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Shares of French pharma major Sanofi were up more than 2% at 95.05 euros by late morning, as it revealed that the Japanese Ministry of Health, Labor, and Welfare (MHLW) has granted marketing authorization for Xenpozyme (olipudase alfa) for the treatment of adult and pediatric patients with non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD), a rare, progressive, and potentially life-threatening genetic disease. 28 March 2022
Pharmaceutical
Shares of Timber Pharmaceuticals rose as much as 14% to $0.41 yesterday, after the rare and orphan dermatologic diseases announced a late-breaking presentation of a sub-analysis of the Phase IIb CONTROL study that evaluated TMB-001, a topical isotretinoin formulated using the company’s patented IPEG delivery system, at the American Academy of Dermatology (AAD) 2022 Annual Meeting being held in Boston, MA, from March 25-29 26 March 2022
Biotechnology
A feature on the recent fortunes of and prospects for Belgian drugmaker UCB. 25 March 2022
Pharmaceutical
Shares of Danish biopharma firm Orphazyme were down 4.8% at 5.90 kroner by late morning today, after it announced a setback with the regulatory filing for its lead product candidate in Europe. 23 March 2022
Pharmaceutical
Transdermal cannabinoid specialist Zynerba Pharmaceuticals’ pipeline drug Zygel is expected to achieve $18.1 million in sales by 2030, according to GlobalData, due to its potential to help fragile X syndrome (FXS) patients with irritability or aggression symptoms. 23 March 2022
Pharmaceutical
The UK’s medicine costs watchdog the National Institute for Health and Care Excellence (NICE) has published final draft guidance recommending US biotech company BioMarin Pharmaceutical Vimizim (elosulfase alfa) for routine use in the National Health Service (NHS) for the treatment of mucopolysaccharidosis type 4A (also known as MPS 4A and Morquio A syndrome). 22 March 2022
Pharmaceutical
The US Food and Drug Administration on Friday said it has approved Ztalmy (ganaxolone) to treat seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients two years of age and older. 19 March 2022
Biotechnology
Rare diseases specialist Swedish Orphan Biovitrum, also known as Sobi, today announced that China’s National Medical Products Administration (NMPA) has approved Gamifant (emapalumab) for use in China. 18 March 2022
Biotechnology
Chiesi Global Rare Diseases, a business unit of privately-held Italian drugmaker Chiesi Farmaceutici, and Israeli firm Protalix BioTherapeutics today announced final results from the Phase III BRIGHT study evaluating pegunigalsidase alfa (PRX-102) for the treatment of Fabry disease. 18 March 2022
Pharmaceutical
In Germany and France, a new research collaboration will investigate the neurodegenerative disease spinocerebellar ataxia type 3 (SCA3), in collaboration with Servier Laboratories. 18 March 2022
Biotechnology
Alexion, AstraZeneca’s Rare Disease group, has entered into a settlement agreement with Japan’s Chugai Pharmaceutical resolving all patent disputes between the two companies related to Ultomiris (ravulizumab). 17 March 2022
Biotechnology
Krystal Biotech, which is focussed on redosable gene therapies for rare diseases, saw its shares rise as much as 4.6% to $62.61 on Monday, as is said it has reached a binding term sheet with fellow US biotech firm PeriphaGen - and former lab partner - to resolve all claims it copied the latter’s gene therapy technology litigation filed by PeriphaGen on May 20, 2020. 16 March 2022
Biotechnology
Metagenomi, a California, USA-based genetic medicines company with a versatile portfolio of next-generation gene editing tools, today announced the appointment of Christine Foster, as chief business officer and Alan Cohen, as chief medical officer and senior vice president of Monogenic Diseases. 15 March 2022
Pharmaceutical
Japanese firm Mitsubishi Tanabe has submitted to the Ministry of Health, Labor and Welfare in Japan an application for the manufacturing and marketing approval of an investigational oral formulation of edaravone (MT-1186) for the treatment of amyotrophic lateral sclerosis (ALS). 14 March 2022
Biotechnology
US biotech major Biogen today announced new data and updates from its Spinraza (nusinersen) and spinal muscular atrophy (SMA) research program aimed at improving clinical outcomes for people impacted by the disease, including the ASCEND, RESPOND and NURTURE studies. 14 March 2022
Biotechnology
Swiss pharma giant Novartis has announced new data that reinforce the benefit of Zolgensma (onasemnogene abeparvovec), an essential one-time gene therapy treatment that has been approved in many markets to treat spinal muscular atrophy (SMA). 14 March 2022
Biotechnology
Amsterdam-based Scenic Biotech has closed a Series A financing of around $31 million. 10 March 2022
Pharmaceutical
Sentynl Therapeutics, a wholly-owned subsidiary of Indian pharma company Zydus Lifesciences, has announced a deal with US biopharma company BridgeBio Pharma. 9 March 2022
Biotechnology
Midway through its annual report, Massachusetts-based gene therapy company bluebird bio (Nasdaq: BLUE) made the startling admission that it’s running out of cash, fast. 7 March 2022
Biotechnology
Swiss pharma giant Novartis today announced that new data from the ALITHIOS open-label extension study was published in the peer-reviewed Multiple Sclerosis Journal. 2 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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