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Biotechnology
US drug developer SpringWorks Therapeutics today announced what it described as positive top-line results from its Phase IIb ReNeu trial evaluating mirdametinib. 16 November 2023
Biotechnology
The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) today granted conditional marketing authorization for Casgevy (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy, for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT), developed by USA-based Vertex Pharmaceuticals and Swiss firm CRISPR Therapeutics. 16 November 2023
Generics
The Russian Medical Research Company, which is a subsidiary of Swiss firm Genfa Global, is considering the production of Trilexa, which is a generic of Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor), a drug for treatment of cystic fibrosis from the USA’s Vertex Pharmaceuticals, The Pharma Letter’s local correspond reports. 15 November 2023
Pharmaceutical
The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has today extended the license of the cystic fibrosis medicines Kaftrio (ivacaftor, tezacaftor and elexacaftor) and Kalydeco (ivacaftor) for children aged two to five years old. 15 November 2023
Pharmaceutical
Autifony Therapeutics, an independent biotech company formed in 2011 as a spin-out from GSK, has announced a collaboration with Ireland-headquartered Jazz Pharmaceuticals. 14 November 2023
Biotechnology
The US Food and Drug Administration yesterday approved Japanese pharma major Takeda’s Adzynma (ADAMTS13, recombinant-krhn; TAK-755), adding to the approval a day earlier of the firm’s bowel cancer drug Fruzaqla (fruquintinib). 10 November 2023
Biotechnology
Belgium’s largest pharma company, UCB, has announced new long-term data from the Bimzelx (bimekizumab) Phase IIb study BE AGILE and its open label extension (OLE). 10 November 2023
Biotechnology
The UK's National Institute for Health and Care Excellence (NICE) has recommended Lamzede (velmanase alfa) in its final evaluation document as an option for treating the non-neurological signs and symptoms of mild to moderate alpha-mannosidosis in England and Wales. 9 November 2023
Biotechnology
The market for myasthenia gravis (MG), an autoimmune and neuromuscular disease characterized by muscle weakness and fatigue, is set to experience a compound annual growth rate (CAGR) of 8.3% across the seven major markets. 9 November 2023
Biotechnology
US biotech major Biogen today reported third quarter 2023 results and updated full year 2023 guidance that failed to impress investors, with its shares falling 4.7% to $235.34 in early trading’ 8 November 2023
Generics
Israeli generics giant Teva Pharmaceutical Industries has reported its latest quarterly financial results against a backdrop of war in its home region. 8 November 2023
Pharmaceutical
Italian pharma major Recordati saw its shares rise 3.6% to 45.57 euros yesterday, after it released a strong set of financial results for the first-nine months of 2023. 8 November 2023
Biotechnology
South Korea’s Chong Kun Dang Pharmaceutical's stock soared 26.1% to end at 128,000 won on Monday, on news of a deal with Swiss pharma giant Novartis. 7 November 2023
Biotechnology
Australian inflammatory diseases specialist Dimerix has received an initial payment of 6.5 million euros ($7 million) in line with the recently-announced license agreement with Advanz Pharma. 6 November 2023
Biotechnology
Working together through their trade body, the BioIndustry Association (BIA), a group of British rare disease drug developers is calling for the UK to lead greater collaboration between stakeholders, and uptake of best practice both in the UK and internationally, to improve access to new treatments for patients. 6 November 2023
Biotechnology
US RNAi therapy specialist Alnylam Pharmaceuticals saw its rise 4.2% to $168.29 on Friday, after it presented new 24-month results from the ILLUMINATE-C Phase III open-label study of lumasiran in patients with advanced primary hyperoxaluria type 1 (PH1), an inherited rare disease, at the American Society of Nephrology (ASN) Kidney week 2023. 6 November 2023
Biotechnology
Research news last week included US biotech Sarepta Therapeutics releasing mixed top-line result for its gene therapy Elevidys in Duchenne Muscular dystrophy (DMD). On the licensing front, Eli Lilly entered into a collaboration with US biotech Beam Therapeutics to acquire the latter’s opt-in right to Verve Therapeutics’ editing program for cardiovascular diseases. Additionally, US ophthalmic drug specialist Aldeyra Therapeutics signed an option agreement with AbbVie for its dry eye disease candidate reproxalap. Also of note, the US Food and Drug Administration (FDA) granted approval for Pfizer’s Penbraya, the first pentavalent vaccine for meningococcal disease in adolescents. 5 November 2023
Pharmaceutical
The American business of Israeli drugmaker Teva Pharmaceutical has announced new Huntington’s disease (HD) data to be presented at the ongoing Huntington Study Group annual meeting. 3 November 2023
Biotechnology
Independent French drugmaker Pierre Fabre has expanded a global partnership for Ebvallo (tabelecleucel) with US biotech Atara Biotherapeutics. 3 November 2023
Biotechnology
San Diego, USA-based biotech AnaptysBio’s ground breaking Phase III trial results for imsidolimab, a novel inhibitor, bring renewed hope to patients battling generalized pustular psoriasis (GPP). 3 November 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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