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
Vanda Pharmaceuticals’ shares were down more than 10% at $4.45 pre-market today, as the) company provided an unfavorable update on its tradipitant development program. 19 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
Pharma giant Merck & Co has announced that the US Food and Drug Administration (FDA) has approved Keytruda (pembrolizumab), alongside pemetrexed and platinum chemotherapy, for the first-line treatment of adults with unresectable advanced or metastatic malignant pleural mesothelioma (MPM). 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
Yesterday, the US Food and Drug Administration (FDA) issued a draft guidance, “Integrating Randomized Controlled Trials for Drug and Biological Products into Routine Clinical Practice” 18 September 2024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
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
On Thursday, the US Food and Drug Administration released a Drug Safety Communication warning that Veozah (fezolinetant), which is used to treat hot flashes due to menopause, can cause rare but serious liver injury. 13 September 2024
The US Food and Drug Administration (FDA) has approved Tecentriq Hybreza (atezolizumab and hyaluronidase-tqjs), the first and only PD-(L)1 inhibitor for subcutaneous (SC) injection for patients in the USA, Swiss pharma giant Roche subsidiary Genentech announced yesterday. 13 September 2024
The US Food and Drug Administration (FDA) has approved Tremfya (guselkumab) for the treatment of adults with moderately to severely active ulcerative colitis (UC). 12 September 2024
Intercept Pharmaceuticals’ Ocaliva (obeticholic acid) will come under the microscope when the US Food and Drug Administration’s (FDA) Gastrointestinal Drugs Advisory Committee meets on Friday. 12 September 2024
The US Food and Drug Administration (FDA) yesterday announced revised final guidance for industry entitled, “ANDA Submissions – Amendments to Abbreviated New Drug Applications under GDUFA.” 12 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
ProKidney announced strategic updates to its Phase III program for rilparencel, an investigational treatment to potentially preserve kidney function in patients with type 2 diabetes and advanced chronic kidney disease (CKD). 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
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 released a briefing document ahead of a planned committee meeting on Tuesday, which will consider authorization of Pfizer (NYSE: PFE) and BioNTech’s (Nasdaq: BNTX) coronavirus vaccine in younger children. 25 October 2021
US immune-oncology focussed biotech Agenus said Friday it has withdrawn its Biologics License Application (BLA) for balstilimab, its PD-1 inhibitor. 23 October 2021
The US Food and Drug Administration has approved Susvimo (ranibizumab) 100mg/mL for intravitreal use via ocular implant for the treatment of people with neovascular or “wet” age-related macular degeneration (nAMD) who have previously responded to at least two anti-vascular endothelial growth factor (VEGF) injections. 23 October 2021
Indian generic drugs major Dr Reddy’s Laboratories has received final approval from the US Food and Drug Administration of its Abbreviated New Drug Application (ANDA) for lenalidomide Capsules, in 2.5mg and 20mg strengths, and tentative approval for 5mg, 10mg, 15mg, and 25mg strengths. 22 October 2021
The US Food and Drug Administration has approved Dupixent (dupilumab) as an add-on maintenance treatment of patients aged six to 11 years with moderate-to-severe asthma characterized by an eosinophilic phenotype or with oral corticosteroid-dependent asthma. 21 October 2021
Looking ahead to the potential implications of Biogen’s landmark approval in Alzheimer’s (AD), industry analyst GlobalData has conducted research into the market potential for a next-generation candidate. 21 October 2021
The US Food and Drug Administration said yesterday that it has taken action to expand the use of a booster dose for COVID-19 vaccines in eligible populations for vaccine developed by Johnson & Johnson and Moderna. 21 October 2021
Following recent discussions with the US regulator, Sage Therapeutics and partner Biogen said yesterday they plan to submit a New Drug Application (NDA) to the US Food and Drug Administration for zuranolone (SAGE-217), an investigational two-week, once-daily therapeutic, in the second half of 2022. 20 October 2021
Shares of Omeros Corporation plummeted nearly 27% to $5.67 by close of trading Monday, as the US biotech revealed another setback in its development of narsoplimab. 19 October 2021
Oyster Point Pharma saw its shares gain almost 12% to $15.30 pre-market today, as it revealed that the US the Food and Drug Administration has approved Tyrvava (varenicline solution) Nasal Spray 0.03mg for the treatment of the signs and symptoms of dry eye disease. 18 October 2021
The US Food and Drug Administration approved the supplemental Biologics License Application (sBLA) for Cyltezo (adalimumab-adbm), from German family-owned pharma major Boehringer Ingelheim, as the first Interchangeable biosimilar with AbbVie’s mega-blockbuster Humira (adalimumab). 18 October 2021
One of the latest developments in medicine is the advent of cell and gene therapies (CGTs). Cell therapies include cellular immunotherapies, cancer vaccines, and stem cells. Gene therapies are designed to modify the expression of specific genes or alter the biological properties of cells for the treatment of diseases. 18 October 2021
Significant regulatory news last week included Swiss pharma giant Roche gaining Breakthrough Therapy designation from the US Food and Drug Administration for its Alzheimer’s candidate gantenerumab. Also, Eli Lily last Wednesday was granted FDA approval for its CDK 4/6 inhibitor Verzenio in an early breast cancer indication. Swiss biotech Idorsia suffered a setback last Monday when the firm reported that its Fabry disease hopeful lucerastat missed endpoints in a Phase III trial. An FDA advisory panel last Thursday voted in favor of approving Moderna’s COVID-19 vaccine – Spikevax - to be used for booster jabs. And on Friday the AdCom also backed booster jabs of Johnson & Johnson’s vaccine. 17 October 2021
The US Food and Drug Administration’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) unanimously voted 19-0 to recommend Emergency Use Authorization (EUA) for a booster dose of the Johnson & Johnson COVID-19 vaccine for adults aged 18 and older at least two months following initial vaccination with the single-shot vaccine, the company stated. 16 October 2021
The US regulator is expected to grant an Emergency Use Authorization (EUA) for the use of a booster shot of the coronavirus vaccine developed by Moderna. 15 October 2021
The US Food and Drug Administration has accepted a supplemental New Drug Application (sNDA) for the treatment of schizophrenia in adolescents with Rexulti (brexpiprazole) and has granted the drug’s makers – Japan’s Otsuka Pharmaceutical Denmark’s Lundbeck Priority Review. 14 October 2021
Shares in US drugmaker Eli Lilly were 1% higher following Wednesday morning's trading after the US Food and Drug Administration’s (FDA) approval of Verzenio (abemaciclib) in an early breast cancer indication. 13 October 2021
Notching up another indication for its mega-blockbuster anti-PD-1 therapy, the US Food and Drug Administration has now approved Merck & Co’s Keytruda (pembrolizumab) in combination with chemotherapy, with or without bevacizumab, for patients with persistent, recurrent or metastatic cervical cancer whose tumors express PD-L1 (CPS ≥1), as determined by an FDA-approved test. 13 October 2021
Protagonist Therapeutics saw its share leap nearly 100% to $35 after the company revealed that the US Food and Drug Administration has lifted the full clinical hold on its rusfertide clinical studies, announced on September 17, 2021. 13 October 2021
The US Food and Drug Administration (FDA) has approved Rethymic (allogeneic processed thymus tissue‐agdc), a one‐time regenerative tissue‐based therapy for immune reconstitution in pediatric patients with congenital athymia. 11 October 2021