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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
Ireland-headquartered biopharma Horizon Therapeutics is to acquire US biotech Viela Bio for $3.05 billion. 1 February 2021
Biotechnology
Hypereosinophilic syndrome (HES) refers to a group of rare blood disorders that are characterized by a marked overproduction of eosinophils (white blood cells that lay an important role in immunity). While the exact prevalence of HES is unknown, the World Health Organization reported age-adjusted incidence of about 3-4 people per 10,000,000 between 2001 and 2005. 28 January 2021
Pharmaceutical
Agios Pharmaceuticals has announced positive results from the Phase III ACTIVATE trial of mitapivat. 28 January 2021
Pharmaceutical
Recordati has announced that the US Food and Drug Administration (FDA) has approved a new indication for Carbaglu (carglumic acid) tablets 200mg. 27 January 2021
Biotechnology
Boston, USA-based Albireo Pharma - a spinoff of UK-based AstraZeneca - is moving ahead with the regulatory process for odevixibat. 25 January 2021
Biotechnology
Shares of Swedish Orphan Biovitrum, also known as Sobi, gained more than 2% in early trading, as the company revealed that the European Commission has approved an extension of the indication for Doptelet (avatrombopag) to include the treatment of primary chronic immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments (eg, corticosteroids, immunoglobulins) in all European Union member states. 20 January 2021
Pharmaceutical
Anglo-Irish rare diseases specialist Amryt Pharma (LSE: AMYT) has won a positive reimbursement decision in the UK for Myalepta (metreleptin). 20 January 2021
Biotechnology
US cancer metabolism specialist Agios Pharmaceuticals has reported a full analysis of the final data, including mature overall survival (OS) results, from its global Phase III ClarIDHy trial of Tibsovo (ivosidenib tablets) in patients with previously treated isocitrate dehydrogenase 1 (IDH1) mutated cholangiocarcinoma, a rare cancer of the bile ducts. 19 January 2021
Biotechnology
Late Friday, Janssen announced that the US Food and Drug Administration has granted accelerated approval for Darzalex Faspro (the subcutaneous formulation of daratumumab), in combination with bortezomib, cyclophosphamide and dexamethasone (VCd or CyBorD for short), for the treatment of newly-diagnosed light chain (AL) amyloidosis. 16 January 2021
Biotechnology
Late December last year, there were Russian media reports suggesting that Swiss pharma giant Novartis was expected to face delays in the regulatory approval for its gene therapy Zolgensma (onasemnogene abeparvovec) in Russia, notably the Kommersant journal, on the basis of which were also reported by The Pharma Letter. 14 January 2021
Biotechnology
USA-based biotech firm bluebird bio has announced a plan to split into two separate companies, with one focusing on severe genetic disease and another on oncology. 12 January 2021
Pharmaceutical
Endocrine diseases specialist Diurnal Group has made one of the first submissions of a new application to the Medicines and Healthcare Regulatory Agency (MHRA) for independent UK approval, following the end of the Brexit transition period. 12 January 2021
Pharmaceutical
Russia continues to experience a shortage of at least 42 drugs, which are used for the treatment of some serious diseases including COVID-19, according to recent statements, by analysts of the All-Russian Union of Patients (VSP) and some local media, reports The Pharma Letter’s local correspondent. 12 January 2021
Pharmaceutical
As 2021 began, the first M&A deal of the year was announced, with Italy’s Angelini revealing the biggest acquisition in the company’s history, buying US CNS specialist Arvelle Therapeutics for up to $960 million. Also of note, Regeneron Pharmaceuticals announced positive initial data for its casirivimab plus imdevimab antibody cocktail in the treatment of hospitalized COVID-19 patients. US oncology focussed Calithera Biosciences released disappointing results for its lead candidate telaglenastat in renal cell carcinoma (RCC). Additionally, Alnylam Pharmaceuticals looks se for regulatory filings for its vutrisiran in the treatment of transthyretin-mediated ATTR amyloidosis following positive Phase III results. 10 January 2021
Biotechnology
Spanish skincare specialist Almirall has partnered with gene therapy company Tyris Therapeutics to tackle orphan dermatological conditions. 8 January 2021
Biotechnology
The HELIOS-A Phase III study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis, met its primary and both secondary endpoints at nine months in patients with hATTR amyloidosis with polyneuropathy. 8 January 2021
Biotechnology
Shares in rare disease specialist Sarepta Therapeutics fell around 45% after hours on Thursday, following mixed results from a Phase II study in Duchenne muscular dystrophy (DMD). 8 January 2021
Biotechnology
Registration of Russia’s first gene therapy drug Zolgensma (onasemnogene abeparvovec) for the treatment of spinal muscular atrophy (SMA), may be delayed by one and a half years as, in order to assess the quality of the drug, the Russian Ministry of Health has demanded from the Swiss pharma giant Novartis up to 40 vials with a total price of up to $88 million, reports The Pharma Letter’s local correspondent. 29 December 2020
Biotechnology
US biotech Rhythm Pharmaceuticals today reported top-line results from its pivotal Phase III clinical trial evaluating setmelanotide, the company’s melanocortin-4 receptor (MC4R) agonist, for the treatment of insatiable hunger and severe obesity in individuals with Bardet-Biedl syndrome (BBS) or Alström syndrome, two ultra-rare genetic diseases that often manifest with these symptoms. 22 December 2020
Pharmaceutical
As 2020 was drawing to a close, AstraZeneca announced the year’s largest M&A deal with a $39 billion bid for US rare diseases firm Alexion Pharmaceuticals. Also, Eli Lilly revealed an agreement to acquire gene therapy company Prevail Therapeutics for around $1 billion. On the research front, AbbVie presented new data on its Rinvoq, which showed superiority to Sanofi’s Dupixent in atopic dermatitis, and Novo Nordisk last week revealed it will test its diabetes drug Rybelsus (semaglutide) as a possible treatment for Alzheimer’s disease. Last Wednesday saw Gilead Sciences hand back most rights to rheumatoid arthritis drug Jyseleca (filgotinib) to Belgian partner Galapagos. 20 December 2020
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