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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
US clinical-stage drug developer Sio Gene Therapies has presented positive interim data from its ongoing Phase I/II study of AXO-AAV-GM1, its adeno-associated viral vector (AAV)9-based gene therapy candidate for the treatment of GM1 gangliosidosis. 22 October 2021
Biotechnology
Rare diseases specialist Swedish Orphan Biovitrum, also known as Sobi, today posted financial results for the third quarter of 2021, with total revenue of 3,761 million Swedish kronor ($437 million), an increase of 27% compared with the same period a year ago and 29% at constant exchange rates (CER). 22 October 2021
Biotechnology
UK health technology assessor the National Institute for Heath and Care Excellence (NICE) has recommending the use of Givlaari (givosiran) on the NHS in England as an option for the treatment of acute hepatic porphyria (AHP) in people 12 years and older who have clinically confirmed severe recurrent attacks (four attacks or more within 12 months). 21 October 2021
Pharmaceutical
Parisian drugmaker Ipsen has reported total sales of 727 million euros (846 million) in the third quarter, representing growth of 14.9% from the same period of 2020. 21 October 2021
Pharmaceutical
AI-driven drug combo specialist Pharnext saw its shares leap nearly a tenth in morning trading on Tuesday, after a positive result from the Phase III PLEO-CMT trial. 19 October 2021
Biotechnology
German family-owned pharma major Boehringer Ingelheim today said it has exercised its option to license UK-based Oxford Biomedica’s lentiviral vector technology to manufacture, register and commercialize BI 3720931, a lentiviral vector-based gene therapy for the treatment of cystic fibrosis (CF). 19 October 2021
Biotechnology
Shares of Omeros Corporation plummeted nearly 27% to $5.67 by close of trading Monday, as the US biotech revealed another setback in its development of narsoplimab. 19 October 2021
Pharmaceutical
A positive reimbursement decision in the UK will see patients in England and Wales gain access to Boehringer Ingelheim’s Ofev (nintedanib) through the country’s national healthcare provider. 18 October 2021
Biotechnology
US biotech major Biogen has released disappointing top-line results from its pivotal Phase III VALOR study of tofersen (BIIB067), an investigational antisense drug being evaluated for people with superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS). 18 October 2021
Biotechnology
Seattle, USA-based cell therapy company Immusoft has signed a research collaboration and license option agreement with Japan’s Takeda Pharmaceutical to discover, develop and commercialize transformative cell therapies in rare inherited metabolic disorders with central nervous system (CNS) manifestations and complications using Immusoft’s Immune System Programming (ISP) technology platform, which modifies a patient’s B cells and instructs the cells to deliver gene-encoded therapies. 18 October 2021
Biotechnology
Significant regulatory news last week included Swiss pharma giant Roche gaining Breakthrough Therapy designation from the US Food and Drug Administration for its Alzheimer’s candidate gantenerumab. Also, Eli Lily last Wednesday was granted FDA approval for its CDK 4/6 inhibitor Verzenio in an early breast cancer indication. Swiss biotech Idorsia suffered a setback last Monday when the firm reported that its Fabry disease hopeful lucerastat missed endpoints in a Phase III trial. An FDA advisory panel last Thursday voted in favor of approving Moderna’s COVID-19 vaccine – Spikevax - to be used for booster jabs. And on Friday the AdCom also backed booster jabs of Johnson & Johnson’s vaccine. 17 October 2021
Biotechnology
German pharma and life science firm Merck KGaA has shared data in an oral presentation from a Phase II post-hoc analysis, demonstrating that the oral, central nervous system (CNS)-penetrant, fully covalent Bruton’s tyrosine kinase inhibitor (BTKi) evobrutinib impacted brain lesions associated with chronic inflammation in the CNS. 15 October 2021
Biotechnology
Shares of USA/Israel-based biotech Brainstorm Cell Therapeutics were up 7% at $3.15 pre-market, but dropped back as regular trading came into play. 14 October 2021
Biotechnology
Swiss pharma giant Roche has announced new longer-term efficacy and safety data for the approved therapy Enspryng (satralizumab). 14 October 2021
Biotechnology
Privately-held Italian biopharma company Dompé Farmaceutici and its Dompé US Inc unit are planning to expand commercial operations for Oxervate (cenegermin ophthalmic solution) to Canada. 14 October 2021
Pharmaceutical
London-listed endocrine disease specialist Diurnal has signed a distribution agreement with ExCEEd Orphan for Alkindi (hydrocortisone) and Efmody (hydrocortisone). 14 October 2021
Pharmaceutical
French drugmaker Ipsen has appointed Mari Scheiffele as executive vice president and president, Specialty Care International, effective November 1, 2021. 11 October 2021
Pharmaceutical
Shares in Swiss biotech Idorsia were down 6% on Monday morning, after the firm announced negative results from the Phase III MODIFY trial of lucerastat. 11 October 2021
Biotechnology
Following an evenly split vote from the US regulator’s advisory committee in May this year, there was good news for US biotech ChemoCentryx on Friday, as the US Food and Drug Administration approved the firm’s Tavneos (avacopan), an orally administered selective complement 5a receptor inhibitor, propelling its shares 69% higher to $33.13. 9 October 2021
Pharmaceutical
Fortress Biotech subsidiary Cyprium Therapeutics, with support from its licensing partner Sentynl Therapeutics, which is wholly-owned by India’s Cadila Healthcare, today announced positive results from an efficacy and safety analysis of data integrated from two completed pivotal studies in patients with Menkes disease treated with CUTX-101, copper histidinate (CuHis). 8 October 2021
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