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Pharmaceutical
US drugmaker Vertex Pharmaceuticals’ shares were unmoved on Friday, despite receiving two new regulatory authorizations. 23 December 2024
Pharmaceutical
The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has granted a conditional marketing authorization for Duvyzat (givinostat) to treat Duchenne muscular dystrophy (DMD). 22 December 2024
Pharmaceutical
The US Food and Drug Administration (FDA) has approved Tryngolza (olezarsen) as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS), a rare, genetic form of severe hypertriglyceridemia (sHTG) that can lead to potentially life-threatening acute pancreatitis (AP). 20 December 2024
Biotechnology
December 19, 2024
The US Food and Drug Administration (FDA) approved Ryoncil (remestemcel-L-rknd), from Australia’s Mesoblast, whose shares rocketed 54% to A$3.05 on the news. 19 December 2024
Pharmaceutical
Sweden-based biotech Hansa Biopharma has released promising full results from a Phase II study evaluating imlifidase, its IgG-cleaving enzyme, in severe Guillain-Barré syndrome (GBS). 18 December 2024
Biotechnology
Flagship Pioneering-founded genome engineering biotech Tessera Therapeutics today announced an investment of up to $50 million from the Bill & Melinda Gates Foundation to develop curative, globally accessible in vivo genetic therapies for sickle cell disease. 18 December 2024
Generics
Venture capital firm BC Partners has entered into an agreement under which the private equity business at Goldman Sachs Alternatives will acquire a majority stake in Netherlands-based Synthon, from funds advised by BC Partners. 17 December 2024
Pharmaceutical
Neurocrine Biosciences has secured US Food and Drug Administration approval for Crenessity (crinecerfont). 17 December 2024
Biotechnology
US health technology assessor the Institute for Clinical and Economic Review (ICER) as released a Final Evidence Report assessing the comparative clinical effectiveness of French drugmaker Pierre Fabre’s tabelecleucel (tab-cel) for the treatment of Epstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD). 17 December 2024
Pharmaceutical
Japan’s largest drugmaker, Takeda Pharmaceuticals, announced it is working with the European Medicines Agency (EMA) to voluntarily withdraw the marketing authorization of Alofisel (darvadstrocel), a treatment for complex perianal fistulas in patients with Crohn’s disease, in the European Union (EU). 16 December 2024
Pharmaceutical
Denmark-based diabetes and obesity giant Novo Nordisk says it plans to invest 8.5 billion Danish kroner ($1.2 billion) to establish a completely new production facility in Odense, Denmark. 16 December 2024
Biotechnology
Dutch biotech Pharming has announced a public cash offer to acquire all outstanding shares of Swedish firm Abliva for $66 million. 16 December 2024
Pharmaceutical
US antivirals major Gilead Sciences announced that Dr Dietmar Berger will join the company on January 2, 2025, as chief medical officer (CMO) and will become a member of Gilead’s senior leadership team. Dr Berger succeeds Dr Merdad Parsey. 13 December 2024
Pharmaceutical
UK-based Essential Pharma has announced the completion of a 900 million euros ($945 million) financing. 12 December 2024
Pharmaceutical
Swiss specialty pharmaceutical company Santhera Pharmaceuticals has received approval from the National Medical Products Administration in China for Agamree (vamorolone). 11 December 2024
Biotechnology
Netherlands-based Pharming Group today released positive top-line results of data from its Phase III clinical trial (NCT05438407) evaluating the investigational drug leniolisib, an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, in children aged 4 to 11 years with activated phosphoinositide 3-kinase delta syndrome (APDS). 11 December 2024
Biotechnology
Deal-making featured strongly among last week’s news, first with PCT Therapeutics inking an up to 2.9 billion collaboration with Swiss pharma giant Novartis on its PTC518 program for Huntington’s disease. Dutch firm Merus entered a licensing deal with Partner Therapeutics on the development of zenocutuzumab. Japan’s Takeda announced a deal with Keros Therapeutics for the development of myelofibrosis candidate elritercept. Also of note, Merus scored a first with US Food and Drug Administration (FDA) approval for its Bizengri for lung cancer. 8 December 2024
Biotechnology
Germany-headquartered CNS drug specialist Neuraxpharm today revealed that UK health technology assessor the National Institute for Health and Care Excellence (NICE) has issued a positive recommendation for the use of ublituximab (150mg concentrate for solution for infusion) in the treatment of relapsing-remitting multiple sclerosis (RRMS). 5 December 2024
Biotechnology
The global precision medicine market is projected to expand significantly, with an estimated growth of $66 billion between 2024 and 2028, according to Technavio. 5 December 2024
