7 January 2025 Unquestionably, US drugmaker Vertex Pharmaceuticals is the dominant drug developer in the cystic fibrosis (CF) space.
How has the company achieved this — on the R&D and patient access side — and what is the company doing to help the remaining patients? Vertex’s Andrew Wilfin, Senior Country Medical Director, UK and Ireland, took part in a Q&A with The Pharma Letter to reveal all.
Insilico Medicine, a biotechnology firm specializing in artificial intelligence-powered drug discovery, has raised over $100 million in a series E funding round. 8 January 2025
Calico Life Sciences, a biotech founded by Alphabet and Arthur Levinson focused on the biology of aging and age-related diseases, has announced a setback. 8 January 2025
Denmark’s Novo Nordisk and Valo Health today announced they have entered into an expanded agreement to discover and develop novel treatments for obesity, type 2 diabetes, and cardiovascular disease based on Valo’s extensive human dataset and computation powered by artificial intelligence (AI). 8 January 2025
Antag Therapeutics, a Denmark-based biopharma developing novel treatments for obesity, has announced the appointment of Joerg Moeller as chief executive officer, effective immediately. 8 January 2025
Metsera today announced positive data from a 12-week Phase IIa clinical trial of MET-097i, its potential once-monthly, ultra-long acting, subcutaneously injectable, fully-biased, GLP-1 receptor agonist (RA). 7 January 2025
Central nervous system (CNS) specialist Neuraxpharm Group has acquired Provigil (modafinil) and Nuvigil (armodafinil), both of which are indicated for the treatment of excessive daytime sleepiness (EDS) in adults with narcolepsy. 7 January 2025
Japan-based FUSO Pharmaceutical Industries and Australia’s Dimerix Limited have entered into an exclusive development and license agreement for the development and commercialization of Dimerix’ Phase III drug candidate DMX-200 for the treatment of focal segmental glomerulosclerosis (FSGS) kidney disease in Japan. 7 January 2025
The heart failure market is projected to grow at a compound annual growth rate of 9.6% over the next decade, increasing from $13.5 billion in 2022 to $33.7 billion in 2032 across seven major markets, according to a report from analytics firm GlobalData. 7 January 2025
A late-stage trial failure has dented Denali Therapeutics stock, with an announcement Monday revealing the HEALEY ALS study missed all endpoints. 7 January 2025
Unquestionably, US drugmaker Vertex Pharmaceuticals is the dominant drug developer in the cystic fibrosis (CF) space.
How has the company achieved this — on the R&D and patient access side — and what is the company doing to help the remaining patients? Vertex’s Andrew Wilfin, Senior Country Medical Director, UK and Ireland, took part in a Q&A with The Pharma Letter to reveal all. 7 January 2025
The US Food and Drug Administration (FDA) issued a draft guidance to provide recommendations on the use of artificial intelligence (AI) intended to support a regulatory decision about a drug or biological product’s safety, effectiveness or quality. 7 January 2025
Sentynl Therapeutics, wholly owned by Zydus Lifesciences, has announced that the US Food and Drug Administration has accepted its New Drug Application for CUTX-101, a potential first treatment for Menkes disease. The application has been granted priority review, with a target action date set for June 30, 2025. 7 January 2025
rug Administration (FDA) approved an update to the US Prescribing Information (USPI) for Pyrukynd (mitapivat) for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency, Agios Pharmaceutical revealed in a Security and Exchange Commission (SEC) notice. 7 January 2025
Patrick Branch, head of Japan and APAC business development at Nxera Pharma,
Japan’s most innovative and nimble biopharma company, provides a summary of the year in the important eastern market. 6 January 2025
Immedica Pharma, a Swedish company specializing in rare diseases, has announced its acquisition of Marinus Pharmaceuticals in a deal valued at $151 million. The agreement, finalized through a cash tender offer, marks a significant expansion of Immedica’s presence in the US market. 6 January 2025
