fibrodysplasia ossificans progressiva (FOP). 27 June 2023
Biotechnology
The US Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) for fidanacogene elaparvovec for the treatment of adults with hemophilia B, said US pharma giant Pfizer. 27 June 2023
Biotechnology
A new Pompe disease therapy has been approved by the European Medicines Agency, with Amicus Therapeutics picking up authorization for the enzyme stabilizer Opfolda (miglustat). 27 June 2023
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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