PREP

THURSDAY, Sept. 12, 2024 -- Individuals with sickle cell trait (SCT) have an increased risk for venous thromboembolism (VTE), according to a study published online Sept. 12 in Blood Advances . Keng-Han Lin, Ph.D., from 23andMe in Sunnyvale, California, and colleagues leveraged data from the 23andMe Research cohort (4,184,082 participants) to calculate the ancestry-independent risk for VTE associated with SCT. A meta-analysis of three genetic ancestry groups (European [3,183,142 participants], Latine [597,539 participants], and South Asian [41,257 participants]) was used to calculate odds ratios. The researchers found that 2.25 percent of participants from the full cohort reported a history of VTE. In a meta-analysis, the risk for VTE was increased 1.45-fold among individuals with SCT versus non-SCT carriers, similar to the full cohort estimate. In SCT, the risk for pulmonary embolism (PE) was higher than that of isolated deep vein thrombosis (DVT; odds ratios, 1.95 versus 1.04). Carriers of factor V Leiden had a 3.30-fold increased risk for VTE versus noncarriers, with a higher risk for isolated DVT versus PE (odds ratios, 3.59 and 2.72, respectively). "Our study also provides high-powered data to confirm the PE-predominant pattern of VTE in SCT carriers. These data may inform clinical practice guidelines, future research, and public health initiatives in SCT," the authors write. Several authors are employed by and hold stock or stock options in 23andMe.

The TARPEYO delayed-release capsules are indicated for reducing proteinuria to treat primary immunoglobulin A nephropathy in adult patients. Credit: Alexander Grey on Unsplash. Calliditas Therapeutics has submitted a supplemental new drug application (sNDA) to the US Food and Drug Administration (FDA) for complete approval of TARPEYO (budesonide). The TARPEYO delayed-release capsule is a corticosteroid indicated for reducing proteinuria to treat primary immunoglobulin A nephropathy (IgAN) in adult patients at risk of quick disease progression, generally having a urine protein-to-creatinine ratio greater than or equal to 1.5g/g. Recommended Reports Reports LOA and PTSR Model - (Aspirin + Dimethyl Fumarate) in Relapsing Remitting Multiple Sclerosis (RRMS) GlobalData Reports Prolyl Endopeptidase (Post Proline Cleaving Enzyme or PREP or EC 3.4.21.26) Drugs in Development ... GlobalData View all Companies Intelligence Calliditas Therapeutics AB SNDA Nefecon AB View all The therapy received accelerated approval based on showing a reduction in proteinuria. The submission of sNDA to the regulator was based on the findings obtained from the Phase III NefIgArd clinical trial. The multicentre, randomised, double-blind trial was designed to evaluate the safety and efficacy of TARPEYO 16 mg compared with placebo against a background of optimised RASi (renin–angiotensin system inhibitor) therapy in primary IgA adult patients. TARPEYO showed a highly statistically significant benefit in estimated glomerular filtration rate (eGFR) over a two-year period of treatment. The treatment period comprises nine months on TARPEYO or placebo, then 15 months of follow-up. Calliditas Therapeutics CEO Renée Aguiar-Lucander stated: “The eGFR treatment benefit observed across the entire study population, irrespective of UPCR levels, provides further evidence that targeting IgAN at its source can offer patients a treatment that holds the promise of being disease-modifying. “We are pleased to be able to provide the FDA with the full results of our Phase III study, and we look forward to interactions with the FDA regarding full approval of TARPEYO.” The company is also collaborating with its European commercial partner STADA Arzneimettel to seek complete approval for Nefecon, branded as Kinpeygo, from the European Commission.