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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
The US Food and Drug Administration on Thursday approved Uplizna (inebilizumab-cdon) injection for intravenous use for the treatment of neuromyelitis optica spectrum disorder (NMOSD), also called Sjögren’s syndrome, in adult patients with a particular antibody (patients who are anti-aquaporin-4 or AQP4 antibody positive). 12 June 2020
Biotechnology
New results from the NURTURE study show ongoing benefits for people with spinal muscular atrophy (SMA), when treated with Spinraza (nusinersen). 12 June 2020
Biotechnology
The European Commission has granted approval for a label extension on Kalydeco (ivacaftor), the only drug approved in Europe to treat the underlying cause of cystic fibrosis. 10 June 2020
Pharmaceutical
June 9th is International Batten Disease Awareness Day. We take this opportunity to explore the current therapeutic landscape for Batten disease and discuss some of the emerging therapies offering hope for the future. 9 June 2020
Pharmaceutical
Soleno Therapeutics saw its shares dive 48.8% to $1.66 in after-hours trading yesterday, after it announced top-line results from the Phase III trial, DESTINY PWS (C601), evaluating once-daily diazoxide choline controlled-release (DCCR) tablets for patients with Prader-Willi syndrome (PWS), a rare genetic disorder that failed to meet its primary endpoint of change from baseline in hyperphagia. 9 June 2020
Pharmaceutical
The share of Russian drugs in public procurements, which are carried out with the state program for high-cost nosologies program, which involves purchases of the most expensive drugs for the needs of Russian hospitals and other medical institutions – has reached a historic maximum of 42.7% and currently continues to grow, according to recent statements made by some local analysts and Russian media, reports The Pharma Letter’s local correspondent. 9 June 2020
Biotechnology
The US Food and Drug Administration has granted Breakthrough Therapy designation for brensocatib (formerly known as INS1007) for the treatment of adult patients with non-cystic fibrosis bronchiectasis (NCFBE) for reducing exacerbations, says the drug’s developer, US rare-diseases focussed biopharma Insmed. 8 June 2020
Biotechnology
Positive Phase III results from the ILLUMINATE-A study of lumasiran, an investigational RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) – the gene encoding glycolate oxidase (GO) – in development for the treatment of primary hyperoxaluria type 1 (PH1), were presented by US RNAi specialist Alnylam Pharmaceuticals at the European Renal Association-European Dialysis and Transplant Association (ERA-EDTA) International Congress on Sunday. 8 June 2020
Biotechnology
Japan’s largest drugmaker Takeda Pharmaceutical has announced findings from two new interim analyses of data from the Phase III HELP (Hereditary Angioedema Long-term Prophylaxis) Study Open-label Extension (OLE) at the 2020 European Academy of Allergy and Clinical Immunology (EAACI) Digital Congress. 8 June 2020
Pharmaceutical
By Barbara Obstoj-Cardwell. Editor
Last week’s news included disappointing Phase III trial results for Pfizer’s drug Ibrance in the treatment of breast cancer. Gilead Sciences released data from its own study of remdesivir, now trade-named Veklury, showing the drug, though useful, did not produce statistically-significant results in the treatment of COVID-19, but analysts still see commercial success in the longer term. On a positive note, Novo Nordisk provided further evidence of the value of its semaglutide in achieving weight loss. Elsewhere, Novartis revealed last Wednesday that the US Food and Drug Administration had extended its review of the multiple sclerosis therapy ofatumumab. 7 June 2020
Pharmaceutical
Indian drugmaker Lupin has entered into distribution agreements with three companies for its orphan drug NaMuscla (mexiletine), which received European Commission approval in December 2018, for the symptomatic treatment of myotonia in adults with non-dystrophic myotonic (NDM) disorders. 3 June 2020
Biotechnology
Pfizer, at a time when the COVID-19 pandemic is disrupting drugmakers clinical trials activities, on Tuesday announced the establishment of the Pfizer Breakthrough Growth Initiative, through which the US pharma giant will invest up to $500 million in biotechnology companies to help provide funding and access to Pfizer’s scientific expertise to ensure continuity of the biotechnology companies’ most promising clinical development programs. 3 June 2020
Biotechnology
Clinical-stage USA-based biotech Aruvant Sciences today announced the appointment of V "Palani" Palaniappan, as chief technology officer (CTO) effective immediately. 1 June 2020
Biotechnology
French drugmaker Ipsen, which has been under interim management since the surprise departure of its chief executive David Meek in December last year, today announced it has appointed David Loew as its new CEO and as board member. The CEO appointment will take effect on July 1, 2020 29 May 2020
Biotechnology
Israeli company Protalix BioTherapeutics and family-owned Italian group Chiesi have submitted a Biologics License Application (BLA) to the US Food and Drug Administration (FDA) for pegunigalsidase alfa to treat Fabry disease. 28 May 2020
Biotechnology
Akcea Therapeutics today announced publication of long-term data from the open-label extension (OLE) of the pivotal NEURO-TTR study of Tegsedi (inotersen) in patients with hereditary amyloidosis transthyretin (hATTR) with polyneuropathy. 28 May 2020
Biotechnology
Azitra, a privately-held dermatology and microbiome specialist, has been awarded Rare Pediatric Disease designation for its candidate ATR-12, for the treatment of Netherton syndrome (NS). 28 May 2020
Biotechnology
Belgo-Dutch clinical-stage biotech argenx saw its shares close up nearly 30% at $190.00 on Tuesday, after it released top-line trial results for its myasthenia gravis drug candidate. 27 May 2020
Biotechnology
Roche data from two pivotal Phase III open-label extension studies shows satralizumab was well-tolerated as a monotherapy or in combination with baseline immunosuppressive therapy in neuromyelitis optica spectrum disorder (NMOSD). 22 May 2020
Pharmaceutical
The US Food and Drug Administration has approved Ferriprox (deferiprone) twice-a-day tablets for the treatment of patients with transfusional iron overload due to thalassemia syndromes when current chelation therapy is inadequate, to be marketed by the US subsidiary of family-controlled Italian drugmaker Chiesi Farmaceutici. 22 May 2020
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