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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
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
Shares of US biotech firm Alexion Pharmaceuticals crashed 8% in pre-market trading on Friday, reacting to the news that peer Amgen is challenging of three patents covering top seller Soliris (eculizumab). 31 August 2019
Pharmaceutical
By Dr Nicola Davies
September 7th is World Duchenne Awareness Day and the perfect opportunity to delve into the details of Duchenne muscular dystrophy (DMD) and pharma’s role in helping individuals living with this condition. 30 August 2019
Biotechnology
The US Food and Drug Administration has granted Breakthrough Therapy designation for nirogacestat, an oral, selective, small molecule, gamma-secretase inhibitor, for the treatment of adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis, says USA-based biopharma firm SpringWorks Therapeutics. 29 August 2019
Biotechnology
Zogenix saw its stock drop as much as 16% on Tuesday, after it announced an acquisition of a privately held California start-up launched just 10 months ago with $30 million Series A investment led by F-Prime Capital Partners and OrbiMed. 28 August 2019
Biotechnology
ReveraGen BioPharma today announced positive study data with vamorolone in patients with Duchenne muscular dystrophy (DMD). 27 August 2019
Biotechnology
Eiger BioPharmaceuticals says that the US Food and Drug Administration has granted Breakthrough Therapy designation for peginterferon lambda (Lambda) for the treatment of hepatitis delta virus (HDV) infection. 22 August 2019
Biotechnology
Sarepta Therapeutics revealed that it has received a Complete Response Letter (CRL) from the US Food and Drug Administration regarding the New Drug Application (NDA) for Vyondys 53 (golodirsen) injection for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation amenable to exon 53 skipping. 20 August 2019
Pharmaceutical
A start-up set up by US pharma giant Pfizer to take forward a number of drug programs, is planning an initial public offering (IPO). 19 August 2019
Biotechnology
Celgene Corp today announced the US Food and Drug Administration has approved Inrebic (fedratinib) for the treatment of adult patients with intermediate-2 or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis. 16 August 2019
Biotechnology
The Institute for Clinical and Economic Review (ICER) could scarcely have been more damning of two companies behind treatments for Duchenne muscular dystrophy (DMD), the most common fatal genetic disease diagnosed in childhood. 16 August 2019
Biotechnology
Ultra-rare disease specialist Ultragenyx Pharmaceutical has inked a deal with GeneTx Biotherapeutics in relation to the antisense oligonucleotide GTX-102. 16 August 2019
Biotechnology
If the accusation of withholding information over Zolgensma (onasemnogene abeparvovec-xioi) is the first major test for Novartis chief executive Vasant Narasimhan, then it is increasingly looking like a big one. 14 August 2019
Biotechnology
Struggling rare diseases company Novelion Therapeutics admitted further difficulties on Tuesday related to its outgoing Aegerion subsidiaries. 13 August 2019
Pharmaceutical
Dutch drugmaker Pharming Group has entered into a development collaboration and license agreement with Novartis to develop and commercialize CDZ173, a small molecule phosphoinositide 3-kinase delta (PI3Kẟ inhibitor being developed by Novartis to treat patients with Activated Phosphoinositide 3-kinase Delta Syndrome (APDS). 13 August 2019
Biotechnology
Ireland-headquartered Jazz Pharmaceuticals late Monday announced the acquisition of clinical-stage biotech firm Cavion, through a merger with a Jazz subsidiary. 13 August 2019
Biotechnology
Shares of Eidos Therapeutics closed up 6.1% at $37.32 yesterday, after it was revealed that, under a non-binding proposal, BridgeBio Pharma will purchase all of the outstanding common stock of the company not already owned. 13 August 2019
Pharmaceutical
Research news last week featured a blow for US biotech GlycoMimetics when, along with partner Pfizer, it revealed that the pivotal study of rivipansel had failed in sickle cell disease. In patent wrangle news, Amgen won a favorable court ruling against Novartis’ Sandoz unit for its arthritis drug Enbrel, but an appeal is being lodged by the Erelzi biosimilar maker. On the regulatory front, Novartis is facing an embarrassing investigation over data manipulation relating to the BLA and subsequent approval of gene therapy Zolgensma. Also, Germany’s Bayer announced that it is acquiring full control of its joint venture BlueRock Therapeutics, a cell therapy company. 11 August 2019
Biotechnology
The self-styled leader in precision genetic medicine for rare diseases suffered a bad turn in the stock market on Thursday. 9 August 2019
Biotechnology
As it goes about launching Waylivra (volanesorsen) in Europe, the Ionis Pharmaceuticals affiliate Akcea Therapeutics has announced the publication of data on the rare disease therapy in The New England Journal of Medicine (NEJM). 8 August 2019
Biotechnology
Rhythm Pharmaceuticals has announced positive top-line results from two pivotal, Phase III clinical trials evaluating setmelanotide, the company’s melanocortin-4 receptor (MC4R) agonist, for the treatment of pro-opiomelanocortin (POMC) and leptin receptor (LEPR) deficiency obesities. 8 August 2019
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