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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
Netherlands-based Pharming Group today announced it has entered into a definitive agreement with Swedish Orphan Biovitrum (“Sobi”) to reacquire the commercial rights to Pharming’s Ruconest (C1 esterase inhibitor [recombinant]) in all remaining territories, which includes all remaining European Union markets. 30 December 2019
Biotechnology
Sarepta has signed a licensing agreement providing Swiss pharma giant Roche with exclusive commercial rights to SRP-9001 (AAVrh74.MHCK7.micro-dystrophin), Sarepta’s investigational gene therapy for Duchenne muscular dystrophy (DMD), outside the USA. 23 December 2019
Biotechnology
In a surprise move, Ipsen chief executive David Meek has quit his position at the French drugmaker to take the helm at a small gene therapy startup, FerGene. 18 December 2019
Biotechnology
Cancer-focused Chinese biotech BeiGene today announced results from the Phase III ASPEN trial comparing its BTK inhibitor Brukinsa (zanubrutinib) to ibrutinib for the treatment of Waldenström’s macroglobulinemia (WM). 16 December 2019
Biotechnology
The US Food and Drug Administration has granted accelerated approval for its Vyondys 53 (golodirsen) injection to treat Duchenne muscular dystrophy (DMD) patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping. 13 December 2019
Biotechnology
Japanese drugmaker Kyowa Kirin says that Crysvita (burosumab) was launched in Japan on December 6, 2019. 11 December 2019
Biotechnology
Cambridge, UK-based artificial intelligence specialist Healx has launched a collaboration with Germany’s Boehringer Ingelheim to identify indications related to rare neurological diseases. 9 December 2019
Pharmaceutical
Since taking over as chief executive in July 2016, David Meek has overseen a period of near-seamless growth at French drugmaker Ipsen, but he was hit by his first major setback on Friday. 6 December 2019
Pharmaceutical
In her exclusive column for The Pharma Letter, this month, Dr Nicola Davies identifies progress being made within the therapeutic area of Klinefelter syndrome (KS), also known as 47,XXY or XXY. 6 December 2019
Generics
The US Food and Drug Administration has approved three applications for first generics of fingolimod capsules for the treatment of relapsing forms of multiple sclerosis (MS) in adult patients. 6 December 2019
Pharmaceutical
US rare and orphan diseases drug developer Cerecor has entered into a definitive merger agreement to acquire Aevi Genomic Medicine. 6 December 2019
Biotechnology
Australian plasma and cell and gene therapy specialist CSL Limited is steadily advancing its R&D pipeline and capabilities to deliver a highly differentiated product portfolio mix, addressing a broader range of patients’ unmet needs,” said head of Research & Development Dr William Mezzanotte today. 4 December 2019
Generics
Israeli generics giant Teva Pharmaceutical Industries plans to significantly diversify its business and to expand into new sales markets during the next several years, according to recent statements made by Kåre Schultz, chief executive of the company. 3 December 2019
Pharmaceutical
Swiss pharma giant Novartis set the scene in M&A news, announcing a $9.7 billion acquisition of US biotech firm The Medicines Company and its cholesterol-lowerer inclisiran. Clinical trials news featured ChemoCentryx, which saw its shares triple on Tuesday after reporting strong Phase III results with its ANCA vasculitis drug candidate avacopan. Last week also saw a second sickle cell disease drug approval from the US Food and Drug Administration, this time Global Blood Therapeutics’ Oxbryta (voxelotor). Meanwhile, Hutchison China MediTech, also known as Chi-Med, gained approval and price listing for its Elunate for advanced colorectal cancer in China. 1 December 2019
Pharmaceutical
Multinationals pharmaceutical companies have cut prices significantly for some of the bestselling drugs in China to get covered by China’s newly-updated reimbursement drug list, effective on January 1 ,2020, reports The Pharma Letter’s local correspondent, Wang Fangqing. 29 November 2019
Biotechnology
The US Food and Drug Administration has accepted for Priority Review the New Drug Application (NDA) for pemigatinib, a selective fibroblast growth factor receptor (FGFR) inhibitor, as a treatment for patients with previously treated, locally-advanced or metastatic cholangiocarcinoma with FGFR2 fusions or rearrangements. 28 November 2019
Biotechnology
Orchard Therapeutics has big ambitions for its role in pioneering some of the most advanced treatments in development today. 25 November 2019
Pharmaceutical
Regulatory news last week included a nice surprise for Swiss pharma giant Novartis with the earlier-than-expected US Food and Drug Administration approval for its Adakveo (crizanlizumab) for the treatment of a sickle cell disease complication. Also, Alnylam Pharmaceuticals gained FDA approval for its givosiran, now trade-named Givlaari, for acute hepatic porphyria. On the M&A front, Alkermes last Monday announced its first company acquisition in the past 20 years, agreeing to buy CNS specialist Rodin Therapeutics. Meantime, diabetes care giant Novo Nordisk inked a deal with US RNAi specialist Dicerna Pharmaceuticals on novel therapies for liver-related cardio-metabolic diseases involving an upfront payment of $175 million, and USA-based Karuna Therapeutics pleased investors with the release of strong Phase II data for its schizophrenia candidate KarXT. 24 November 2019
Pharmaceutical
EURORDIS-Rare Diseases Europe, an alliance of over 800 rare disease patient organizations, is today urging member states of the European Union to take off the table a proposal from the Presidency of the EU Council to withdraw Article 7 of the current proposal for European cooperation on health technology assessment (HTA). 22 November 2019
Pharmaceutical
The overlap between developing medicines for pediatric conditions and for rare diseases is something that impacts many drug companies, bringing opportunities and potential complexities. 21 November 2019
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