The US Food and Drug Administration’s Cellular, Tissue and Gene Therapies Advisory Committee has voted that available evidence does not support the effectiveness of Capricor Therapeutics’ Deramiocel for the treatment of cardiomyopathy in patients with Duchenne muscular dystrophy (DMD). DMD is a rare and life-threatening disease for… Read More
The Medicines and Healthcare products Regulatory Agency (MHRA) has granted Myqorzo (aficamten) a marketing authorisation across the UK for the treatment of symptomatic (New York Heart Association, NYHA class II-III) obstructive hypertrophic cardiomyopathy (oHCM). At the same time, the National Institute for Health and Care Excellence (NICE)… Read More
Radiopharmaceuticals company Perspective Therapeutics has received orphan medicinal product designation from the European Commission (EC) for [²¹²Pb]VMT-α-NET for the treatment of gastroenteropancreatic neuroendocrine tumours (GEP-NETs). The designation follows a positive opinion from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA). … Read More
AstraZeneca’s fixed-dose triple-combination Trixeo Aerosphere (budesonide/glycopyrronium/formoterol fumarate or BGF) has been recommended for approval in the EU for the maintenance treatment of asthma in patients 12 years of age and older. It is recommended in patients who are not adequately controlled by a combination of a medium… Read More
The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended granting marketing authorisation approval under exceptional circumstances for Nezglyal (leriglitazone) as a treatment for male cALD patients, aged two to 12 years with gadolinium (Gd) negative brain lesions. European Commission approval… Read More
The Medicines and Healthcare products Regulatory Agency (MHRA) has granted marketing authorisation to a treatment for metastatic hormone-sensitive prostate cancer (mHSPC). Akeega (niraparib and abiraterone acetate), developed by Johnson & Johnson, has been granted the authorisation as part of a combination regimen with prednisone or prednisolone and andogren deprivation therapy (ADT). … Read More
The European Commission (EC) has granted marketing authorisation for Tepkinly (epcoritamab) in combination with lenalidomide and rituximab (R2) for the treatment of relapsed or refractory follicular lymphoma. The combination regimen, developed by AbbVie and Genmab for the treatment of adult patients, is the first and only bispecific-based therapy approved in… Read More
The US Food and Drug Administration (FDA) has expanded the use of Casgevy (exagamglogene autotemcel) for the treatment of people ages two years and older with either sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) or transfusion-dependent beta thalassemia (TDT). The approval makes Casgevy the first… Read More
The European Commission (EC) has approved Itvisma (onasemnogene abeparvovec) for the treatment of spinal muscular atrophy (SMA). Itvisma can now be used in the European Union by children two years and older, teens and adults living with 5q SMA with a bi-allelic mutation in the survival motor neuron 1… Read More
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion recommending a cream to treat atopic dermatitis (AD). Opzelura (ruxolitinib) cream, developed by Incyte International for the treatment of moderate atopic dermatitis (AD), has been recommended for adult patients for whom topical corticosteroids… Read More
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending a treatment for severe alopecia. The recommended approval of upadacitinib is for the treatment of adult and adolescent patients with severe alopecia areata (AA), an unpredictable autoimmune disease causing a… Read More
The US Food and Drug Administration (FDA) has approved Lumvoa (veligrotug-vvze) as a treatment for thyroid eye disease (TED). TED is a rare, debilitating autoimmune disease characterised by inflammation and tissue remodelling around and behind the eyes, which can lead to proptosis, diplopia, pain and vision impairment. Read More
The US Food and Drug Administration (FDA) has granted Dicot Pharma approval to roll out the Phase IIb study of a treatment for erectile dysfunction. The approval follows an Investigational New Drug (IND) Application submitted to the FDA, with the trial evaluating LIB-01 now set to commence in the US. … Read More
The US Food and Drug Administration (FDA) has cleared the investigational new drug (IND) application for Beam Therapeutics’ BEAM-304 for the treatment of phenylketonuria (PKU). PKU is a rare, inherited metabolic disorder that results in toxic accumulation of phenylalanine (Phe), leading to serious neurologic and neurocognitive impairments… Read More
The National Institute for Health and Care Excellence (NICE) has recommended cemiplimab for the treatment of advanced cervical cancer. The immunotherapy, developed by biotechnology company Regeneron, has been recommended in England and Wales to treat recurrent or metastatic cervical cancer that has progressed on or after platinum-based chemotherapy… Read More
The European Commission (EC) has approved a combination therapy for the treatment of metastatic colorectal cancer. Braftovi (encorafenib) has been approved in combination with cetuximab and FOLOX for the first-time treatment of adult patients with BRAFV600E-mutant metastatic colorectal cancer (mCRC). The approval is based on the results from the… Read More
