Rare Diseases
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Biotechnology
New Jersey, USA-based Merck & Co has been granted a Priority Review from the US regulator for its pulmonary arterial hypertension (PAH) candidate sotatercept. 29 September 2023
Pharmaceutical
US biopharma Soleno Therapeutics saw its market capitalization leap five-fold yesterday, with the shares hitting $26.80, after it announced promising research results for its lead pipeline candidate, which has encountered previous setbacks. 27 September 2023
Pharmaceutical
USA-based Ionis Pharmaceuticals yesterday released positive top-line results for the Phase III Balance study of olezarsen in people with familial chylomicronemia syndrome (FCS), a rare genetic disorder. 27 September 2023
Pharmaceutical
A $250 million private financing for California’s BridgeBio Pharma will support a possible launch of acoramidis, as well as further late-stage pipeline development. 26 September 2023
Biosimilars
The European Commission (EC) granted marketing authorization for Tyruko (natalizumab), the first and only biosimilar multiple sclerosis drug, developed by Dutch company Polpharma Biologics. 26 September 2023
Biotechnology
The Japanese Ministry of Health, Labor and Welfare (MHLW) has granted approval for Rystiggo (rozanolixizumab) and for Zilbrysq (zilucoplan). 26 September 2023
Pharmaceutical
Swiss pharma major Novartis has announced the Phase III NETTER-2 trial of Lutathera (lutetium Lu 177 dotatate) has met its primary endpoint. 25 September 2023
Pharmaceutical
Plans to overhaul the European Union (EU) regulation on rare disease therapies risk undermining two decades of progress, a new report has found. 25 September 2023
Pharmaceutical
Shares of Travere Therapeutics plunged more than 40% to $7.65 yesterday after it released top-line two-year confirmatory secondary endpoint results from the pivotal head-to-head Phase III PROTECT study of Filspari (sparsentan) in IgA nephropathy (IgAN), which narrowly missed eGFR total slope endpoint compare to irbesartan. 22 September 2023
Pharmaceutical
Swiss pharma firm Acino has acquired M8 Pharmaceuticals, a specialty biopharmaceutical company focused on licensing, marketing, and distributing innovative and established medicines in Mexico and Brazil. 20 September 2023
Biotechnology
Gene therapy company Orchard Therapeutics has announced that the US Food and Drug Administration (FDA) has accepted the filing of its Biologics License Application (BLA) for OTL-200 in metachromatic leukodystrophy (MLD) under Priority Review. 20 September 2023
Biotechnology
ALSA Ventures has announced the acquisition of Axovia Therapeutics Inc. 19 September 2023
Biotechnology
The European Medicines Agency (EMA) has recommended not renewing the marketing authorization for Translarna (ataluren). 18 September 2023
Pharmaceutical
Thyroid cancer is a rare cancer that encompasses several variations (Table 1).1-3 The incidence is higher in women compared to men.4 16 September 2023
Pharmaceutical
Following its September 2023 meeting, the European Medicines Agency’s (EMA) human medicines committee (CHMP) has recommended approval of six novel medicines. 15 September 2023
Biosimilars
The Indian government has floated a scheme for the promotion of research and innovation in the pharmaceutical and medical technology sector, with an aim to reduce the country’s dependence on imports of products such as orphan drugs. 15 September 2023
Biotechnology
US health technology assessor the Institute for Clinical and Economic Review ICER) today posted its revised Evidence Report assessing the comparative clinical effectiveness and value of atidarsagene autotemcel (“arsa-cel”), from USA and UK-based Orchard Therapeutics, for metachromatic leukodystrophy (MLD). 14 September 2023
Biotechnology
Stock in New Jersey, USA-based Rocket Pharmaceuticals shot up around 40% on Wednesday, following good news on the financing and regulatory fronts. 14 September 2023
Biotechnology
US biotech Moderna has spent much of the last couple of years banging the drum about the potential of its mRNA platform beyond producing the COVID-19 vaccine that brought the company multi-billion dollar sales and global acclaim. 13 September 2023
Biotechnology
Neurocrine Biosciences saw its shares rise 6.4% to $115.50 this morning, as it announced positive top-line data from the Phase III CAHtalyst Adult Study. 12 September 2023
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
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