The European Commission has conditionally approved the marketing application submitted by Ipsen for Iqirvo (elafibranor) 80mg tablets for the treatment of primary biliary cholangitis (PBC) 21 September 2024
Today, the US Food and Drug Administration approved Miplyffa (arimoclomol), an oral medication for the treatment of Neimann-Pick disease, type C (NPC). 20 September 2024
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
Anglo-Swedish drug major AstraZeneca has entered a definitive agreement with Genzyme, a Sanofi subsidiary to divest rare disease medicine Caprelsa (vandetanib). 27 July 2015
Australia’s Minister for Health Sussan Ley has approved a life-saving drug that will ensure adult Australian Pompe patients receive treatment that previously would have cost several hundred thousand dollars at no out-of-pocket cost. 27 July 2015
In a second major deal for the Indian drugmaker within two days, Lupin said on Friday it has entered into a strategic asset purchase agreement with Temmler Pharma, a part of the Aenova Group. 26 July 2015
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended granting a marketing authorization for Ireland-headquartered Shire’s (LSE: SHP) Intuniv (guanfacine). 26 July 2015
US health care giant Johnson & Johnson’s psoriasis drug, Stelara (ustekinumab), has progressed into Phase III development for axial spondyloarthritis (axSpA) and, following its anticipated approval, is forecast to achieve impressive axSpA sales of $244.7 million in 2024,. 23 July 2015
New Zealand’s Pharmaceutical Management Agency PHARMAC is seeking feedback on a proposal to list Firazyr (icatibant) resulting from a provisional agreement formed between the local subsidiary of Ireland-headquartered Shire and PHARMAC. 21 July 2015
Dutch biotech firm Pharming Group and the UK’s Clinigen Group are collaborating to provide access to Pharming’s Ruconest (conestat alfa) and say the program is now live. 21 July 2015
Belgian clinical-stage biotech company Galapagos NV has entered into a licensing agreement with the HUB Foundation for use of Organoid Technology for preclinical research in cystic fibrosis (CF) and inflammatory bowel disease (IBD). 20 July 2015
The Ensuring Access to Clinical Trials Act of 2015, which was introduced by Senators Ron Wyden (Democrat, Oregon), Orrin Hatch (Republican, Utah), Edward Markey (Democrat, Maryland) and Sherrod Brown (Democrat, Ohio) and co-sponsored by an additional 12 Senators, was passed yesterday. 17 July 2015
Isis Pharmaceuticals says it has earned a $2.15 million milestone payment from Biogen related to advancing the ongoing pivotal Phase III study (CHERISH) evaluating ISIS-SMNRx in children with spinal muscular atrophy (SMA). 15 July 2015
Celsus Therapeutics has reached agreement to acquire privately held Swiss biotech firm Volution Immuno Pharmaceuticals in an all-stock transaction. 13 July 2015
The European Commission has approved Janssen-Cilag’s Imbruvica (ibrutinib) capsules as a treatment option for adult patients with Waldenström's macroglobulinemia (WM). 10 July 2015
Japan’s Ministry of Health, Labor and Welfare has approved the New Drug Application from US pharma company Alexion for Strensiq (asfotase alfa) as a treatment for hypophosphatasia (HPP). 7 July 2015
Akcea Therapeutics says that the US Food and Drug Administration has granted Orphan Drug designation to volanesorsen (ISIS-APOCIIIRx) for the treatment of patients with familial chylomicronemia syndrome (FCS). 6 July 2015
The US Food and Drug Administration yesterday approved the first drug for cystic fibrosis (CF) directed at treating the underlying cause of the disease in people who have two copies of a specific mutation. 3 July 2015
US RNA-based drug developer Sarepta Therapeutics says it has completed the rolling submission of a New Drug Application to the US Food and Drug Administration for eteplirsen on June 26, 2015. 29 June 2015
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has adopted positive opinions recommending marketing authorization of US drugmaker Alexion Pharmaceuticals’ Strensiq (asfotase alfa) and Kanuma (sebelipase alfa). 26 June 2015
US biotech firm GlycoMimetics says it is about to get a milestone payment from pharma giant Pfizer (NYSE: PFE) under its rare diseases collaboration worth a potential $340 million (The Pharma Letter October 12, 2011). 24 June 2015
The Russian Parliament (State Duma) is considering adopting a bill allowing unregistered imports of some types of drugs into Russia, reports the Pharma Letter’s local correspondent. 22 June 2015