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Pharmaceutical
For Fragile X Awareness Month, we reflect on the current and future treatment landscape. 28 July 2022
Pharmaceutical
Russia plans to take additional measures to speed registration of new drugs in the domestic market – in a move, that will allow the country to avoid a potential shortage caused by sanctions and their consequences, reports The Pharma Letter’s local correspondent. 27 July 2022
Biotechnology
The US Food and Drug Administration (FDA) has accepted a New Drug Application (NDA) for tofersen, an investigational drug for superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS). 26 July 2022
Pharmaceutical
Netherlands-based specialty pharma firm Pleco Therapeutics today announced its expansion, with the incorporation if a US subsidiary, Pleco Therapeutics USA. 26 July 2022
Biosimilars
Sandoz announced today that the US Food and Drug Administration (FDA) has accepted its biologics license application (BLA) for a proposed first-of-a-kind biosimilar natalizumab, developed by Polpharma Biologics. 25 July 2022
Biotechnology
The European Medicines Agency’s human medicines committee (CHMP) recommended 10 novel medicines for approval at its July 2022 meeting. 22 July 2022
Biotechnology
CAMP4 Therapeutics, a biotech promising to harness the power of RNA to restore healthy protein expression, has announced the close of a $100 million Series B financing round, led by Enavate Sciences. 21 July 2022
Biotechnology
Avista Therapeutics, which recently launched as a spin-out of the University of Pittsburgh Medical Center (UPMC), aims to develop innovative gene therapies for rare ophthalmic conditions. The new company today announced a partnership with Swiss pharma giant Roche to develop novel AAV gene therapy vectors for the eyes. 21 July 2022
Biotechnology
New Jersey, USA-based rare disease drug developer PTC Therapeutics today announced that Upstaza (eladocagene exuparvovec) was granted marketing authorization by the European Commission. 20 July 2022
Biotechnology
Unofficial American cost-effectiveness watchdog the Institute for Clinical and Economic Review (ICER) has published a Final Evidence Report on betibeglogene autotemcel for the treatment of beta thalassemia. 20 July 2022
Biotechnology
US biotech major Biogen today reported second quarter 2022 financial results. 20 July 2022
Biotechnology
Along with providing a program update on GTX-102 for the treatment of Angelman syndrome (AS), Ultragenyx Pharmaceuticals today announced it will acquire GeneTx Biotherapies, its partner on the candidate. 19 July 2022
Biotechnology
Swedish rare disease specialist Hansa Biopharma has entered into an agreement with USA-based NovaQuest Capital Management, securing $70 million in non-dilutive financing, with the news propelling Hansa’s shares 30% higher to 72.70 kronor. 19 July 2022
Pharmaceutical
The UK’s National Institute for Health and Care Excellence (NICE) has issued guidance that recommends Imcivree (setmelanotide) as an option for treating hyperphagia and obesity and controlling hunger caused by pro-opiomelanocortin (POMC) deficiency, including proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency in people six years of age and over. 18 July 2022
Pharmaceutical
The European Commission (EC) has granted conditional marketing authorization for Kinpeygo (budesonide) for the treatment of primary immunoglobulin A (IgA) nephropathy (IgAN) in adults at risk of rapid disease progression with a urine protein-to-creatinine ratio (UPCR) ≥1.5 g/gram. 16 July 2022
Biotechnology
Clene Nanomedicine, a part of Clene Inc, has reported significantly improved survival in amyotrophic lateral sclerosis (ALS) patients initially treated with CNM-Au8 compared to initially randomized placebo treated participants during the long-term open-label extension of its RESCUE-ALS trial 15 July 2022
Biosimilars
Poland-based drugmaker Polpharma Biologics today announced that the European Medicines Agency (EMA) has accepted the first-ever Marketing Authorization Application (MAA) for biosimilar natalizumab, a proposed biosimilar to branded multiple sclerosis treatment Tysabri. 15 July 2022
Biotechnology
Agios Pharmaceuticals, a leader in the field of cellular metabolism pioneering therapies for genetically defined diseases, today announced that effective August 8, 2022, Jackie Fouse will transition to the role of chairwomen of the board of directors and Brian Goff will assume the role of chief executive officer. 13 July 2022
Biosimilars
A proposed biosimilar to Soliris (eculizumab) has shown clinical equivalence in efficacy and safety in people with paroxysmal nocturnal hemoglobinuria (PNH). 12 July 2022
Pharmaceutical
Russia plans to speed registration of Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor), an expensive drug from the USA’s Vertex Pharmaceuticals, which is indicated for the treatment of cystic fibrosis. 9 July 2022
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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