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Yesterday, the US Food and Drug Administration (FDA) issued a draft guidance, “Integrating Randomized Controlled Trials for Drug and Biological Products into Routine Clinical Practice” 18 September 2024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
Liminal BioSciences has filed a resubmission of the Biologics License Application (BLA) for Ryplazim (plasminogen) with the US Food and Drug Administration for the treatment of congenital plasminogen deficiency (C-PLGD). 8 September 2020
The US Food and Drug Administration (FDA) has approved Gavreto (pralsetinib) for the treatment of adult patients with metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC) as detected by an FDA approved test. 7 September 2020
Kite today announced that it has submitted a supplemental Biologics License Application (sBLA) to the US Food and Drug Administration for Yescarta (axicabtagene ciloleucel). 4 September 2020
The US Food and Drug Administration (FDA) has approved Onureg (azacitidine 300mg tablets, CC-486) for the continued treatment of adult patients with acute myeloid leukemia (AML). 2 September 2020
US Food and Drug Administration (FDA) Commissioner Stephen Hahn finds himself walking a tightrope between bowing to the political pressure to approve a COVID-19 vaccine and waiting till late-stage trial data proves that the product is safe and effective. 1 September 2020
On Friday, as part of its ongoing efforts to fight COVID-19, the US Food and Drug Administration broadened the scope of the existing emergency use authorization for Veklury (remdesivir) to include treatment of all hospitalized adult and pediatric patients with suspected or laboratory-confirmed COVID-19, irrespective of their severity of disease. 29 August 2020
The first novel medicine for acne in decades has been approved in the USA, lifting shares in Italian developer Cassiopea 16% on Thursday, and a further 6% on Friday morning. 28 August 2020
Based on a US Food and Drug Administration review of new data from three clinical trials, the agency has removed the Boxed Warning about amputation risk from the diabetes medicine canagliflozin. 27 August 2020
Trilaciclib’s new drug application (NDA) acceptance signals a significant moment for cancer patients that need support with chemotherapy-induced toxicities - further highlighted by the fact that the therapy has been granted Breakthrough Therapy designation by the US Food and Drug Administration. 26 August 2020
Recordati Rare Diseases, a subsidiary of Italy’s Recordati Farmaceutici, says that the US Food and Drug Administration (FDA) has approved Cystadrops (cysteamine ophthalmic solution) 0.37%. 26 August 2020
Chicago’s AbbVie has submitted to the US regulator for approval to market Rinvoq (upadacitinib) for people with active ankylosing spondylitis (AS). 25 August 2020
Shares in San Franciscan biotech Tricida fell by a quarter on Monday, after the firm announced the US regulator had rebuffed a submission for veverimer (TRC101). 25 August 2020
The US Food and Drug Administration on Sunday issued an emergency use authorization (EUA) for investigational convalescent plasma for the treatment of COVID-19 in hospitalized patients as part of the agency’s ongoing efforts to fight COVID-19. 24 August 2020
US biotech major Gilead Sciences and co-developer Belgium-based Galapagos received a big shock following the Food and Drug Administration’s complete response letter (CRL) for its rheumatoid arthritis (RA) candidate filgotinib earlier this week. 21 August 2020
Ireland-headquartered Alkermes has announced that a tentative date has been set for a US Food and Drug Administration (FDA) advisory committee virtual review of the New Drug Application (NDA) for ALKS 3831 (olanzapine/samidorphan). 21 August 2020
The US Food and Drug Administration has approved Kesimpta (ofatumumab, formerly OMB157) as an injection for subcutaneous use for the treatment of relapsing forms of multiple sclerosis (RMS), to include clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease, in adults. 21 August 2020
The US Food and Drug Administration has approved the expansion of the Kyprolis (carfilzomib) US prescribing information to include its use in combination with Darzalex (daratumumab) plus dexamethasone (DKd) in two dosing regimens — once weekly and twice weekly — for the treatment of patients with relapsed or refractory multiple myeloma (R/R MM) who have received one to three previous lines of therapy. 21 August 2020
The US Food and Drug Administration yesterday published a revision to the Manual of Policies and Procedures (MAPP 5241.2), “Consolidation of ANDAs by the Office of Generic Drugs.” 20 August 2020