United States

A diversity study has shown that clinical trials should use real world diseases epidemiology data, rather than US Census Bureau race and ethnicity data, to ensure enrolment reflects the populations affected by different diseases.  GSK’s retrospective study looked at clinical trial demographic data from 495 GSK and ViiV clinical trials involving US-based participants from the […]

Intercept Pharmaceuticals’ obeticholic acid (OCA) could be first drug to be approved in the US for non-alcoholic steatohepatitis (NASH), according to analysts GlobalData.  The Food and Drug Administration (FDA) accepted the company’s resubmission of its new drug application (NDA) for OCA in patients with pre-cirrhotic liver fibrosis due to NASH in December 2022.  According to […]

Leqembi’s prospects in the Alzheimer’s disease market remain uncertain despite its advantages over Aduhelm, says analytics company GlobalData.  Eisai and Biogen’s anti-amyloid beta (Aβ) monoclonal antibody (mAb), Leqembi (lecanemab), recently gained approval from the Food and Drug Administration (FDA) under the accelerated approval pathway for the treatment of Alzheimer’s disease (AD) in patients with early […]

Airsupra (albuterol/budesonide) has been approved in the US for the as-needed treatment or prevention of bronchoconstriction and to reduce the risk of exacerbations in people with asthma aged 18 years and older.  Airsupra is a pressurised metered-dose inhaler (pMDI), fixed-dose combination of albuterol, a short-acting beta2-agonist (SABA), and budesonide, an anti-inflammatory inhaled corticosteroid (ICS). It […]

The US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for VAX-24, Vaxcyte’s 24-valent pneumococcal conjugate vaccine for the prevention of invasive pneumococcal disease.  The decision was based on positive topline results from a Phase I/II proof-of-concept study, which evaluated the safety, tolerability and immunogenicity of VAX-24 in adults 18-64 years of age.  […]

The US Food and Drug Administration (FDA) has granted Orphan Drug Designation for iOnctura’s IOA-244, which is in development for solid and hematologic malignancies including uveal melanoma.  Uveal melanoma is a disease in which cancer originates in the tissues of the eye, causing symptoms such as blurred vision or a dark spot on the iris.   […]

The United States Food and Drug Administration (FDA) has granted Fast Track Designation (FTD) to Bicycle Therapeutics’ BT8009 monotherapy for the treatment of adult patients with previously treated locally advanced or metastatic urothelial cancer.   BT8009 is a potential first in class Bicycle Toxin Conjugate (BTC) targeting Nectin-4, a protein that is highly expressed in urothelial […]

Celltrion USA has submitted a Biologics License Application (BLA) under the 351 (a) pathway of the Public Health Service Act for CT-P13 SC for the treatment of inflammatory bowel disease (IBD).  The submission is based on results from the Phase III pivotal data that evaluated the efficacy and safety of CT-P13 SC as maintenance therapy […]

Orgenesis has agreed up to $50 million investment from Metalmark Capital in its US-based point of care services subsidiary, Morgenesis.  The investment will consist of an upfront investment of $30 million and two future investments of $10 million each, subject to the achievement of certain milestones.  Morgenesis will use the investment to develop and expand […]