Applied Therapeutics saw its stock price climb by over two-thirds after an encouraging update on the US Food and Drug Administration review of govorestat. 19 September 2024
TG Therapeutics yesterday shared new five-year data from the ULTIMATE I & II Phase III trials evaluating Briumvi (ublituximab-xiiy) in patients with relapsing forms of multiple sclerosis (RMS). 19 September 2024
Canada’s NanoVation Therapeutics, a privately-held platform company developing innovative technologies to overcome the barriers of nucleic acid delivery, has announced a multi-year partnership with Danish drugmaker Novo Nordisk. 18 September 2024
AstraZeneca’s Fasenra (benralizumab) has been approved in the USA for the treatment of adult patients with eosinophilic granulomatosis with polyangiitis (EGPA, the UK pharma major announced today. 18 September 2024
US biotech Capricor Therapeutics has entered into a binding term sheet with Japanese drugmaker Nippon Shinyaku for the commercialization and distribution in Europe of Capricor’s lead asset, deramiocel. 17 September 2024
Dystonia is a neurological movement disorder characterized by involuntary muscle contractions that cause repetitive movements, abnormal postures, or twisting. 17 September 2024
The US Food and Drug Administration (FDA) has approved Ocrevus Zunovo (ocrelizumab and hyaluronidase-ocsq) for the treatment of relapsing multiple sclerosis (RMS) and primary progressive multiple sclerosis (PPMS), from Swiss pharma giant Roche. 16 September 2024
The Gastrointestinal Drugs Advisory Committee (GIDAC) of the US Food and Drug Administration (FDA) met on Friday to discuss Intercept Pharmaceuticals’ supplemental New Drug Application (sNDA) for Ocaliva (obeticholic acid, OCA). 16 September 2024
Boehringer Ingelheim today announced that the FIBRONEER-IPF trial met its primary endpoint, which was the absolute change from baseline in Forced Vital Capacity [mL] (FVC) at week 52 versus placebo. FVC is a measure of lung function. 16 September 2024
Oligonucleotide therapeutics are rapidly emerging as a promising class of drugs capable of addressing a variety of genetic and complex diseases by targeting RNA sequences.1 16 September 2024
Thursday was a wretched day for Fulcrum Therapeutics, a US biopharma company focused on developing small molecules to improve the lives of patients with genetically defined rare diseases. 13 September 2024
California, USA-based biotech BridgeBio Pharma saw its shares dip 4% to $29.60 yesterday on disappointing clinical results and the company’s decision to drop an early-stage candidate. 11 September 2024
Santhera Pharmaceuticals has signed an exclusive distribution agreement with GENESIS Pharma for Agamree (vamorolone) for the treatment of Duchenne muscular dystrophy (DMD) in 20 markets in Central and Eastern Europe. 10 September 2024
Cartesian Therapeutics today announced that the US Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation to Descartes-08 for the treatment of juvenile dermatomyositis (JDM). 9 September 2024
Last week’s research news included French pharma major Sanofi releasing mixed Phase III results for it tolebrutinib in progressive multiple sclerosis. Also of note, US vaccine developer Vaxcyte announced strong early-stage trial results for its VAX-31 in pneumococcal disease. On the regulatory front, US drugmaker Travere Therapeutics gained full approval from the US Food and Drug Administration (FDA) for its kidney disease drug Filspari. US cancer drug developer IN8bio announced a prioritization of its R&D pipeline to focus on INB-100 in acute myeloid leukemia, along with a workforce cutback. 8 September 2024
The Institute for Clinical and Economic Review (ICER) has posted its revised Evidence Report assessing the comparative clinical effectiveness of three medicines for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM). 6 September 2024
San Diego, USA-based Travere Therapeutics saw its shares close up more than 8% at $10.75 yesterday on positive news form the US regulator. 6 September 2024
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
Japanese companies JCR Pharmaceuticals and Sumitomo Dainippon Pharma have agreed a marketing alliance for the Fabry disease treatment agalsidase beta BS IV infusion at 5mg and 35mg in Japan. 2 March 2022
Shares of Ireland-headquartered rare diseased biotech Amryt Pharma fell as much as 31% yesterday, after it announced the receipt of a Complete Response Letter (CRL) from the US Food and Drug Administration regarding its New Drug Application (NDA) for Oleogel-S10. 1 March 2022