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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
Tennessee, USA-based firm Rare Disease Therapeutics (RDT) has secured a new expanded indication for its antivenom product Anavip (crotalidae immune). 6 April 2021
Pharmaceutical
US clinical-stage biopharma AzurRx BioPharma saw its shares plunge nearly 24% to $1.02 last week, after the company released top-line results from its Phase IIb OPTION 2 clinical trial investigating MS1819 in cystic fibrosis (CF) patients with exocrine pancreatic insufficiency (EPI) that did not consistently meet the primary efficacy goals. 3 April 2021
Biotechnology
The US Food and Drug Administration has approved Praluent (alirocumab) injection for adult patients with homozygous familial hypercholesterolemia (HoFH), a genetic condition that causes severely high cholesterol. Praluent is not intended to be used alone but instead added to other treatments for HoFH. 2 April 2021
Biotechnology
Three years after launching in the UK, Swedish Orphan Biovitrum's Kineret (anakinra) has secured a positive reimbursement decision from the country’s health technology assessor. 1 April 2021
Biotechnology
Privately-held US biotech Omega Therapeutics has closed an upsized Series C financing of $126 million. 30 March 2021
Biotechnology
Swiss pharma giant Roche has seen its spinal muscular atrophy (SMA) drug Evrysdi (risdiplam) follow up its US approval by getting the green light from the European Commission (EC). 30 March 2021
Pharmaceutical
The Delhi High Court in India has set out three major directives with regard to patients suffering from rare diseases. 29 March 2021
Biotechnology
For Amyloidosis Awareness Month, we take the opportunity to review advances in the pharmacological treatment for amyloidosis. 29 March 2021
Pharmaceutical
The European Medicines Agency’s Committee for Medicinal Products for Human Use recommended five novel medicines for approval at its March 2021 meeting. A final decision on the approval by the European Commission is usually issues in about two-three months. 27 March 2021
Pharmaceutical
Following its March meeting, the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) announced recommendations for the extension of indications on six already approved drugs. 27 March 2021
Biotechnology
Life sciences investment trust Syncona has invested $42.3 million UK-based Gyroscope Therapeutics, a clinical-stage gene therapy company focused on diseases of the eye, in an $148.0 million (£107.8 million) Series C financing. 26 March 2021
Pharmaceutical
Medicines to treat infectious diseases and inflammatory disorder, as well as cancers, Alzheimer’s and rare diseases that collectively affect millions of people are under development by pharma and biotech, but the problem now is to deliver them to patients. 26 March 2021
Biotechnology
US biotech Vertex Pharmaceuticals has announced that the Australian Therapeutic Goods Administration (TGA) has approved the use of Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for people with cystic fibrosis (CF). 25 March 2021
Pharmaceutical
Italian drugmaker Recordati has presented positive results from the Phase III LINC 4 study of Isturisa (osilodrostat) at the Endocrine Society’s Annual Meeting. 23 March 2021
Biotechnology
US oral peptides specialist Chiasma’s shares were up 13% at $3.89 pre-market today, after the company announced the presentation of new positive clinical data from its MPOWERED Phase III trial of Mycapssa (octreotide) at the Endocrine Society’s annual meeting, ENDO 2021, being held virtually March 20-23, 2021. 23 March 2021
Pharmaceutical
According to a summary published by the Hungarian Association of Innovative Pharmaceutical Manufacturers (AIPM) on the occasion of the Rare Disease Day, increasingly effective innovative therapies present a new opportunity for patients. 22 March 2021
Pharmaceutical
China has been relying on daily human growth hormone (HGH) injections to treat growth hormone deficiency (GHD), but this is likely to change, if not soon. 19 March 2021
Biotechnology
The US Food and Drug Administration has approved Arcalyst (rilonacept) injection to treat recurrent pericarditis and reduce the risk of recurrence in adults and children 12 years and older. 19 March 2021
Biotechnology
Results from the Phase III PEGASUS trial have been published by Nordic rare diseases firm Swedish Orphan Biovitrum, also known as Sobi, and its partner Apellis Pharmaceuticals. 18 March 2021
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