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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
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
article
Swedish drugmaker Calliditas Therapeutics has received full US Food and Drug Administration approval for Tarpeyo (budesonide), upgrading a previous nod under the Accelerated Approval scheme. 21 December 2023
Biotechnology
Japanese drugmaker JCR Pharmaceuticals saw its shares dip 1.75% to 1,121 yen today, despite news of a second deal with Alexion. 21 December 2023
Biotechnology
Shares of Dutch immunology specialist argenx slumped as much as 30% to 290.60 euros this morning, after it announced disappointing top-line results from the ADDRESS study 20 December 2023
Pharmaceutical
The rare disease unit of Italian drugmaker Chiesi has secured US approval for Filsuvez (birch triterpenes), a new treatment for people with junctional epidermolysis bullosa (JEB) and dystrophic epidermolysis bullosa (DEB). 20 December 2023
Pharmaceutical
Midsize Japanese drugmaker Otsuka Pharmaceuticals has entered into a licensing agreement with Ionis Pharmaceuticals related to its candidate donidalorsen. 19 December 2023
Biotechnology
The European Commission has extended the approval of Evkeeza (evinacumab) as an adjunct to diet and other lipid-lowering therapies to treat children aged five to 11 years with homozygous familial hypercholesterolemia (HoFH). 19 December 2023
Biotechnology
US biotech bluebird bio saw a negative reaction to its proposed sales of shares, with the stock closing down 6.7% and a further15.6% to $2.575 in after-hours trading on Monday. 19 December 2023
Pharmaceutical
Swiss specialty pharma Santhera Pharmaceuticals has announced that Agamree (vamorolone) has been approved in the European Union (EU) to treat Duchenne muscular dystrophy (DMD) in patients four years of age and older, independent of the underlying mutation and ambulatory status. 18 December 2023
Biotechnology
Australian biotech Neuren Pharmaceuticals saw its share jump more than 22% to A$29.5 today, after it announced top-line results from its Phase II clinical trial of NNZ-2591 in children with Phelan-McDermid syndrome (PMS). 18 December 2023
Biotechnology
Regulatory news last week saw the US Food and Drug Administration (FDA) make a land-mark decision to approve the first gene therapies for sickle cell disease (SCD) – Vertex Pharmaceuticals and CRISPR’s Casgevy and bluebird bio’s Lyfgenia. Also of note, the FDA announced the creation of a new advisory committee for genetic metabolic diseases. M&A and deal-making news saw the UK’s AstraZeneca reveal a $1.1 billion takeover bid for Icosavax, aiming to strengthen its late-stage vaccine pipeline. Additionally, USA-based C4 Therapeutics announced a license and research collaboration with pharma giant Merck & Co on degrader antibody conjugates. On the research front, Vertex reported compelling Phase II data on VX-548, its non-opioid treatment for diabetic peripheral neuropathy (DPN). 17 December 2023
Biotechnology
The European Medicines Agency’s (EMA) human medicines committee (CHMP) recommended a number of new and generic medicines for approval at its December 2023 meeting. 15 December 2023
Pharmaceutical
Two years after Welireg's (belzutifan) original approval in the rare von Hippel-Lindau (VHL) disease, the Food and Drug Administration has now approved the Merck & Co drug for a new indication. 15 December 2023
Pharmaceutical
Shares in Californian rare disease company Reneo Pharmaceuticals have plummeted after the firm announced the failure of its pivotal STRIDE study. 15 December 2023
Biotechnology
Former Novartis executive James Bradner has joined Amgen as executive vice president of research and development (R&D), and chief scientific officer. 14 December 2023
Pharmaceutical
The US Food and Drug Administration (FDA) yesterday announced the establishment of a new Genetic Metabolic Diseases Advisory Committee (GeMDAC) to help the agency explore the complex issues related to genetic metabolic disease drug development. 14 December 2023
Biotechnology
South Korea medicines regulator has issued guidelines to monitor the side effects of the two approved CAR-T therapies – Novartis’ Kymriah (tisagenlecleucel) and Johnson & Johnson subsidiary Janssen’ Carvykti (ciltacabtagene autoleucel). 13 December 2023
Pharmaceutical
Rare disease drug development has grown exponentially in recent years, and 2023 has continued to expand on this trend.1 13 December 2023
Biotechnology
The US Federal Trade Commission (FTC) is seeking to block French pharma major Sanofi’s proposed acquisition of an exclusive license to Maze Therapeutics therapy in development for treatment of Pompe disease. 12 December 2023
Pharmaceutical
Swiss pharm giant Novartis has announced positive results from the extension period of the pivotal Phase III APPLY-PNH trial of oral monotherapy Fabhalta (iptacopan). 11 December 2023
Biotechnology
The US Food and Drug Administration (FDA) on Friday approved two milestone treatments, Casgevy (exagamglogene autotemcel [exa-cel]), and Lyfgenia (lovotibeglogene autotemcel [lovo-cel)], representing the first cell-based gene therapies for the treatment of sickle cell disease (SCD). 11 December 2023
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