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
The US Food and Drug Administration has approved Praluent (alirocumab) injection for adult patients with homozygous familial hypercholesterolemia (HoFH), a genetic condition that causes severely high cholesterol. Praluent is not intended to be used alone but instead added to other treatments for HoFH. 2 April 2021
Three years after launching in the UK, Swedish Orphan Biovitrum's Kineret (anakinra) has secured a positive reimbursement decision from the country’s health technology assessor. 1 April 2021
Swiss pharma giant Roche has seen its spinal muscular atrophy (SMA) drug Evrysdi (risdiplam) follow up its US approval by getting the green light from the European Commission (EC). 30 March 2021
The European Medicines Agency’s Committee for Medicinal Products for Human Use recommended five novel medicines for approval at its March 2021 meeting. A final decision on the approval by the European Commission is usually issues in about two-three months. 27 March 2021
Following its March meeting, the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) announced recommendations for the extension of indications on six already approved drugs. 27 March 2021
Life sciences investment trust Syncona has invested $42.3 million UK-based Gyroscope Therapeutics, a clinical-stage gene therapy company focused on diseases of the eye, in an $148.0 million (£107.8 million) Series C financing. 26 March 2021
Medicines to treat infectious diseases and inflammatory disorder, as well as cancers, Alzheimer’s and rare diseases that collectively affect millions of people are under development by pharma and biotech, but the problem now is to deliver them to patients. 26 March 2021
US biotech Vertex Pharmaceuticals has announced that the Australian Therapeutic Goods Administration (TGA) has approved the use of Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for people with cystic fibrosis (CF). 25 March 2021
Italian drugmaker Recordati has presented positive results from the Phase III LINC 4 study of Isturisa (osilodrostat) at the Endocrine Society’s Annual Meeting. 23 March 2021
US oral peptides specialist Chiasma’s shares were up 13% at $3.89 pre-market today, after the company announced the presentation of new positive clinical data from its MPOWERED Phase III trial of Mycapssa (octreotide) at the Endocrine Society’s annual meeting, ENDO 2021, being held virtually March 20-23, 2021. 23 March 2021
According to a summary published by the Hungarian Association of Innovative Pharmaceutical Manufacturers (AIPM) on the occasion of the Rare Disease Day, increasingly effective innovative therapies present a new opportunity for patients. 22 March 2021
China has been relying on daily human growth hormone (HGH) injections to treat growth hormone deficiency (GHD), but this is likely to change, if not soon. 19 March 2021
The US Food and Drug Administration has approved Arcalyst (rilonacept) injection to treat recurrent pericarditis and reduce the risk of recurrence in adults and children 12 years and older. 19 March 2021
Results from the Phase III PEGASUS trial have been published by Nordic rare diseases firm Swedish Orphan Biovitrum, also known as Sobi, and its partner Apellis Pharmaceuticals. 18 March 2021
The US subsidiary of family-owned Italian drugmaker Chiesi Farmaceutici today announced the launch of Bronchitol (mannitol) inhalation powder, an add-on maintenance therapy to improve pulmonary function in cystic fibrosis (CF) patients 18 years of age and older. 17 March 2021
A new strategy for the development of the domestic pharmaceutical sector until 2030 may soon be approved by the Russian government, according to recent statements some senior state officials and local media, reports The Pharma Letter’s local correspondent. 16 March 2021