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Biotechnology
Swiss rare diseases specialist Santhera Pharmaceuticals has appointed Shabir Hasham as chief medical officer (CMO) and a member of its executive management team, effective May 1. 13 April 2022
Biotechnology
Japan’s largest drugmaker Takeda today announced that the Phase III SHP643-301 study evaluating the safety profile and pharmacokinetics (PK) of Takhzyro (lanadelumab) in patients aged two to 13 April 2022
Pharmaceutical
The Delhi High Court has issued a permanent injunction against two Indian companies protesting Novartis patent for eltrombopag olamine, an active pharmaceutical ingredient in its brand Revolade, till May 21, 2023. 13 April 2022
Biotechnology
Unofficial US drug price watchdog the Institute for Clinical and Economic Review (ICER) has published a new white paper in collaboration with researchers at NORC at the University of Chicago. 11 April 2022
Biotechnology
The UK's National Institute for Health and Care Excellence (NICE) has recommended AstraZeneca and Merck & Co’s Koselugo (selumetinib) for use by the National Health Service (NHS) in England. 6 April 2022
Biotechnology
Swiss pharma giant Novartis today announced the US Food and Drug Administration has granted accelerated approval to Vijoice (alpelisib) for the treatment of adult and pediatric patients years of age and older with severe manifestations of PIK3CA-Related Overgrowth Spectrum (PROS) who require systemic therapy. 6 April 2022
Biotechnology
The latest results from the Phase III CHAMPION-MG trial open-label extension showed that Ultomiris (ravulizumab-cwvz) demonstrated long-term efficacy in adults with anti-acetylcholine receptor (AChR) antibody-positive generalised myasthenia gravis (gMG). 6 April 2022
Biotechnology
Shares of US drug developer Imara closed down 22% at $1.27 yesterday having tanked as much as 34%, after it announced disappointing results from interim analyses of its Ardent Phase IIb clinical trial of tovinontrine (IMR-687) in patients with sickle cell disease (SCD) and Forte Phase IIb clinical trial of tovinontrine in patients with beta-thalassemia. 6 April 2022
Biotechnology
Israeli pharma company Protalix BioTherapeutics and family-owned Italian drugmaker Chiesi have announced positive top-line results from the 24-Month Phase III BALANCE trial of PRX-102 (pegunigalsidase alfa) in Fabry disease. 5 April 2022
Biotechnology
Coming as no surprise given the company recently admitted it was running out of cash, US biotech firm bluebird bio today announced that it is initiating a comprehensive restructuring intended to deliver up to $160 million in cost savings over the next two years. 5 April 2022
Biotechnology
Multiple sclerosis (MS) treatments developed by Biogen (Nasdaq: BIIB) have featured in data presentations at the annual congress of the American Academy of Neurology (AAN). 5 April 2022
Biotechnology
The US Food and Drug Administration has extended the review timeline of the New Drug Application (NDA) for vutrisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis, to allow for the review of newly added information related to the new secondary packaging and labeling facility. 4 April 2022
Biotechnology
German life sciences major Bayer underlined its determination to drive breakthrough innovations during an event on Friday. 2 April 2022
Pharmaceutical
Privately-held Italian drugmaker Chiesi has reported strong financial results for 2021, with revenues of 2.42 billion euros ($2.65 billion), an increase of 8.6% from the year before. 1 April 2022
Biotechnology
Positive Phase II/III data for leniolisib have been presented at the annual congress of the Clinical Immunology Society (CIS 2022). 1 April 2022
article
The Ministry of Health has begun collecting data on drugs that are not produced in Russia as well as a number of “friendly” countries as part of its efforts to prevention their shortage in the domestic market, according to recent statements by an official spokesman of the Ministry and local media, reports The Pharma Letter’s local correspondent. 30 March 2022
Biotechnology
Japan’s largest drugmaker Takeda says it has received approval from the Ministry of Health, Labor and Welfare (MHLW) for Takhzyro (lanadelumab) subcutaneous injection 300mg syringes for prophylaxis against acute attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years of age and older in Japan. 29 March 2022
Pharmaceutical
March is Wilson disease awareness month – an autosomal recessive genetic disorder characterized by excess copper accumulation in the body.1 It is caused by a mutation in the ATP7B gene, with an estimated one in 90 people carrying the mutation.1 29 March 2022
Pharmaceutical
East and West Coast-based American firms Biogen and Ionis Pharmaceuticals (Nasdaq: IONS) are dropping BIIB078, an amyotrophic lateral sclerosis (ALS) candidate. 29 March 2022
Biotechnology
New York-based cancer giant Bristol Myers Squibb has announced that the US Food and Drug Administration has extended the review of its supplemental biologics license application (sBLA) for Reblozyl (luspatercept-aamt). 28 March 2022
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
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