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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
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
For the first time in 20 years, a new therapy for sickle cell disease is to be made available on the UK's National Health Service (NHS). 5 October 2021
Biotechnology
Ireland-headquartered biotech Horizon Therapeutics has announced the publication of a post-hoc analysis from the N-MOmentum Phase II/III pivotal trial of Uplizna (inebilizumab-cdon), which highlights a sustained effect on attack risk with no new safety signals in people with neuromyelitis optica spectrum disorder (NMOSD) who received the treatment for four or more years. 5 October 2021
Pharmaceutical
The list of rare (orphan) diseases, which consists of more than 200 diseases in Russia, needs to be updated, according to recent statements by Galina Izotova, deputy chairman of the Russian Audit Chamber. 5 October 2021
Biotechnology
Seattle, USA-based biotech Omeros Corporation saw its shares plummet 38% to $8.54 on Friday, as it revealed that the US Food and Drug Administration notified the company that its marketing application for narsoplimab could not be approved. 4 October 2021
Biotechnology
Among significant news last week, US biotechs Incyte and Syndax Pharmaceuticals signed a deal with an upfront payment of $117 million for the chronic graft-versus-host disease (cGVHD) candidate axatilimab. Biohaven Pharmaceutical suffered a setback last Monday, with the Phase III failure of its multiple system atrophy (MSA) candidate verdiperstat. AstraZeneca has exercized the option of its newly acquired Alexion unit to acquire Caelum Biosciences and its rare disease drug CAEL-101. US pharma giant Merck & Co hit the headlines on two fronts; first by confirming rumors that it had agreed an $11.5 billion bid to buy Acceleron Pharma and then with strong Phase III results for its oral antiviral molnupiravir in the treatment of non-hospitalized COVID-19 patients. 3 October 2021
Biotechnology
Rumors that US pharma giant Merck & Co was interested in acquiring Acceleron Pharma proved to be well-founded after an $11.5 billion deal was confirmed today. 30 September 2021
Biotechnology
US biotech Amicus Therapeutics is to create a new genetic medicine company called Caritas Therapeutics as part of a deal that will see its gene therapy business acquired by ARYA Sciences Acquisition Corp IV (Nasdaq: ARYD), a special purpose acquisition company (SPAC). 30 September 2021
Pharmaceutical
As September, Duchenne Action Month, comes to a close, we take a look at advances in pharmacological treatments for this rare condition. 30 September 2021
Biotechnology
Mirum Pharmaceuticals, a leader in rare liver disease, says that the US Food and Drug Administration has approved Livmarli (maralixibat) oral solution for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) one year of age and older. 30 September 2021
Biotechnology
Of the five late-stage pipeline products expected to be licensed in the seven major pharma markets for cystic fibrosis (CF) between 2020 and 2030, four will be CF transmembrane conductance regulator (CFTR) modulators, according to GlobalData. 29 September 2021
Biotechnology
Anglo-Swedish pharma major AstraZeneca’s recently-acquired Alexion rare diseases unit has exercised its option to acquire all remaining equity in Caelum Biosciences. 29 September 2021
Pharmaceutical
The market for neuroendocrine tumors (NETs) – a rare type of tumor where cancerous growth can begin in any cell within the hormone or nervous system – is expected to see a very slow 0.9% compound annual growth rate (CAGR) over the next decade in the world’s eight major markets (8MM; The USA, France, Germany, Italy, Spain, the UK, Japan and China) due to substantial generic erosion and low levels of innovation. 29 September 2021
Pharmaceutical
The lead program of Connecticut, USA-based neurology specialist Biohaven Pharmaceutical has been dealt a severe blow, with a Phase III flop in multiple system atrophy (MSA). 28 September 2021
Pharmaceutical
The Indian subsidiary of UK pharma major AstraZeneca has gained approval from the Drugs Controller General of India (DCGI) to import and market selumetinib capsules, indicated for pediatric patients with neurofibromatosis type 1. 27 September 2021
Biotechnology
Californian biotech company Capricor Therapeutics was trading 6% higher after 90 minutes of trading on Friday. 24 September 2021
Biotechnology
Japan’s Kyowa Kirin has provided new data for Crysvita (burosumab), revealing benefits for people with the rare metabolic bone disorder X-linked hypophosphatemia (XLH). 24 September 2021
Pharmaceutical
Swiss pharma giant Roche today announced data from across its growing neuromuscular portfolio at the World Muscle Society (WMS) Virtual Congress 20–24 September 2021. 24 September 2021
Biotechnology
China’s Worg Pharmaceuticals (Hangzhou) has entered into an asset purchase agreement to acquire Belgian biotech’s Apitope International’s proprietary platform and therapeutic pipeline. 23 September 2021
Biotechnology
Boston, USA-based Rhythm Pharmaceuticals has presented new data and analyses from Phase II and III trials of its candidate setmelanotide. 23 September 2021
Biotechnology
Announcing a second Asian out-licensing deal for its rare liver disease drug candidate, US biotech Mirum Pharmaceuticals has now granted exclusive rights to market maralixibat in Japan to Takeda Pharmaceutical. 22 September 2021
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