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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 pharma major AbbVie revealed it has received a notification from the Food and Drug Administration relating to a regulatory delay for its supplemental New Drug Application (sNDA) for Rinvoq in psoriatic arthritis and ankylosing spondylitis. Merck & Co last Tuesday gained European approval for blockbuster immune-oncology drug Keytruda, in the new indication of esophageal or GES carcinoma. USA-based biotech Orchard Therapeutics entered into a strategic collaboration with Dutch firm Pharming on its HSC gene therapy for hereditary angioedema, a life-threatening rare disorder. UK pharma major GlaxoSmithKline on Friday announced an immuno-neurology deal worth a potential $2.2 billion with US biotech Alector, which hopefully will lead to treatments for Alzheimer’s and Parkinson’s diseases. Indian drugmaker Zydus Cadila last week applied for emergency use approval for its ZyCoV-D, a plasma DNA COVID-19 vaccine candidate. 4 July 2021
Biotechnology
Shares of Arrowhead Pharmaceuticals were down 26% at $62.84 mid-morning today, after the US biotech notified regulatory agencies, institutional review boards, and investigators that effective immediately it is voluntarily pausing AROENaC1001, a Phase I/II clinical study of ARO-ENaC. 2 July 2021
Biotechnology
The last decade has seen disease-modifying cystic fibrosis transmembrane conductance regulator (CFTR) modulators transform the treatment paradigm for patients, according to GlobalData, greatly improving quality and longevity of life. 2 July 2021
Biotechnology
The UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) has granted marketing authorization for two new medicines, one from UK-based Diurnal Group and the other from French biotech firm Advicenne. 2 July 2021
Biotechnology
Austria-based rare diseases specialist AOP Orphan Pharmaceuticals announced its latest analysis on Besremi (ropeginterferon alfa-2b) in patients with Polycythemia Vera (PV), a rare blood cancer, from its CONTINUATION-PV study presented at the European Hematology Association (EHA) 2021 meeting by Professor Jean-Jacques Kiladjian from the Saint-Louis Hospital & Université de Paris in France. 1 July 2021
Biotechnology
Parisian biotech GenSight Biologics saw its stock drop 10% in value on Wednesday, after the gene therapist announced more confusing results from its Phase III program. 1 July 2021
Biotechnology
UK and USA-based clinical-stage gene therapy specialist Orchard Therapeutics as entered into a strategic collaboration with Dutch firm Pharming Group to research, develop, manufacture and commercialize OTL-105. 1 July 2021
Biotechnology
Dutch gene therapy company uniQure recently announced the enrolment of the first two patients in the second cohort of a Phase I/II clinical trial of AMT-130, a micro ribonucleic acid (miRNA) gene therapy, in development for Huntington’s disease (HD). 30 June 2021
Pharmaceutical
Adding to a string of distribution deals, UK specialty pharma firm Diurnal Group (AIM: DNL) says it has extended its distribution arrangements with Er-Kim. 29 June 2021
Biotechnology
The European Commission (EC) has approved Enspryng (satralizumab), from Swiss pharma giant Roche, for the treatment of adults and adolescents from 12 years of age living with anti-aquaporin-4 antibody (AQP4-IgG) seropositive neuromyelitis optica spectrum disorder (NMOSD), as a monotherapy or in combination with immunosuppressive therapy (IST). 28 June 2021
Pharmaceutical
For Dravet Syndrome Awareness Month (June), we explore advances in pharmacological treatments for this rare, drug-resistant epilepsy that is characterized by prolonged seizures and fevers that affect one side of the body.1 28 June 2021
Biotechnology
At its June meeting, the European Medicines Agency’s human medicines committee (CHMP) recommended six novel medicines for approval, and issued a negative opinion on one. 26 June 2021
Biotechnology
The Fabry disease (FD) market in the USA and Japan will see significant expansion between 2020 and 2030, as total sales in the two markets are set to rise from $820.15 million in 2020 to $985.81 million in 2030, at a compound annual growth rate (CAGR) of 1.9%. 25 June 2021
Pharmaceutical
Japan’s Ministry of Health, Labor and Welfare (MHLW) has granted regulatory approval for Evrysdi (risdiplam) Dry Syrup 60mg for the treatment of spinal muscular atrophy (SMA). 24 June 2021
Biotechnology
US biotech Fulcrum Therapeutics today announced results from the company’s Phase IIb trial, ReDUX4, in people with facioscapulohumeral muscular dystrophy (FSHD). 24 June 2021
Pharmaceutical
Rare diseases specialist Santhera Pharmaceuticals has announced positive topline results from its long-term Phase IV LEROS study with Raxone (idebenone) in the treatment of Leber’s hereditary optic neuropathy (LHON). 23 June 2021
Biotechnology
AstraZeneca’s Koselugo (selumetinib) has been granted conditional approval in the European Union (EU) for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in pediatric patients with neurofibromatosis type 1 (NF1) aged three years and above. 22 June 2021
Pharmaceutical
Japanese drugmaker Takeda’s $62 billion acquisition of rare disease specialist Shire showed its ambition to be a leader in the space. 21 June 2021
Biotechnology
Having spiked 95% the day before the anticipated positive US regulatory decision on its rare disease treatment arimoclomol, Danish biopharma company Orphazyme fell sharply on Friday (June 18). 21 June 2021
Biotechnology
Japan’s Daichi Sankyo last Monday gained approval to market its malignant glioblastoma candidate, Delytact (teserpaturev) on the domestic market. USA-based Novavax released new Phase III data on its COVID-19 vaccine, giving the firm a leg up in the development program. Belgian biotech iTeos Therapeutics signed a collaboration deal for its anti-TIGIT candidate EOS-448 with UK pharma major GlaxoSmithKline, potentially worth more than $2 billion. US biotech Biogen last Monday released disappointing Phase III results for its investigational rare eye disease drug timrepigene emparvovec. Last Wednesday, the US Food and Drug Administration granted approval for Blueprint Medicines’ Ayvakit as a treatment for systemic mastocytosis. 20 June 2021
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