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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
French drugmaker Sanofi has revealed the results from the Phase III LUNA 3 trial of rilzabrutinib, an oral bruton tyrosine kinase (BTK) inhibitor, in persistent or chronic immune thrombocytopenia (ITP). 6 November 2024
Pharmaceutical
The US Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) for donidalorsen, an investigational RNA-targeted medicine for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years of age and older, under development by Ionis Pharmaceuticals. 4 November 2024
Pharmaceutical
Disc Medicine today announced a successful End of Phase 2 with the US Food and Drug Administration (FDA) supporting the regulatory path forward, including potential for accelerated approval, for bitopertin in erythropoietic protoporphyria (EPP). 4 November 2024
Pharmaceutical
On Friday, the US Food and Drug Administration published the FDA Voices: “FDA Takes Exciting Steps Toward Establishing the Rare Disease Innovation Hub,” by Patrizia Cavazzoni, director of the FDA’s Center for Drug Evaluation and Research (CDER) and Dr Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research (CBER). 4 November 2024
Biotechnology
The US Food and Drug Administration (FDA) has granted a Rare Pediatric disease designation for BPM31510T, an investigational treatment for epidermolysis (EB) from BPGbio. 4 November 2024
article
The European Union's antitrust enforcer hit Israeli drugmaker Teva Pharmaceutical Industries (NYSE: TEVA) with a 463 million-euro ($502 million) fine Thursday for launching a smear campaign against a rival multiple sclerosis drug and misusing the patent system to thwart the competitor's attempt to entering the market. 31 October 2024
Biotechnology
US biotech major Amgen late yesterday released financial results for the third quarter of 2024, showing strong sales and earnings growth. 31 October 2024
Pharmaceutical
Italy’s Chiesi has announced the publication of results from the Phase III BRIGHT study of pegunigalsidase alfa 2mg/kg, administered every four weeks for 52 weeks, in adults with Fabry disease who were previously treated with agalsidase alfa or beta, administered every two weeks. 30 October 2024
Biotechnology
US biotech Biogenraised its annual earnings guidance and reported better-than-expected top and bottom lines for the third quarter of 2024, sending the company’s shares rising 3% in pre-market trading Wednesday. 30 October 2024
Biotechnology
Swedish Orphan Biovitrum, the Nordic biotech also known as Sobi, is presenting new research on the use of emapalumab in patients with macrophage activation syndrome (MAS) in Still’s disease, including systemic juvenile idiopathic arthritis (sJIA) and adult-onset still’s disease (AOSD) who had an inadequate response to high-dose glucocorticoids (GCs). 30 October 2024
Pharmaceutical
UK pharma major AstraZeneca today announced that the National Institute for Health and Care Excellence (NICE) published its final draft guidance (FDG) recommending Wainzua (eplontersen) as a treatment option for hereditary transthyretin-related amyloidosis (ATTR) in adults with stage 1 or stage 2 polyneuropathy across England and Wales. 29 October 2024
Biotechnology
AstraZeneca’s Fasenra (benralizumab) has been approved in the European Union (EU) as an add-on treatment for adult patients with relapsing or refractory eosinophilic granulomatosis with polyangiitis (EGPA). 28 October 2024
Biotechnology
California’s Vera Therapeutics has released 96-week follow-up results from its ORIGIN Phase IIb trial of atacicept, targeting IgA nephropathy (IgAN). 28 October 2024
Biotechnology
Intellia Therapeutics has announced Phase II results for its CRISPR-based therapy, NTLA-2002, aimed at providing a one-time treatment for hereditary angioedema (HAE). 25 October 2024
Pharmaceutical
Florida, USA-based biotech Hesperos has published early-stage research into a novel drug development technology that has caught the attention of French pharma major Sanofi. 21 April 2022
Biotechnology
Global Market Insights (GMI) has predicted 5% compound annual growth rate (CAGR) in the global hemophilia market between 2022 and 2028. 20 April 2022
Pharmaceutical
Boston, USA-based Sionna Therapeutics, a life sciences company founded in 2019 to develop highly effective and differentiated treatments for cystic fibrosis (CF), has announced the official launch of the company and the closing of a $111 million Series B financing. 20 April 2022
