Rare Diseases
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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
US rare and orphan dermatologic diseases firm Timber Pharmaceuticals saw it shares almost double to $2.79 this morning, on the news of a takeover bid. 21 August 2023
Biotechnology
The US Food and Drug Administration (FDA) on Friday approved Veopoz (pozelimab-bbfg) injection, a complement inhibitor, for the treatment of adult and pediatric patients aged one year and older with CD55-deficient protein-losing enteropathy (PLE), also known as CHAPLE disease. 19 August 2023
Pharmaceutical
Although Latin America has a broad portfolio of innovative medicines to treat orphan diseases and cancer, only 13% of patients who would benefit from these therapies have access to them. 18 August 2023
Biotechnology
French drugmaker Ipsen has won a landmark US approval of Sohonos (palovarotene) for an ultra-rare bone disease, having been rebuffed last month in its efforts to do so in Europe. 17 August 2023
Biotechnology
Privately-held drugmaker Chiesi has secured approval from the British medicines regulator for Elfabrio (pegunigalsidase alfa) in Fabry disease (FD). 16 August 2023
Pharmaceutical
Chiesi Global Rare Diseases, a business unit of privately-held Italian drugmaker Chiesi Farmaceutici established to deliver innovative therapies and solutions for people living with rare diseases, has announced a co-development agreement with Aliada Therapeutics. 16 August 2023
Biotechnology
US rare diseases focused biotech Amicus Therapeutics today revealed that the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has granted marketing authorizations for Pombiliti (cipaglucosidase alfa) + Opfolda (miglustat) for adults living with late-onset Pompe disease (acid α glucosidase [GAA] deficiency). 15 August 2023
Pharmaceutical
Swedish biopharma Calliditas Therapeutics continues to press for full approval of Nefecon/Tarpeyo (budesonide) delayed release capsules/Kinpeygo. 15 August 2023
Pharmaceutical
US developer of rare neurological drugs Harmony Biosciences on Monday revealed it has reached a definitive agreement to acquire Zynerba Pharmaceuticals
. 15 August 2023
Biotechnology
US clinical-stage drug developer Taysha Gene Therapies saw its share price leap 133% to $1.72 yesterday, after it announced a securities purchase agreement for a private placement financing (the PIPE) that is expected to result in gross proceeds of about $150 million, before deducting placement agent commissions and offering expenses. 15 August 2023
Pharmaceutical
Family-owned Italian drugmaker Chiesi Group says it is continuing to enjoy major growth this year. 14 August 2023
Biotechnology
San Diego, USA-based next generation RNA therapeutics company ADARx Pharmaceuticals today revealed it has closed an oversubscribed Series C financing of $200 million. 9 August 2023
Biotechnology
Agios Pharmaceuticals has signed exclusive worldwide license agreement with fellow USA-based Alnylam Pharmaceuticals to acquire the rights to develop and commercialize Alnylam’s novel pre-clinical siRNA targeting TMPRSS6, as a potential disease-modifying treatment for patients with polycythemia vera (PV). 4 August 2023
Biotechnology
US precision genetic rare diseases specialist Sarepta Therapeutics yesterday reported financial results for the second quarter 2023. 3 August 2023
Generics
Israeli generics giant Teva Pharmaceutical Industries made stock market gains of around 12% during Wednesday morning’s trading. 2 August 2023
Pharmaceutical
US biopharma company Vertex Pharmaceuticals has reported consolidated financial results for the second quarter ended June 30, 2023 and updated its full year 2023 financial guidance. 2 August 2023
Biotechnology
Traders looked unfavorably on Apellis Pharmaceuticals stock on Monday, after the company's report into the safety of its newly-approved Syfovre (pegcetacoplan) detailed a small number of retinal vasculitis cases. 1 August 2023
Pharmaceutical
Russian drugmaker Nanolek is aiming to become the largest supplier of drugs for state needs this year, The Pharma Letter’s local correspondent reports 1 August 2023
Pharmaceutical
The European Commission has granted marketing authorization in the European Union (EU) for Ztalmy (ganaxolone) oral suspension. 31 July 2023
Pharmaceutical
Swiss rare diseases specialist Santhera Pharmaceuticals says it has fully divested Raxone (idebenone) for all indications to Italian family-owned drugmaker Chiesi Group, replacing the 2019 agreement between the companies. 31 July 2023
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