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Yesterday, the US Food and Drug Administration (FDA) issued a draft guidance, “Integrating Randomized Controlled Trials for Drug and Biological Products into Routine Clinical Practice” 18 September 2024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
Shares of French biopharma company DBV Technologies (Euronext: DBV) leapt more than 17% to 7.78 euros in early trading, after it revealed receipt of written responses from the US Food and Drug Administration to questions provided in the Type A meeting request the company submitted in October 2020. 15 January 2021
In October 2020, the US Food and Drug Administration issued guidance for industry entitled “Referencing Approved Drug Products in Abbreviated New Drug Application (ANDA) Submissions.” 12 January 2021
US biotech firm BioMarin Pharmaceutical saw its shares close down 9.6% at $81.24 on Monday, after it announced positive top-line results from its ongoing global Phase III GENEr8-1 study of valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A, which has been issued two brand names – Roctavian and Valrox. 12 January 2021
The US Food and Drug Administration has approved a supplemental New Drug Application (sNDA) to add overall survival (OS) and other secondary endpoint data from the Phase III ARAMIS trial to the Nubeqa (darolutamide) Prescribing Information, German pharma and crop sciences major Bayer announced late Friday. 9 January 2021
On Friday, the US Food and Drug Administration’s Center for Drug Evaluation and Research (CDER) announced the approval of the Office of Generic Drugs’ (OGD) reorganization. 9 January 2021
The HELIOS-A Phase III study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis, met its primary and both secondary endpoints at nine months in patients with hATTR amyloidosis with polyneuropathy. 8 January 2021
Despite dealing with the urgent need to evaluate vaccines, drugs and diagnostics tests relating to the COVID-19 pandemic, the US Food and Drug Administration cleared a bumper number of novel drugs last year. 6 January 2021
The US regulator has accepted the New Drug Application for Priority Review submitted for Farxiga (dapagliflozin), raising hopes the diabetes med could become the first SGLT2 inhibitor for people with chronic kidney disease (CKD), with and without type 2 diabetes. 6 January 2021
The US. Food and Drug Administration has accepted for priority review the New Drug Application (NDA) for mirabegron for oral suspension and its supplemental New Drug Application (sNDA) for Myrbetriq (mirabegron) tablets for the treatment of neurogenic detrusor overactivity (NDO) in pediatric patients aged three years and older. 6 January 2021
Specialty biopharma Marius Pharmaceuticals has submitted a New Drug Application (NDA) to the US Food and Drug Administration (FDA) seeking approval of Kyzatrex, its oral testosterone undecanoate soft gelatin capsule for the treatment of primary and secondary hypogonadism in men. 5 January 2021
Swiss pharma giant Roche’s novel cancer immunotherapy tiragolumab has been granted Breakthrough Therapy designation (BTD) by the US Food and Drug Administration, in combination with Tecentriq (atezolizumab). 5 January 2021
The US Food and Drug Administration has weighed in on the question of coronavirus vaccine dosing, as health experts and the general public alike discuss the best way to optimise limited supplies. 5 January 2021
Chinese company Hutchison China MediTech has initiated the filing of a New Drug Application (NDA) to the US Food and Drug Administration (FDA) for surufatinib. 29 December 2020
Adding more feathers to their cap in recent months, Indian drug majors have garnered 45% of all new Abbreviated New Drug Application (ANDA) approvals over the past nine months. 29 December 2020
The US Food and Drug Administration has accepted for Priority Review the supplemental New Drug Application (sNDA) for Lorbrena (lorlatinib) as a first-line treatment for people with anaplastic lymphoma kinase (ALK)-positive metastatic non-small cell lung cancer (NSCLC). 29 December 2020
The US Food and Drug Administration has approved the New Drug Application (NDA) for once-daily Gemtesa (vibegron), a beta-3 adrenergic receptor (β3) agonist, for the treatment of overactive bladder (OAB) with symptoms of urge urinary incontinence (UUI), urgency, and urinary frequency in adults, from Urovant Sciences. 24 December 2020
Netherlands-based gene therapy company uniQure saw its shares down 15.7% at $38.75 around midday in New York trading today, after it announced that its hemophilia B gene therapy program, including the pivotal, Phase III HOPE-B study, has been placed on clinical hold by the US Food and Drug Administration. 21 December 2020