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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
Biotechnology
People with eosinophilic granulomatosis with polyangiitis (EGPA) could benefit from a convenient monthly injection of Fasenra (benralizumab), according to new data. 12 September 2023
Biotechnology
Last week, the USA’s Seagen and Danish partner Genmab released positive new Phase III data on their Tivdak in the treatment of cervical cancer. Also, US clinical-stage biotech CymaBay Therapeutics released positive Phase II results for its seladelpar in biliary cholangitis. On the regulatory front, the US Food and Drug Administration (FDA) issued a complete response letter (CRL) on UK pharma major AstraZeneca’s supplemental biologic license application (sBLA) for its rare disease drug Ultomiris. Meantime, US clinical-stage biopharma Nurix Therapeutics revealed a collaboration with Seagen to develop degrader-antibody conjugates for cancer 10 September 2023
Biotechnology
US clinical-stage biotech Verge Genomics has announced a multi-target collaboration with Alexion, UK pharma major AstraZeneca’s Rare Disease unit to identify novel drug targets for rare neurodegenerative and neuromuscular diseases. 9 September 2023
Biotechnology
US commercial stage precision-care company Octave Bioscience has announced plans to conduct an observational, real-world study with biotech major Biogen as the sponsor. 9 September 2023
Biotechnology
The US Food and Drug Administration (FDA) has issued a complete response letter (CRL) regarding the supplemental Biologics License Application (sBLA) for long-acting C5 complement inhibitor Ultomiris (ravulizumab-cwvz). 6 September 2023
Pharmaceutical
Italy’s privately-held drug developer Italfarmaco Group today revealed that its Marketing Authorization Application (MAA) for Givinostat as a potential treatment for Duchenne muscular dystrophy (DMD) has been submitted to the European Medicine Agency. 5 September 2023
Pharmaceutical
Mid-sized French drugmaker Ipsen today revealed the appointment of Christelle Huguet as executive vice president, head of research and development. 4 September 2023
Biotechnology
Last week saw US biotech major Amgen reach a settlement with the Federal Trade Commission (FTC) regarding competition issues with its $27.8 billon proposed acquisition of Horizon Therapeutics, with particular issues concerning the potential bundling of the latter’s Krystexxa and Tepezza products. BridgeBio Pharma released new Phase III data on its acoramidis in ATTR cardiomyopathy at the European Society of Cardiology. On the deal-making front, ImmunoGen out-licensed its cancer treatment Elahere to Takeda for commercialization in Japan. The US Centers for Medicare and Medicaid (CMS) last Tuesday released the names of the first 10 drugs for price negotiations under the Inflation Reduction Act (IRA). Also of note, US biotech BioMarin Pharmaceuticals revealed it has treated a first patient with its gene therapy Roctavian for severe hemophilia A in Germany, marking the first commercial use in Europe. 3 September 2023
Biotechnology
Shares of Swedish enzyme technology firm Hansa Biopharma were up 3% at 43.82 kronor, after it announced the appointment of Dr Hitto Kaufmann as its chief scientific officer (CSO) effective December 1, 2023. 1 September 2023
Biotechnology
Following swiftly on regulatory approval from the UK’s Medicines and Healthcare products Regulatory Agency (MHRA), privately-held Italian drugmaker Chiesi has now also won backing from the health technology assessor for its Fabry disease drug. 1 September 2023
Biotechnology
Shares of US biotech FibroGen were down 22% at $0.995 pre-market today, after it announced top-line data from the Phase III LELANTOS-2 trial of pamrevlumab for the treatment of ambulatory patients with Duchenne muscular dystrophy (DMD) on background systemic corticosteroids. 30 August 2023
Pharmaceutical
Japanese drugmaker Kyowa Kirin has received approval in its home country for a higher-dose formulation of its oral calcimimetics agent Orkedia (evocalcet). 30 August 2023
Biotechnology
Bristol Myers Squibb has picked up a new approval in the USA for its anemia med Reblozyl (luspatercept-aamt), a first-in-class therapy which promotes late-stage red blood cell maturation. 30 August 2023
Pharmaceutical
The European Commission (EC) has approved the extension of the Evrysdi (risdiplam) European Union (EU) marketing authorization to include infants with a clinical diagnosis of spinal muscular atrophy (SMA) type one, type two or type three or with one to four SMN2 copies from birth to below two months. 29 August 2023
Pharmaceutical
The list of companies and groups that are suing the US government over its Medicare price-setting policies continues to grow. 25 August 2023
Biotechnology
Shares in Apellis Pharmaceuticals soared 30.2% to close at $40.04 yesterday after it provided more information regarding the safety of its eye disease product. 24 August 2023
Biotechnology
UK pharma major AstraZeneca today announced extended indication approvals of two major products in Japan. 24 August 2023
Pharmaceutical
Fulcrum Therapeutics saw its shares gain as much as 48.5% to $5.82 pre-market yesterday following an update from the US Food and Drug Administration (FDA). 23 August 2023
Biotechnology
August is Spinal Muscular Atrophy (SMA) Awareness Month – an opportune time to explore pharmacological advances in the treatment of this autosomal-recessive, progressive neuromuscular disease.1 23 August 2023
Biotechnology
The USA’s health technology assessor, the Institute for Clinical and Economic Review (ICER), today released a Final Evidence Report assessing the comparative clinical effectiveness of two sickle cell disease gene therapies. 21 August 2023
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