Alexion Pharmaceuticals said that it has submitted a biologics license application (BLA) to the US Food and Drug Administration (FDA), seeking approval for its C5 complement inhibitor ALXN1210 for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH).
AbbVie has submitted a biologics license application (BLA) to the US Food and Drug Administration seeking approval of risankizumab for the treatment of moderate to severe plaque psoriasis.
Sarepta Therapeutics has is planning to submit a new drug application (NDA) for accelerated approval of its duchenne muscular dystrophy (DMD) drug Golodirsen (SRP-4053).
Alnylam Pharmaceuticals and Sanofi Genzyme have submitted a marketing authorisation application (MAA) to the European Medicines Agency (EMA) for patisiran, an investigational RNAi therapeutic targeting transthyretin (TTR) for the treatment of adults with hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis).
Pharming Group has submitted a supplemental biologics license application (BLA) to the US Food and Drug Administration (FDA) for Ruconest [Recombinant Human C1 Esterase Inhibitor/ conestat alfa] for routine prophylaxis to prevent attacks in adult and adolescent patients with hereditary angioedema (HAE).
Janssen Biotech has submitted a new drug application (NDA) to the US Food and Drug Administration (FDA) for apalutamide, an investigational, next generation oral androgen receptor (AR) inhibitor for men with non-metastatic castration-resistant prostate cancer (CRPC).
Sunovion Pharmaceuticals announced that it has submitted a new drug application (NDA) to the US Food and Drug Administration (FDA) for dasotraline, a novel investigational, dual-acting dopamine and norepinephrine reuptake inhibitor (DNRI), for the treatment of children, adolescents and adults with attention deficit hyperactivity disorder (ADHD).
Kite Pharma has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for axicabtagene ciloleucel as a treatment for patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL), transformed follicular lymphoma (TFL), and primary mediastinal B-cell lymphoma (PMBCL) who are not eligible for autologous stem cell transplant.
Amicus Therapeutics has revealed its plans to file a new drug application (NDA) with the US FDA for the oral precision medicine migalastat for Fabry disease in the fourth quarter of the year.
BioMarin Pharmaceutical has submitted a Biologics License Application (BLA) to the US Food and Drug Administration (FDA) for pegvaliase, a PEGylated recombinant phenylalanine ammonia lyase enzyme product, to reduce blood phenylalanine (Phe) levels in adult patients with PKU who have uncontrolled blood Phe levels on existing management.
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