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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
Pharmaceutical
The National Institute for Health and Clinical Excellence (NICE), the health technology appraisal body in England and Wales, has issued its Final Appraisal Determination (FAD) recommending Takhzyro (lanadelumab) subcutaneous injection as an option for preventing recurrent attacks of hereditary angioedema (HAE). 18 September 2019
Pharmaceutical
With the news of data manipulation in Novartis’ preclinical trials of Zolgensma (onasemnogene abeparvovec-xioi), questions have arisen around the FDA’s opinions on data accuracy and integrity. Zolgensma, a gene therapy for spinal muscular atrophy (SMA) in children, is currently the most expensive drug on the market, with a single dose costing up to $2.1million.1,2 In her exclusive column for the Pharma Letter, Dr Nicola Davies explores the measures put in place to tackle data accuracy after the fallout of the Novartis scandal. 18 September 2019
Biotechnology
A Phase II trial flop for rare disease candidate ACE-083 has prompted Acceleron Pharma to abandon development of the program in facioscapulohumeral muscular dystrophy (FSHD). 17 September 2019
Pharmaceutical
Epidermolysis bullosa (EB) is a class of rare diseases that result in fragile skin prone to injury and blistering.1 The last week in October is EB Awareness Week – an apt time to build knowledge on this severe condition for which common subtypes are estimated to occur in 1 in every 50,000 births globally across all ethnicities and genders.1 17 September 2019
Pharmaceutical
Last month the independent Scottish Medicines Consortium assessed that Vertex Pharmaceuticals’ (Nasdaq: VRTX) ‘justification of the treatment’s [Orkambi (lumacaftor/ivacaftor) and Symkevi (tezacaftor/ivacaftor)] costs in relation to its health benefits was not sufficient’ - meaning the company was charging an unreasonably high price for its product. 16 September 2019
Biotechnology
US biotech major Biogen] has completed registration of Spinraza (nusinersen), one of the world’s most expensive drugs, for the treatment of spinal muscular atrophy (SMA) in Russia, according to the Russian Ministry of Health, reports The Pharma Letter’s local correspondent. 16 September 2019
Pharmaceutical
Longer-term data from the Phase III open-label extension studies OPERA I, OPERA II and ORATORIO show a positive impact for people treated with Ocrevus (ocrelizumab) continuously for six years or more. 13 September 2019
Biotechnology
Roche today presented full pivotal Phase III study results for satralizumab as a monotherapy for neuromyelitis optica spectrum disorder (NMOSD), a rare, debilitating central nervous system disease. 12 September 2019
Biotechnology
Johnson & Johnson subsidiary has announced positive results from the Phase III OPTIMUM study for ponesimod, an investigational selective S1P1 receptor modulator, showing superior efficacy on the primary endpoint and most secondary endpoints compared to Sanofi’s Aubagio (teriflunomide) 14mg in adults with relapsing multiple sclerosis (MS). 12 September 2019
Biotechnology
Cambridge, USA-based biotech Biogen has presented 10-year data on Tecfidera (dimethyl fumarate), demonstrating the long-term benefits for people with multiple sclerosis (MS). 11 September 2019
Pharmaceutical
At its Capital Markets Day, German life sciences and pharma firm Merck KGaA said it expects to achieve sustainable profitable growth in all three business sectors in the coming years. 11 September 2019
Pharmaceutical
A new licensing deal between Alexion Pharmaceuticals and Eidos Therapeutics will see the development of rare disease candidate AG10 in Japan. 10 September 2019
Pharmaceutical
The US Food and Drug Administration on Friday approved Ofev (nintedanib) capsules to slow the rate of decline in pulmonary function in adults with interstitial lung disease associated with systemic sclerosis or scleroderma, called SSc-ILD. 7 September 2019
Biosimilars
Swiss pharma giant Novartis’ Sandoz biosimilars division today announced that it has entered into a global commercialization agreement with Poland-based Polpharma Biologics for a proposed natalizumab biosimilar. 3 September 2019
Pharmaceutical
The Russian government has announced its plans to increase state funding for the purchases of orphan drugs and create conditions for the beginning of their production within the country, according to recent statements from the state press-service, reports The Pharma Letter’s local correspondent. 2 September 2019
Biotechnology
Shares of US biotech firm Alexion Pharmaceuticals crashed 8% in pre-market trading on Friday, reacting to the news that peer Amgen is challenging of three patents covering top seller Soliris (eculizumab). 31 August 2019
Pharmaceutical
By Dr Nicola Davies
September 7th is World Duchenne Awareness Day and the perfect opportunity to delve into the details of Duchenne muscular dystrophy (DMD) and pharma’s role in helping individuals living with this condition. 30 August 2019
Biotechnology
The US Food and Drug Administration has granted Breakthrough Therapy designation for nirogacestat, an oral, selective, small molecule, gamma-secretase inhibitor, for the treatment of adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis, says USA-based biopharma firm SpringWorks Therapeutics. 29 August 2019
Biotechnology
Zogenix saw its stock drop as much as 16% on Tuesday, after it announced an acquisition of a privately held California start-up launched just 10 months ago with $30 million Series A investment led by F-Prime Capital Partners and OrbiMed. 28 August 2019
Biotechnology
ReveraGen BioPharma today announced positive study data with vamorolone in patients with Duchenne muscular dystrophy (DMD). 27 August 2019
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