Drug approvals

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 Duchenne muscular dystrophy (DMD) to gain marketing authorisation in […]

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 bronchodilator and non-steroidal anti-inflammatory activities in one molecule.   […]

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 cancer was approved in the US in February. The […]

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 breast cancer cells evade therapies, and […]

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 (CNI) Lupkynis (voclosporin) in combination with mycophenolate mofetil […]

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 previously been treated with two or more tyrosine […]

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 with Janssen Pharmaceutical Companies of Johnson & Johnson’s Rekambys and Edurant .  […]

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 treatment for adult patients with epidermal growth factor […]

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), the Medicines and Healthcare products Regulatory […]

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 is condition impacting more than 10 million people globally, which […]

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 ASM, after failure of the first […]

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 for treating CKD in adults. It is recommended only if:  it […]

AstraZeneca’s Saphnelo (anifrolumab) has been approved in the European Union (EU) as an add-on therapy for the treatment of adult patients with moderate to severe, active autoantibody-positive systemic lupus erythematosus (SLE), despite receiving standard therapy.   Context   Saphnelo is the first biologic for SLE approved in Europe with an indication that is not restricted to patients […]