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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
Positive Phase III trial results from Belgian pharma major UCB’s two clinical trials in its generalized myasthenia gravis (gMG) program will enable it to become a key future player in the MG market, with both zilucoplan and rozanolixizumab expected to enter the market next year. 20 May 2022
Pharmaceutical
Shares of Denmark’s Zealand Pharma closed up more than 15% at 96.00 kroner, after it announced positive data from its Phase III trial of dasiglucagon in pediatric patients with congenital hyperinsulinism (CHI), a rare pediatric disease that affects mainly newborns, infants and toddlers. 19 May 2022
Biotechnology
US ultra-rare disease specialist Ultragenyx Pharmaceutical and gene and cell therapy company Abeona Therapeutics have announced an exclusive license agreement for AAV gene therapy ABO-102 (now UX111) for the treatment of Sanfilippo syndrome type A (MPS IIIA). 18 May 2022
Biotechnology
Swedish rare disease specialist Hansa Biopharma today revealed that the Swiss Agency for Therapeutic Products (Swissmedic) has granted time-limited market approval for Idefirix (imlifidase) for adult patients with a positive cross-match against an accessible organ from a deceased donor. 13 May 2022
Pharmaceutical
The US Food and Drug Administration has approved Radicava ORS (edaravone) oral suspension for the treatment of adults with amyotrophic lateral sclerosis (ALS). 13 May 2022
Pharmaceutical
Switzerland’s EffRx Pharmaceuticals, Slovenia’s Medis, Russia’s Orpharm, Brazil’s OrphanDC, and Dubai-based Vector Pharma, announced today the newly-formed World Orphan Drug Alliance (WODA). 12 May 2022
Pharmaceutical
Japanese drugmaker Takeda reported a rise in revenue at the end of Japan’s financial year, but saw a significant drop in its profits. 11 May 2022
Biotechnology
The market for T-cell lymphomas (TCLs) – a rare type of the blood cancer group of non-Hodgkin’s lymphomas – is expected to see strong growth at a 6.4% compound annual growth rate (CAGR) over the next decade in the world’s eight major markets (8MM). 11 May 2022
Pharmaceutical
The US Food and Drug Administration’s Center for Drug Evaluation and Research (CDER) has announced the launch of the new Accelerating Rare disease Cures (ARC) Program. 11 May 2022
Biotechnology
Massachusetts, USA-based biotech incubator Flagship Pioneering is to work with diabetes giant Novo Nordisk to create a portfolio of novel research programs. 10 May 2022
Pharmaceutical
Italian drugmaker Recordati saw its shares gain 2.1% to 42.31 euros by early afternoon, as the company released first-quarter 2022 financial results. 10 May 2022
Biotechnology
Belgium's largest drugmaker UCB today announced results from two Phase III studies evaluating its investigational treatments, zilucoplan, a self-administered, subcutaneous (SC) peptide inhibitor of complement component 5 (C5 inhibitor) and rozanolixizumab, an SC-infused monoclonal antibody targeting the neonatal Fc receptor (FcRn) in adults with generalized myasthenia gravis (gMG). 10 May 2022
Biotechnology
Takeda Pharmaceutical has provided an update on an initiative for improving early access to treatments for rare diseases in India. 9 May 2022
Biotechnology
The year 2021 was important for the development of new gene-, cell- and tissue-based therapies, and the Alliance for Regenerative Medicines (ARM) says this year will be another year of growth and progress for these advanced therapies, according to its 2021 annual report. 9 May 2022
Biotechnology
Sino-American biotech BeiGene has announced that the China National Medical Products Administration (NMPA) has granted conditional approval of Blincyto (blinatumomab) for injection for the treatment of pediatric patients with relapsed or refractory (R/R) CD19-positive B-cell precursor acute lymphoblastic leukemia (ALL). 6 May 2022
Biotechnology
Just a week on from the US Food and Drug Administration (FDA) granting approval for Ultomiris (ravulizumab-cwvz) in generalized myasthenia gravis (gMG), the long-acting C5 complement inhibitor has shown its promise in another potential indication. 5 May 2022
Generics
Israel-based generics behemoth Teva Pharmaceutical Industries today reported financial results for the quarter ended March 31, 2022. 3 May 2022
Pharmaceutical
An in-depth look at the French drugmaker's movements on the deal-making front. 3 May 2022
Pharmaceutical
JAMA Neurology has published data from a Phase III trial of Fintepla (fenfluramine), the anti-seizure med developed by Brussels-based drugmaker UCB. 3 May 2022
Pharmaceutical
France-based orphan drug developer Orphalan has received approval of Cuvrior, a new salt of trientine (trientine tetrahydrochloride) by the US Food and Drug Administration. 3 May 2022
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