Morning Briefing
Summaries of health policy coverage from major news organizations
FDA Approves Pill That Offers New Way To Treat ADHD In Those As Young As 6
MedPage Today: First-In-Class Drug For ADHD Wins FDA Approval
The FDA approved oral centanafadine (Simtriyo), a first-in-class triple reuptake inhibitor for attention deficit-hyperactivity disorder (ADHD), Otsuka announced late last week. The once-daily extended-release capsule -- a norepinephrine, dopamine, and serotonin reuptake inhibitor that functions as a central nervous system stimulant -- is indicated for patients ages 6 years and older who weigh at least 20 kg (44 lb). "By inhibiting the reuptake of norepinephrine, dopamine, and serotonin, Simtriyo increases the availability of these neurotransmitters in pathways involved in attention and behavioral regulation," the drugmaker explained. (Monaco, 7/27)
More pharma and tech developments —
Stat: FDA Questions The Efficacy Of Capricor's Duchenne Drug
The Food and Drug Administration said Monday that Capricor Therapeutics’ stem cell treatment for Duchenne muscular dystrophy did not meet the objectives of a Phase 3 trial — contrary to the company’s claims last year. (Mast, 7/27)
CIDRAP: Trial Supports Switch From IV To Oral Antibiotics For Kids With Severe Pneumonia
A trial of young children in sub-Saharan Africa with severe pneumonia found that switching from intravenous (IV) to oral antibiotics after clinical improvement is safe and effective, a finding that could allow for earlier release from the hospital, researchers reported last week in The Lancet. Although severe pneumonia is not as deadly as it once was in children, it’s still a leading cause of childhood mortality and hospitalization worldwide, particularly in resource-limited regions. (Dall, 7/27)
Stat: How A Single Rare Disease, AATD, Sparked A Biotech Gold Rush
Biotechs are spending billions to cure a rare liver disorder most Americans have never heard of. The contentious race features dueling technologies, patent wars, a broken alliance, and a boiling competition between the U.S. and Chinese drug industries. (Mast, 7/28)
Medical Xpress: Scientists Decipher How T Cells Sense Enemies—Such As Cancer—At Point Of Contact
Every encounter between a T cell and a potential target—especially when that target is a developing tumor—begins with a rapid series of molecular decisions. Within seconds, the immune cell must determine whether to launch an attack or stand down. T cells are so potent, so potentially devastating, that misreading the situation can cause serious tissue injury. But cancer cells come equipped with a bag of tricks that allows them to disarm these powerful warriors of the immune system. All of these activities, whether mediated by T cells or their targets, occur at split-second speed and unfold at the point of cell-to-cell contact. (Ricks, 7/27)
Phys.org: AI Finds Tiny Gene Editor Changes That Reduce Unintended DNA Edits
Gene editing is a highly precise and powerful technology that allows scientists to insert, delete, modify or replace DNA bases in living organisms. It has a variety of uses, including correcting disease-causing mutations and improving crops. Tools like CRISPR act as molecular scissors that target specific places in a genome to make these changes. But the technology is not perfect and can accidentally edit the wrong pieces of DNA or RNA. (Arnold, 7/27)
