Rare Diseases
Refine Search
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
In the UK, two new marketing authorizations have been granted by the country’s medicines regulator, the Medicines and Healthcare products Regulatory Agency (MHRA). 2 February 2023
Biotechnology
Dutch gene therapy company uniQure and University of Massachusetts Medical School (UMMS) spin-out Apic Bio have entered into a global licensing agreement for APB-102 to treat superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS), a rare, genetic form of ALS. 31 January 2023
Biotechnology
Preliminary results from the ongoing global Phase III STAND study in sickle cell disease are not what Novartis had hoped. 30 January 2023
Pharmaceutical
Israel-based dermatology focussed biotech Sol-Gel Technologies says it has acquired rights to the topically-applied patidegib, a hedgehog signaling pathway blocker, for the treatment of Gorlin syndrome from US drug developer PellePharm. 30 January 2023
Biotechnology
Although there were several positive recommendations for label extensions on already approved medicines, January proved a thin month for novel drug approvals in the European Union, with just one new drug and three for generics. 27 January 2023
Biotechnology
The US Food and Drug Administration (FDA) has approved the expanded indication for Enjaymo (sutimlimab-jome), the first and only treatment of hemolysis in adults with cold agglutinin disease (CAD), to include patients with or without a history of transfusions. 27 January 2023
Biotechnology
Histiocytic neoplasms, also known as histiocytosis, are rare disorders in which specialized immune cells (histiocytes) accumulate and form tumors in tissues such as skin, bones, liver, lungs, lymph nodes and spleen.1,2 25 January 2023
Biotechnology
The European Medicines Agency is to review a regulatory submission from Swiss gene editing specialist CRISPR Therapeutics and partner Vertex Pharmaceutical. 25 January 2023
Biotechnology
Boston, USA-based biotech Blueprint Medicines Corporation has announced that the US Food and Drug Administration (FDA) has accepted the company's supplemental new drug application (sNDA) for Ayvakit (avapritinib) to treat indolent systemic mastocytosis (SM). 24 January 2023
Biotechnology
Horizon Therapeutics, a biotech focused on rare, autoimmune and severe inflammatory diseases that is about to be acquired by US biotech major Amgen, has announced an update on its pipeline. 20 January 2023
Biotechnology
The UK health technology assessor, the National Institute for Health and Care Excellence (NICE) has issued a Final Evaluation Document recommending Translarna (ataluren) for reimbursement and use across the National Health Service (NHS) in England and Wales in the treatment of Duchenne muscular dystrophy (DMD). 19 January 2023
Biotechnology
RNAi specialist Alnylam Therapeutics’ Amvuttra (vutrisiran) has been recommended for use on the National Health Service (NHS) in England as an option for treating hereditary transthyretin-related (ATTRv) amyloidosis. 19 January 2023
Biotechnology
Fatal cases of acute liver failure reported in patients treated with Zolgensma (onasemnogene abeparvovec) were raised this week at the European Medicines Agency’s (EMA) Pharmacovigilance Risk Assessment Committee (PRAC). 13 January 2023
Biotechnology
The US Food and Drug Administration’s (FDA) recent approval of TG Therapeutics’ Briumvi (ublituximab) against relapsing forms of multiple sclerosis (MS) is welcome news for the company after suffering a major setback in 2022 when the FDA extended its review of Briumvi by three months. 13 January 2023
Biotechnology
US neuromuscular and cardiac diseases specialist Solid Biosciences and privately-held Dutch biotech Phlox Therapeutics have announced a strategic collaboration focused on genetic cardiac diseases. 12 January 2023
Biotechnology
Californian biotech Jasper Therapeutics has announced that it will focus on the development of its lead product candidate, briquilimab, in chronic diseases and stem cell transplant for rare diseases. 11 January 2023
Biotechnology
Massachusetts, USA-based biotech Selecta Biosciences’ shares were up 11% at $1.37, after it announced an exclusive licensing and development agreement with Japan’s Astellas Pharma for IdeXork (Xork). 10 January 2023
Pharmaceutical
Anglo-Irish company Amryt Pharma has long held ambitions of following in the footsteps of Shire, the rare diseases company that was acquired by Takeda four years ago. 9 January 2023
Pharmaceutical
French pharma firm Ipsen is to acquire the liver disease specialist Albireo, a company focused on the therapeutic potential of bile-acid modulators. 9 January 2023
Biotechnology
US biotech bluebird bio on Friday announced that it has entered into a definitive agreement to sell its second Rare Pediatric Disease Priority Review Voucher (PRV). 7 January 2023
Sign up to receive email updates
Join industry leaders for a daily roundup of biotech & pharma news
Today's issue
Company Spotlight
A US biotech company dedicated to creating oral drugs with an emphasis on indications in virology and immunology.
Copyright © The Pharma Letter 2024 | Headless Content Management with Blaze