Anglo-Swedish drugmaker AstraZeneca has announced that new, positive, long-term results from the pivotal ALPHA Phase III trial have appeared in the journal Blood, which is published by the American Society of Hematology. 6 January 2025
Alon Natanson, a business leader and alumnus of the Hebrew University of Jerusalem, has been appointed chief executive officer (CEO) of Yissum, the Israeli Hebrew University Tech Transfer company. 6 January 2025
Japanese drugmaker Otsuka Pharmaceutical has announced that the European Medicines Agency (EMA) has accepted for review the Marketing Authorization Application (MAA) for the hereditary angioedema(HAE) drug candidate donidalorsen. 6 January 2025
The European Commission (EC) has authorized Biogen’s Skyclarys (omaveloxolone) for the treatment of Friedreich’s ataxia (FA) in adults and adolescents aged 16 years and older. 13 February 2024
Foghorn Therapeutics has announced that its partner, Eli Lilly has selected FHD-909, a first-in-class oral BRM selective inhibitor, for clinical development. 12 February 2024
The USA-based AMR Industry Alliance conducted a review of publicly available data on the state of antimicrobial resistance (AMR) research and development talent. 12 February 2024
Danish drugmaker Lundbeck has its eye on steady growth over the medium term, a strategy which new chief executive Charl van Zyl believes could be key to the firm’s future success. 12 February 2024
UK-based Poolbeg Pharma has announced that research conducted on its behalf confirms a market opportunity of at least $10 billon for POLB 001 in cancer immunotherapy-induced cytokine release syndrome (CRS) as an orally-delivered preventative therapy. 12 February 2024
Spanish plasma-derived products specialist Grifols has announced that Raimon Grifols and Víctor Grifols Deu have chosen to step down from their executive functions – chief corporate officer and chief operating officer, respectively. 12 February 2024
The European Medicines Agency’s (EMA) Pharmacovigilance Risk Assessment Committee (PRAC) is reminding healthcare professionals of the risk of serious and potentially fatal adverse reactions with the COVID-19 treatment Paxlovid (nirmatrelvir and ritonavir). 9 February 2024
Japan’s largest drugmaker Takeda Pharma has announced positive top-line results from a randomized, double-blind, placebo-controlled, multiple dose Phase IIb trial evaluating TAK-861, an oral orexin receptor 2 (OX2R) agonist, in patients with narcolepsy type 1. 9 February 2024
Vicor Pharma has entered into an exclusive licensing agreement with Japan’s Nippon Shinyaku, for the commercialization and development of its C21 idiopathic pulmonary fibrosis (IPF) drug candidate in Japan. 9 February 2024
Despite posting positive clinical trial results for its resmetirom, being trialled in the treatment of non-alcoholic steatohepatitis (NASH,) US drug developer Madrigal Pharmaceuticals saw its shares close down more than 9% at $173.71 yesterday. 9 February 2024
In an Expert View piece, Mark Seymour, Director and Head of Media, Healthcare, at FleishmanHillard London, argues that in the ever-evolving world of healthcare, the rules of communications have not changed, they have just become more intricate, demanding a renewed focus on the essentials. 8 February 2024
The USA’s Federal Trade Commission yesterday issued a comment in response to the National Institute of Standards and Technology’s (NIST) request for information on its Draft Interagency Guidance Framework for Considering the Exercise of March-In Rights under the Bayh-Dole Act. 7 February 2024
French pharma major Sanofi has announced that Rippon Ubhi has been appointed as country lead for the UK and Ireland, taking over immediately from Jessamy Baird. 7 February 2024
CNS specialist Lundbeck has reported record annual revenue for 2023 of 19.91 billion Danish kroner ($2.87 billion), a 9% increase on 2022. 7 February 2024
US biopharma Vertex Pharmaceuticals has announced positive results from its once-daily vanzacaftor/tezacaftor/deutivacaftor program, the most comprehensive Phase III pivotal program ever conducted by the company for cystic fibrosis (CF). 6 February 2024
Clinical-stage US biotech Acumen Pharmaceuticals has appointed James Doherty as president and chief development officer, effective February 1, 2024. 6 February 2024