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
The European Medicines Agency’s (EMA) Committee for Human Medicinal Products (CHMP) recommended approval of five novel medicines at its September meeting 20 September 2024
The Israeli Ministry of Health (IL MOH) has approved Tevimbra (tislelizumab) as monotherapy for the treatment of adult patients with unresectable or metastatic oesophageal squamous cell carcinoma (OESCC). 20 September 2024
The European Medicines Agency’s (EMA) advisory committee the CHMP has adopted a positive opinion for a label update of Danish drugmaker Novo Nordisk’s Wegovy (semaglutide 2.4mg). 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Yesterday, the US Food and Drug Administration (FDA) issued a draft guidance, “Integrating Randomized Controlled Trials for Drug and Biological Products into Routine Clinical Practice” 18 September 2024
Bayer has submitted a Marketing Authorization Application (MAA) to the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) for the investigational compound elinzanetant. 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
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 Russian Ministry of Health wants to speed registration of drugs that are not localized in the Russian Federation at the time of the application submitted by domestic pharmaceutical companies, explaining such changes by "unpredictable geopolitical situation," reports The Pharma Letter’s local correspondent. 12 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
Carima Andrady, senior regulatory writer at PharmaLex, provides an Expert View on a key consideration when seeking to secure an Orphan Drug designation (ODD) in Europe. 8 October 2021
The US Food and Drug Administration’s Antimicrobial Drugs Advisory Committee (AMDAC) voted unanimously to recommend use of maribavir (TAK-620) for the treatment of refractory cytomegalovirus (CMV) infection and disease with genotypic resistance to ganciclovir, valganciclovir, foscarnet or cidofovir in transplant recipients. 8 October 2021
Shares of US clinical-stage biotech Allogene Therapeutics plunged more than 40% in after-hours trading yesterday, following its revelation of a Food and Drug Administration hold on the company’s AlloCAR T clinical trials. 8 October 2021
The Japanese Ministry of Health, Labor and Welfare (MHLW) has approved Cibinqo (abrocitinib), an oral, once-daily, Janus kinase 1 (JAK1) inhibitor, for the treatment of moderate to severe atopic dermatitis (AD) in adults and adolescents aged 12 years and older with inadequate response to existing therapies. 7 October 2021
UK pharma major GlaxoSmithKline (LSE: GSK), having largely missed out in the race for COVID-19 vaccines, has scored a goal in the fight against a malaria. 7 October 2021
Healthcare giant Johnson & Johnson has submitted data to the US Food and Drug Administration (FDA) to support use of a booster shot of its COVID-19 vaccine in adults. 6 October 2021
Australia’s Therapeutic Goods Administration (TGA) has granted provisional determination to the local subsidiary of US pharma giant Pfizer in relation to a new combination therapy containing PF-07321332 and ritonavir, for the treatment of adult patients with symptomatic, confirmed coronavirus infection. 6 October 2021
UK pharma major AstraZeneca has submitted a request to the US Food and Drug Administration for an Emergency Use Authorization (EUA) for AZD7442, its long-acting antibody (LAAB) combination, for prophylaxis of symptomatic COVID-19. 5 October 2021
The European Medicines Agency’s human medicines committee (CHMP) has concluded that an extra dose of the COVID-19 vaccines Comirnaty, from Pfizer and BioNTech, as well as Spikevax, marketed by Moderna, may be given to people with severely weakened immune systems, at least 28 days after their second dose. 5 October 2021
The US Food and Drug Administration (FDA) has received a regulatory submission for a new dispersible tablet formulation of the fixed-dose combination of Triumeq (abacavir/ dolutegravir/ lamivudine), and to extend a current approval to lower the minimum weight at which a child can be prescribed this medicine, from 40kg and above to 14kg and above. 4 October 2021
The European Medicines Agency today said it has started evaluating an application for marketing authorization for the monoclonal antibody Regkirona (regdanvimab, also known as CT-P59) to treat adults with COVID-19 who do not require supplemental oxygen therapy and who are at increased risk of progressing to severe COVID 19. 4 October 2021
The US Food and Drug Administration (FDA) has granted Enhertu (trastuzumab deruxtecan), Breakthrough Therapy designation (BTD) for unresectable or metastatic HER2-positive breast cancer patients who have received one or more prior anti-HER2-based regimens. 4 October 2021
Seattle, USA-based biotech Omeros Corporation saw its shares plummet 38% to $8.54 on Friday, as it revealed that the US Food and Drug Administration notified the company that its marketing application for narsoplimab could not be approved. 4 October 2021
The US Food and Drug Administration has granted approval for Tecartus (brexucabtagene autoleucel) for the treatment of adult patients (18 years and older) with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL), scoring a first for Kite. 2 October 2021
At the latest pharmacovigilance risk assessment meeting of the European Medicines Agency, the organization initiated a review of medicines containing nomegestrol and chlormadinone. 1 October 2021
Indian drugmaker Glenmark Pharmaceuticals today announced it has received marketing approval for its fixed-dose combination nasal spray Ryaltris (olopatadine Hcl and mometasone furoate), in 13 countries across the European Union and the UK. 30 September 2021
Mirum Pharmaceuticals, a leader in rare liver disease, says that the US Food and Drug Administration has approved Livmarli (maralixibat) oral solution for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) one year of age and older. 30 September 2021
On Wednesday, the US Food and Drug Administration issued the draft guidance for industry, “Benefit-Risk Assessment for New Drug and Biological Products.” 30 September 2021
Chicago-based drugmaker AbbVie has had another calcitonin gene-related peptide (CGRP) receptor antagonist approved in the migraine space by the US Food and Drug Administration (FDA). 29 September 2021
A new analysis of five-year data for the gene therapy PTC-AADC shows “profound improvements” in children with aromatic L-Amino acid decarboxylase (AADC) deficiency. 29 September 2021