The marketing application filed by Ultragenyx for its candidate UX007, or triheptanoin, as a treatment for long-chain fatty acid oxidation disorders was accepted by the FDA. The action date set by the agency is July 31, 2020.
Data from a late-stage trial conducted by Roche to assess its drug MabThera, or rituximab, showed the drug outperformed mycophenolate mofetil as a treatment for pemphigus vulgaris, with 40.3% of patients achieving sustained complete remission versus 9.5% in the study's control arm. Results showed the drug achieved its primary and secondary endpoints.
A Series A funding round raised $16 million for UK-based biotech Mogrify, which is developing cell therapies for auto-immune and musculoskeletal disorders, ocular and respiratory diseases and cancer immunotherapy.
The Paper Bottle Company, based in the Netherlands, announced that Avantium is now a part of its Paper Bottle Project, which is focused on creating a paper bottle that is completely plant-based and recyclable. Avantium's polyethylene furanoate technology helps the bottles withstand gas permeability for beverages.
Despite growing interest in patient-centered drug development, developing an engagement framework is complicated, and few clinical trial sponsors have a consistent, company-wide process for obtaining feedback from study participants, writes Mary Elmer, Merck's executive director of patient, caregiver and consumer experience. Sponsors should integrate with patients and patient advocates, study regulations and the current landscape, and build long-term trust with patients, Elmer writes.
A gene therapy designed specifically for a young girl with Batten disease, which causes progressive brain damage and death, masks a mutation in the CLN7 gene and reduced seizure frequency, and the girl's neurologic test scores have also improved, the research team reported in The New England Journal of Medicine. The case could serve as a model for so-called n-of-1 studies for life-threatening neurological or brain conditions with no available treatment, neurologist Timothy Yu says.
A biocompatible lab-on-a-chip described in Analytical Chemistry could accelerate the development of new antiplatelet therapies, researchers report. The proof-of-concept study found the microlab device accurately controlled blood flow, quickly delivered and combined drugs with blood, and efficiently delivered the drug-blood combination to a thrombus assay system.
About 15% of observational, randomized, controlled clinical trials could be replicated by analyses of real-world evidence obtained from insurance claims and electronic health records, according to a study in JAMA Network Open. The authors concluded that real-world evidence is not yet a viable replacement for randomized controlled trials, but it can provide critical post-approval insights into product safety and efficacy and enable quicker identification of safety problems.
The NIH renewed a 5-year, $7.57 million grant to Cincinnati Children's Hospital Medical Center to continue leading the Consortium of Eosinophilic Gastrointestinal Disease Researchers and expand research, develop clinical expertise, train clinical investigators and provide access to information for researchers, patients, physicians and the public.
Capturing and analyzing real-world data could make clinical trials more effective and efficient, and the FDA has already used RWD in some regulatory decisions; however, a lack of standards for recording data, incorrectly entered data and difficulties screening out confounding data are barriers, FDA Center for Drug Evaluation and Research Director Janet Woodcock says. The FDA is "collaborating with a lot of groups" to determine whether clinical practices can randomize patients and collect real-world outcomes data without an elaborate clinical trial framework.