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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
Armed with promising new data from the Phase III interim analysis, Japan’s largest drugmaker Takeda says it aims to seek marketing authorization for TAK-755 as the first recombinant ADAMTS13 (rADAMTS13) for a disorder with considerable unmet patient need. 6 January 2023
Biotechnology
The US Food and Drug Administration (FDA) yesterday accepted Biologics License Applications (BLAs) from Swiss pharma giant Roche and Belgium’s UCB, and granted them accelerated review. 6 January 2023
Pharmaceutical
UK-based ADVANZ PHARMA has acquired exclusive rights to register and commercialize AT-007, a Phase III development compound for the treatment of several rare neurological diseases, including galactosemia and SORD deficiency, in the European Economic Area, the UK, and Switzerland. 4 January 2023
Biotechnology
Shares of US biotech firm Vera Therapeutics nose-dived in after-hours trading on Tuesday, after it announced what it described as “positive” the Phase IIb ORIGIN clinical trial of atacicept in patients with immunoglobulin A nephropathy (IgAN), which met its primary endpoint. 4 January 2023
Biotechnology
Slightly earlier than expected, US pharma giant Pfizer last week announced positive top-line results from the Phase III BENEGENE-2 study evaluating fidanacogene elaparvovec, an investigational gene therapy, for the treatment of adult males with moderately severe to severe hemophilia B. 3 January 2023
Biotechnology
The Food and Drug Administration (FDA) has now approved TG Therapeutics’ Briumvi (ublituximab-xiiy), for the treatment of relapsing forms of multiple sclerosis (RMS), to include clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease, in adults. 31 December 2022
Biotechnology
The Indian pharma market is increasing at an annual growth rate of 15%, twice the world market growth rate, and has become one of the most expansive sectors in India. While the pharma industry is expected to grow to $130 billion by 2030, there are deep seated challenges posed at this industry when it comes to rare disease management. 30 December 2022
Biotechnology
Paris, France-based neurodegenerative disease specialist Pharnext has attracted a strategic investment from fellow French firm Néovacs. 29 December 2022
Biotechnology
Belgium’s privately-held biotech Novadip Biosciences today announced the appointment of Judy Ashworth as chief medical office (CMO). 28 December 2022
Pharmaceutical
Japan’s largest drugmaker Takeda on Monday announced that in the AURORA trial, a Phase III, multicenter, randomized, double-blind, double-dummy, active-controlled study to assess the efficacy and safety of Livtencity (maribavir) compared to valganciclovir for the treatment of cytomegalovirus (CMV) infection in hematopoietic stem cell transplant (HSCT) recipients, maribavir demonstrated clinically-meaningful efficacy in confirmed CMV viremia clearance. 20 December 2022
Biotechnology
Clinical-stage US biotech Mustang Biotech, which is focused on cell and gene therapies for hematologic cancers, solid tumors and rare genetic diseases, today provided updates on its CAR-T portfolio and 2023 anticipated milestones. 19 December 2022
Biotechnology
The European Commission (EC) has granted marketing authorization for Ebvallo (tabelecleucel)vas a monotherapy for treatment of adult and pediatric patients two years of age and older with relapsed or refractory Epstein Barr virus positive post transplant lymphoproliferative disease (EBV+ PTLD) who have received at least one prior therapy. 19 December 2022
Biotechnology
The European Medicines Agency’s human medicines committee (CHMP) recommended four novel medicines for approval at its December 2022 meeting. A final approval decision from the European Commission can be expected within the next two or three months. 16 December 2022
Biotechnology
The European Commission has granted marketing authorization for Livmarli (maralixibat) oral solution for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) aged two months and older. 15 December 2022
Biotechnology
US RNA company Avidity Biosciences closed 55% higher following Wednesday’s trading. 15 December 2022
Pharmaceutical
Nucleic acid therapy specialist Exicure has been dealt a blow by the termination of collaborations with AbbVie and Ipsen. 15 December 2022
Biotechnology
As the year draws to a close, this article presents some of the wins of the orphan drug community in terms of US FDA approvals, while highlighting challenges faced by the sector and looking ahead to the expectations of the next year. 15 December 2022
Pharmaceutical
Drugmakers Novo Nordisk, Daiichi-Sankyo, Lundbeck, AstraZeneca, UCB and Biogen have been named and shamed by the UK pharma trade group. 12 December 2022
Biotechnology
Among notable research news last week, Swiss pharma giant Novartis released positive Phase III data on its prostate cancer candidate Pluvicto. Also, oncology specialist Mirati Therapeutics last Monday reported an analysis of a Phase III trial with its sitravatinib that was mixed, but the firm intends to press on with the study. A day later, Mirati also published mid-stage results for its lung cancer candidate adagrasib. On the deal-making front, the USA’s Summit Therapeutics saw its shares jump almost 200% as it announced a licensing deal with China’s Akeso for the latter’s cancer candidate ivonescimab that could cost it as much as $5 billion. Also, Vertex entered into a licensing deal for rights to Entrada Therapeutics’ myotonic dystrophy program and its lead candidate ENTR-701. 11 December 2022
Biotechnology
US biotech Vertex Pharmaceuticals, best known as a big player in the cystic fibrosis field, has entered another licensing deal aiming to extend its range of therapeutic interests. 8 December 2022
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