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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
Pharmaceutical
Advicenne, a French firm specializing in rare renal diseases, has signed an exclusive distribution agreement with Swedish pharma company FrostPharma for the commercialization of Sibnayal (potassium citrate/potassium hydrogen carbonate). 4 January 2022
Biotechnology
Shares of Swiss biotech Santhera Pharmaceuticals were up 15.4% at 1.50 Swiss francs by early afternoon, after it revealed an exclusive license agreement with China-based rare diseases specialist Sperogenix Therapeutics. 4 January 2022
Biotechnology
Shares in New York-based rare disease firm Applied Therapeutics have fallen by a third, after the company said it would put a US regulatory bid on hold. 4 January 2022
Pharmaceutical
The US Court of Appeals for the Federal Circuit (CAFC) issued its decision upholding the validity of US Patent No 9,187,405 covering a dosing regimen for Gilenya (fingolimod). 4 January 2022
Pharmaceutical
US specialty pharma company Xeris Biopharma saw its share leap nearly 30% to $3.15 post-market on Thursday, after it announced that the US Food and Drug Administration had approved its Recorlev (levoketoconazole) for the treatment of endogenous hypercortisolemia in adult patients with Cushing’s syndrome for whom surgery is not an option or has not been curative. 31 December 2021
Pharmaceutical
Russia will simplify the procedure for imports of unregistered drugs against serious diseases starting from March of next year, according to recent statements by state authorities and local media reports. 28 December 2021
Pharmaceutical
Johnson & Johnson’s pharma arm Janssen has won a positive reimbursement decision in the UK, from the country’s health technology assessor. 24 December 2021
Biotechnology
German pharmaceutical firm Boehringer Ingelheim has published new data from the pivotal Phase II Effisayil trial in the New England Journal of Medicine. 23 December 2021
Biotechnology
The US regulator has placed a clinical trial hold on research into a Duchenne muscular dystrophy (DMD) candidate under development by New York-based pharma giant Pfizer. 22 December 2021
Biotechnology
The Russian Ministry of Health has approved the use of Zolgensma (onasemnogene abeparvovec), the world’s most expensive drug against spinal muscular atrophy (SMA) in the domestic market, reports The Pharma Letter’s local correspondent. 22 December 2021
Biotechnology
AstraZeneca has announced that a supplemental Biologics License Application (sBLA) for Ultomiris (ravulizumab-cwvz) in adults with generalised myasthenia gravis (gMG) has been accepted for Priority Review by the US Food and Drug Administration. 21 December 2021
Biotechnology
In March 2021, the US Food and Drug Administration (FDA) announced its continued support for rare disease programs.1 So far, giant strides have been made in the search to find cures for diseases that affect a small percentage of the population, but are devastating in effect. 21 December 2021
Pharmaceutical
German pharma and life sciences group Merck KGaA today announced a strategically focused expansion of its neurology pipeline with the acquisition of the rights to develop cladribine (marketed as Mavenclad for multiple sclerosis) for the treatment of generalized myasthenia gravis (gMG) and neuromyelitis optica spectrum disorder (NMOSD). 20 December 2021
Biotechnology
The US Food and Drug Administration (FDA) has approved Vyvgart (efgartigimod alfa-fcab) for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive, said Belgo-Dutch clinical-stage immune-oncology biotech argenx. 20 December 2021
Biotechnology
French biotech InnoSkel has been awarded an Innovation Passport under the UK Medicines and Healthcare products Regulatory Agency’s (MHRA) Innovative Licensing and Access Pathway (ILAP). 16 December 2021
Biotechnology
By 2030, patients living with Duchenne muscular dystrophy (DMD) - a genetic muscle wasting disease caused by mutations on the gene that produces dystrophin - will have greater choice when it comes to the drug types used for treatment. 14 December 2021
Biotechnology
Anders Ullman, currently a member of the board of directors at Swedish Orphan Biovitrum, also known as Sobi, will become head of R&D and chief medical officer and replace Dr Ravi Rao. 13 December 2021
Biotechnology
Regulatory news featured last week, with US drugmaker AbbVie announcing that the US Food and Drug Administration is now requiring its Rinvoq to carry a Boxed Warning, following the agency’s identification of safety issues with the JAK inhibitor class of drugs for rheumatoid arthritis. Also, US biotech Reata Pharmaceuticals suffered a setback last Wednesday, when an FDA advisory committee voted against approval of bardoxolone for Alport-caused kidney disease. On the deal-making front, Ionis entered into an agreement with the UK’s AstraZeneca to develop its rare disease drug eplontersen, that could earn the company $3.6 billion. Neurocrine Biosciences last Tuesday released Phase III data showing positive results for its Huntington disease candidate Ingrezza. 12 December 2021
Biotechnology
Belgium’s largest drugmaker UCB has announced positive top-line results from the Phase III MycarinG (MG0003) trial study investigating the efficacy and safety of rozanolixizumab, a subcutaneously infused monoclonal antibody targeting the neonatal Fc receptor (FcRn), versus placebo in patients with generalized myasthenia gravis (gMG). 10 December 2021
Biotechnology
The UK's National Institute for Health and Care Excellence (NICE) has issued the Final Appraisal Decision (FAD) recommending the use of Aspaveli (pegcetacoplan) in adults with paroxysmal nocturnal hemoglobinuria (PNH) in England and Wales. 9 December 2021
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