The US Food and Drug Administration (FDA) has approved Wayrilz (rilzabrutinib) for adults with persistent or chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment. The approval was based on the pivotal LUNA 3 Phase III study, in which Wayrilz met the… Read More
Kaerus Bioscience’s lead candidate KER-0193 has been granted both Orphan Drug Designation and Rare Pediatric Drug Designations for the treatment of Fragile X syndrome (FXS) by the US Food and Drugs Administration (FDA). This follows the recent successful completion of a Phase I trial of KER-0193 in healthy volunteers, which… Read More
The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended granting marketing authorisation for Novartis’ Kisqali (ribociclib). The positive opinion relates to the adjuvant treatment of adults with hormone receptor-positive/human epidermal growth factor receptor 2-negative (HR+/HER2-) early breast cancer (EBC),… Read More
Biotech company 4SC has announced positive new data for its treatment for patients with advanced-stage cutaneous T-cell lymphoma (CTCL). Findings from the RESMAIN study of 4SC’s resminostat (Kinselby) treatment show that the therapy can postpone disease progression in advanced CTCL. The RESMAIN study is one of… Read More
The US Food and Drug Administration (FDA) has granted marketing authorisations to several new therapeutics over the last month that will have a significant impact on areas of unmet need and the evolution of future treatments. Elevidys, Sarepta Therapeutics Elevidys became the first gene therapy for… Read More
Verona Pharma has submitted a New Drug Application (NDA) to the US Food and Drug Administration (FDA) for approval of ensifentrine for the maintenance treatment of patients with chronic obstructive pulmonary disease (COPD). Ensifentrine is a selective dual inhibitor of the enzymes phosphodiesterase 3 and 4 combining… Read More
The Food and Drug Administration (FDA) in the US has made several key drug decisions over the last few weeks. Here’s a summary. Orserdu (elacestrant), Stemline Therapeutics The first and only treatment specifically indicated for patients with ESR1 mutations in ER+, HER2- advanced or metastatic breast… Read More
News round-up for 19-23 September by DDW Editor Reece Armstrong An interesting week for clinical research and drug approvals, with rare diseases being a key focus for both the FDA and European Commission. The last week has seen data emerge on how Omicron infection could protect against influenza, how… Read More
The European Medicines Agency (EMA) has given a positive opinion to a combination therapy for the treatment of adults with lupus nephritis (LN). Otsuka Pharmaceutical Europe received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) for the use of its oral calcineurin inhibitor… Read More
The Medicines and Healthcare products Regulatory Agency (MHRA) has granted Marketing Authorisation to Novartis UK’s treatment for a specific type of cancer. The authorisation was granted for Scemblix (asciminib), Novartis UK’s therapy for adults with Philadelphia chromosome-positive chronic myeloid leukaemia (Ph+ CML) in chronic phase (CP) who have… Read More
Pharmaceutical company ViiV Healthcare has been granted Japanese approval for its treatment of HIV-1 as a combination therapy. The approval comes from the Ministry of Health, Labour and Welfare (MHLW) in Japan and enables the company’s Vocabria (cabotegravir injection and tablets) to be used in combination… Read More
The Medicines and Healthcare products Regulatory Agency (MHRA) has granted a conditional marketing authorisation to Takeda’s EXKIVITY (mobocertinib) for patients in Great Britain with a rare and aggressive form of non-small cell lung cancer (NSCLC). The authorisation means that EXKIVITY (mobocertinib) will be made available as a… Read More
Drug approvals are critical to the drug discovery industry, ensuring innovation thrives while the public remain safe from the risks and benefits associated with new drugs. Organisations responsible for these approvals include the US Food and Drug Administration (FDA), the European Medical Agency (EMA), the Scottish Medicines Consortium (SMC),… Read More
The US Food and Drug Administration (FDA) has cleared investigational new drug (IND) applications for MRx0005 and MRx0029, two 4D Pharma Live Biotherapeutics (LBPs). The company expects to initiate a first-in-human Phase I clinical trial in people with Parkinson’s disease in mid-2022. Context Parkinson’s disease… Read More
Angelini Pharma has announced that cenobamate, an oral anti-seizure medicine (ASM), is now available for use within NHS Scotland as a treatment for eligible adults with uncontrolled focal epilepsy. The Scottish Medicines Consortium (SMC) has accepted cenobamate for restricted use in adults with drug-resistant epilepsy as a second-line adjunctive… Read More
The National Institute for Health and Care Excellence (NICE) in the UK has issued a Final Appraisal Document (FAD) recommending AstraZeneca’s Forxiga (dapagliflozin) within its marketing authorisation for the treatment of adults with chronic kidney disease (CKD).1 Conditions Dapagliflozin is recommended as an option… Read More
