Days after the FDA granted approval for Genentech's hemophilia A candidate emicizumab, or Hemlibra, the company announced results from the HAVEN 3 study that showed the drug was linked with a significant decline in the rate of treated bleeds for patients treated weekly.
Although serum endomysial antibodies and antibodies to tissue transglutaminase tests are useful in screening for celiac disease in patients still eating gluten, the tests have low sensitivity for those with biopsy-confirmed celiac disease who are on gluten-free diets, a meta-analysis shows. "In the absence of a non-invasive biomarker, follow-up duodenal biopsy remains the only appropriate test to assess mucosal recovery in children and adults with celiac disease," researchers said.
Australian researchers have identified exosomal microRNA molecules that appear to be biomarkers for multiple sclerosis and could help in developing new diagnostic assays. The findings were reported in the journal Scientific Reports.
The FDA granted Grifols approval for its blood-based alpha-1 antitrypsin deficiency test for use with DNA extracted from a dry blood spot or a blood sample. The test is intended for the detection of 14 SERPINA1 mutations, which cause AAT deficiency, and enables simultaneous analysis of 192 samples per kit.
A study in the journal Supportive Care in Cancer found that supportive therapies for anemia resulted in better patient-reported outcomes for patients with lymphoma than those with multiple myeloma. The 250-person study also found that increases in hemoglobin levels were tied to improvements in fatigue and health-related quality of life. Supportive care consisted primarily of erythropoiesis-stimulating agents.
A new report in the journal The Lancet Infectious Diseases offers guidance on post-exposure prophylaxis of Ebola virus infection. The review describes six Ebola countermeasures as well as scenarios that warrant prophylaxis.
The European Centre for Disease Prevention and Control reports that antimicrobial resistance is increasing in 30 countries in the European Union and European Economic Area, particularly for Escherichia coli and the Acinetobacter species. A significant decrease, however, was seen in rates of methicillin resistance among Staphylococcus aureus from 2013 to 2016.
Roche Holding was granted approval by the FDA for its Hemlibra, or emicizumab-kxwh, as a treatment for adult and pediatric patients with hemophilia A who have factor VIII inhibitors. The once-weekly administered drug, given via subcutaneous injection, is a preventive treatment for bleeding episodes in patients.
The FDA has released what it calls a regulatory framework to promote and accelerate development of effective regenerative medicine therapies while cracking down on businesses that market inadequately tested procedures that the government says can put patients at risk. The new rules specify the processing protocols that will trigger FDA oversight and outline a framework for advancing development of promising cell and genetic therapies. "With this balanced approach, we're well positioned to support and help advance breakthrough science, like regenerative medicine, and promote responsible and flexible regulation that leverages science to advance public health," FDA Commissioner Scott Gottlieb said in a statement.
Two studies presented at the annual meeting of the American Academy of Ophthalmology detailed how retinal pigment epithelial cells derived from embryonic stem cells were transplanted into patients' eyes to treat retinal degeneration. Although the work was not designed to assess efficacy, researchers said they detected early indications of improved visual acuity, and there were no safety problems associated with the transplanted cells themselves.