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Yesterday, the US Food and Drug Administration (FDA) issued a draft guidance, “Integrating Randomized Controlled Trials for Drug and Biological Products into Routine Clinical Practice” 18 September 2024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
Californian cytokine storm syndrome specialist Humanigen on Friday announced it has submitted an application to the US Food and Drug Administration (FDA) requesting Emergency Use Authorization (EUA) for lenzilumab for the treatment of patients hospitalized with COVID-19. 1 June 2021
The US Food and Drug Administration approved Lumakras (sotorasib: AMG 510) as the first treatment for adult patients with non-small cell lung cancer (NSCLC) whose tumors have a specific type of genetic mutation called KRAS G12C and who have received at least one prior systemic therapy. 29 May 2021
The US Food and Drug Administration has granted accelerated approval to Truseltiq (infigratinib), a kinase inhibitor for adults with previously treated, unresectable locally-advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement as detected by an FDA-approved test. 29 May 2021
Eli Lilly has received a subpoena from the US Department of Justice (DoJ) requesting the production of certain documents relating to its manufacturing site in Branchburg, New Jersey. 28 May 2021
The US Food and Drug Administration has approved Nurtec ODT (rimegepant 75mg) for the preventive treatment of migraine, thus the drug becomes the first oral CGRP antagonist option to both prevent and treat migraines. 28 May 2021
The US Food and Drug Administration yesterday approved Bristol Myers Squibb’s Zeposia (ozanimod) 0.92mg for the treatment of adults with moderately to severely active ulcerative colitis (UC), a chronic inflammatory bowel disease (IBD). 28 May 2021
Myovant Sciences and Pfizer have announced that the US Food and Drug Administration (FDA) has approved Myfembree (relugolix/estradiol/norethindrone acetate), the first once-daily treatment for the management of heavy menstrual bleeding associated with uterine fibroids in premenopausal women, with a treatment duration of up to 24 months. 27 May 2021
The US Food and Drug Administration has granted an Emergency Use Authorization (EUA) for sotrovimab (previously VIR-7831), an investigational single-dose monoclonal antibody, for the treatment of mild-to-moderate COVID-19. 27 May 2021
The US regulator will consider a submission for novel cancer therapy ciltacabtagene autoleucel (cilta-cel) under the agency’s Priority Review scheme, with a decision expected by November 29. 27 May 2021
The US Food and Drug Administration announced on Wednesday that it is restricting the use of US biotech firm Intercept Pharmaceuticals’ liver disease medicine Ocaliva (obeticholic acid) in patients having primary biliary cholangitis (PBC) with advanced cirrhosis of the liver because it can cause serious harm. 27 May 2021
Shares in New Jersey, USA-based company Provention Bio rose by over a quarter on Tuesday, after the firm announced positive regulatory news related to its type 1 diabetes (T1D) candidate teplizumab. 26 May 2021
The US Food and Drug Administration has accepted the Biologics License Application (BLA) for ublituximab, an investigational glycoengineered anti-CD20 monoclonal antibody, in combination with Ukoniq (umbralisib), TG Therapeutics’ once-daily, oral, inhibitor of PI3K-delta and CK1-epsilon, as a treatment for patients with chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL). 26 May 2021
The US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for Verastem’s investigational RAF/MEK inhibitor VS-6766, combined with defactinib, its FAK inhibitor, in recurrent low-grade serous ovarian cancer (LGSOC) regardless of KRAS status after one or more prior lines of therapy, including platinum-based chemotherapy. 25 May 2021
The US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation (BTD) for Boehringer Ingelheim’s BI 425809 for the treatment of cognitive impairment associated with schizophrenia (CIAS). 24 May 2021
The US Food and Drug Administration strictly prohibits the distribution of counterfeit, diverted, subpotent, substandard, adulterated, misbranded, and expired drugs that pose a serious threat to life. Nevertheless, such drugs continue to end up in circulation, compromising patient safety. 24 May 2021
On Friday, the US Food and Drug Administration approved Rybrevant (amivantamab-vmjw) as the first treatment for adult patients with non-small cell lung cancer (NSCLC) whose tumors have specific types of genetic mutations: epidermal growth factor receptor (EGFR) exon 20 insertion mutations. 22 May 2021
The US Food and Drug Administration has approved another important indication for Opdivo (nivolumab, injection for intravenous use), this one for the adjuvant treatment of completely resected esophageal or gastroesophageal junction (GEJ) cancer with residual pathologic disease in patients who have received neoadjuvant chemoradiotherapy (CRT). 21 May 2021
The US Food and Drug Administration FDA has approved Empaveli (pegcetacoplan) injection to treat adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare, life-threatening blood disease. 18 May 2021
The US Food and Drug Administration has published the guidance for industry entitled “M9 Biopharmaceutics Classification System-Based Biowaivers,” a draft of which was issued in October 2018. The guidance provides recommendations to support the biopharmaceutics classification of drug substances and the Biopharmaceutics Classification System (BCS)-based waiver of the in vivo bioequivalence study requirement for drug products. 14 May 2021
US-headquartered Strongbridge Biopharma saw its shares jump as much as 6% this morning, after the rare diseases specialist revealed that the US Food and Drug Administration has accepted for review the company’s New Drug Application (NDA) for Recorlev (levoketoconazole) for the treatment of endogenous Cushing’s syndrome. 13 May 2021