Biotechnology
In a filing with the US Securities and Exchange Commission (SEC), Boston-based rare disease specialist Imara has disclosed savage job cuts as part of an extensive restructuring. 19 April 2022
Biotechnology
US biotech TG Therapeutics saw its shares plunge almost 32% to $6.03, after it announced the voluntary withdrawal of the pending Biologics License Application (BLA)/supplemental New Drug Application (sNDA) for the combination of ublituximab and Ukoniq (umbralisib) - combination referred to as U2 - for the treatment of adult patients with chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL). 19 April 2022
Biotechnology
USA-based Be Biopharma (“Be Bio”), a pioneer in the discovery and development of Engineered B Cell Medicines (BeCM) founded in October 2020, has announced the closing of a $130 million financing, bringing the total investment in the company to over $180 million. 14 April 2022
Biotechnology
Swiss rare diseases specialist Santhera Pharmaceuticals has appointed Shabir Hasham as chief medical officer (CMO) and a member of its executive management team, effective May 1. 13 April 2022
Biotechnology
Japan’s largest drugmaker Takeda today announced that the Phase III SHP643-301 study evaluating the safety profile and pharmacokinetics (PK) of Takhzyro (lanadelumab) in patients aged two to 13 April 2022
Pharmaceutical
The Delhi High Court has issued a permanent injunction against two Indian companies protesting Novartis patent for eltrombopag olamine, an active pharmaceutical ingredient in its brand Revolade, till May 21, 2023. 13 April 2022
Biotechnology
Unofficial US drug price watchdog the Institute for Clinical and Economic Review (ICER) has published a new white paper in collaboration with researchers at NORC at the University of Chicago. 11 April 2022
Biotechnology
The UK's National Institute for Health and Care Excellence (NICE) has recommended AstraZeneca and Merck & Co’s Koselugo (selumetinib) for use by the National Health Service (NHS) in England. 6 April 2022
Biotechnology
Swiss pharma giant Novartis today announced the US Food and Drug Administration has granted accelerated approval to Vijoice (alpelisib) for the treatment of adult and pediatric patients years of age and older with severe manifestations of PIK3CA-Related Overgrowth Spectrum (PROS) who require systemic therapy. 6 April 2022
Biotechnology
The latest results from the Phase III CHAMPION-MG trial open-label extension showed that Ultomiris (ravulizumab-cwvz) demonstrated long-term efficacy in adults with anti-acetylcholine receptor (AChR) antibody-positive generalised myasthenia gravis (gMG). 6 April 2022
Biotechnology
Shares of US drug developer Imara closed down 22% at $1.27 yesterday having tanked as much as 34%, after it announced disappointing results from interim analyses of its Ardent Phase IIb clinical trial of tovinontrine (IMR-687) in patients with sickle cell disease (SCD) and Forte Phase IIb clinical trial of tovinontrine in patients with beta-thalassemia. 6 April 2022
Biotechnology
Israeli pharma company Protalix BioTherapeutics and family-owned Italian drugmaker Chiesi have announced positive top-line results from the 24-Month Phase III BALANCE trial of PRX-102 (pegunigalsidase alfa) in Fabry disease. 5 April 2022
Biotechnology
Coming as no surprise given the company recently admitted it was running out of cash, US biotech firm bluebird bio today announced that it is initiating a comprehensive restructuring intended to deliver up to $160 million in cost savings over the next two years. 5 April 2022
Biotechnology
Multiple sclerosis (MS) treatments developed by Biogen (Nasdaq: BIIB) have featured in data presentations at the annual congress of the American Academy of Neurology (AAN). 5 April 2022
Biotechnology
The US Food and Drug Administration has extended the review timeline of the New Drug Application (NDA) for vutrisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis, to allow for the review of newly added information related to the new secondary packaging and labeling facility. 4 April 2022
Biotechnology
German life sciences major Bayer underlined its determination to drive breakthrough innovations during an event on Friday. 2 April 2022
Pharmaceutical
Privately-held Italian drugmaker Chiesi has reported strong financial results for 2021, with revenues of 2.42 billion euros ($2.65 billion), an increase of 8.6% from the year before. 1 April 2022
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