Pharmaceutical
US drugmaker Vertex Pharmaceuticals’ shares were unmoved on Friday, despite receiving two new regulatory authorizations. 23 December 2024
Pharmaceutical
The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has granted a conditional marketing authorization for Duvyzat (givinostat) to treat Duchenne muscular dystrophy (DMD). 22 December 2024
Pharmaceutical
The US Food and Drug Administration (FDA) has approved Tryngolza (olezarsen) as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS), a rare, genetic form of severe hypertriglyceridemia (sHTG) that can lead to potentially life-threatening acute pancreatitis (AP). 20 December 2024
Biotechnology
December 19, 2024
The US Food and Drug Administration (FDA) approved Ryoncil (remestemcel-L-rknd), from Australia’s Mesoblast, whose shares rocketed 54% to A$3.05 on the news. 19 December 2024
Pharmaceutical
Sweden-based biotech Hansa Biopharma has released promising full results from a Phase II study evaluating imlifidase, its IgG-cleaving enzyme, in severe Guillain-Barré syndrome (GBS). 18 December 2024
Biotechnology
Flagship Pioneering-founded genome engineering biotech Tessera Therapeutics today announced an investment of up to $50 million from the Bill & Melinda Gates Foundation to develop curative, globally accessible in vivo genetic therapies for sickle cell disease. 18 December 2024
Generics
Venture capital firm BC Partners has entered into an agreement under which the private equity business at Goldman Sachs Alternatives will acquire a majority stake in Netherlands-based Synthon, from funds advised by BC Partners. 17 December 2024
Pharmaceutical
Neurocrine Biosciences has secured US Food and Drug Administration approval for Crenessity (crinecerfont). 17 December 2024
Biotechnology
US health technology assessor the Institute for Clinical and Economic Review (ICER) as released a Final Evidence Report assessing the comparative clinical effectiveness of French drugmaker Pierre Fabre’s tabelecleucel (tab-cel) for the treatment of Epstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD). 17 December 2024
Pharmaceutical
Japan’s largest drugmaker, Takeda Pharmaceuticals, announced it is working with the European Medicines Agency (EMA) to voluntarily withdraw the marketing authorization of Alofisel (darvadstrocel), a treatment for complex perianal fistulas in patients with Crohn’s disease, in the European Union (EU). 16 December 2024
Pharmaceutical
Denmark-based diabetes and obesity giant Novo Nordisk says it plans to invest 8.5 billion Danish kroner ($1.2 billion) to establish a completely new production facility in Odense, Denmark. 16 December 2024
Biotechnology
Dutch biotech Pharming has announced a public cash offer to acquire all outstanding shares of Swedish firm Abliva for $66 million. 16 December 2024
Pharmaceutical
US antivirals major Gilead Sciences announced that Dr Dietmar Berger will join the company on January 2, 2025, as chief medical officer (CMO) and will become a member of Gilead’s senior leadership team. Dr Berger succeeds Dr Merdad Parsey. 13 December 2024
Pharmaceutical
UK-based Essential Pharma has announced the completion of a 900 million euros ($945 million) financing. 12 December 2024
Pharmaceutical
Swiss specialty pharmaceutical company Santhera Pharmaceuticals has received approval from the National Medical Products Administration in China for Agamree (vamorolone). 11 December 2024
Biotechnology
Netherlands-based Pharming Group today released positive top-line results of data from its Phase III clinical trial (NCT05438407) evaluating the investigational drug leniolisib, an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, in children aged 4 to 11 years with activated phosphoinositide 3-kinase delta syndrome (APDS). 11 December 2024
Biotechnology
Deal-making featured strongly among last week’s news, first with PCT Therapeutics inking an up to 2.9 billion collaboration with Swiss pharma giant Novartis on its PTC518 program for Huntington’s disease. Dutch firm Merus entered a licensing deal with Partner Therapeutics on the development of zenocutuzumab. Japan’s Takeda announced a deal with Keros Therapeutics for the development of myelofibrosis candidate elritercept. Also of note, Merus scored a first with US Food and Drug Administration (FDA) approval for its Bizengri for lung cancer. 8 December 2024
Biotechnology
Germany-headquartered CNS drug specialist Neuraxpharm today revealed that UK health technology assessor the National Institute for Health and Care Excellence (NICE) has issued a positive recommendation for the use of ublituximab (150mg concentrate for solution for infusion) in the treatment of relapsing-remitting multiple sclerosis (RRMS). 5 December 2024
Biotechnology
The global precision medicine market is projected to expand significantly, with an estimated growth of $66 billion between 2024 and 2028, according to Technavio. 5 December 2024
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