Between common chronic conditions like COPD and asthma, and acute harm inflicted by viruses, lung damage is a big problem. Many treatments focus on the upper airways. But to go deeper, researchers are studying the pair found in some Covid vaccines: mRNA + a lipid nanoparticle. 

New research in mice, published Friday in Nature Communications, posits that a similar technology could be used to stimulate healing in parts of the lung not easily reached by conventional therapies. The team reports a nanoparticle, called ionizable amphiphilic Janus dendrimer, targets mRNA therapy directly to the lung and promotes tissue repair.

“If introduced early in the inflammatory process,” this mechanism could speed up healing, “ultimately reducing the incidence of complex lung injury and disease,” write the authors (several of whom are named on patents and patent applications related to the research).  — Isabella Cueto

FDA approves first drug for rare lipid storage disease

The FDA has approved Ctexli, or chenodiol, as the first treatment for a rare genetic disorder called cerebrotendinous xanthomatosis, which impairs fat metabolism and can lead to abnormal cholesterol deposits in multiple organs. Two years ago, Mirum Pharmaceuticals paid $210 million to Travere Therapeutics to acquire its bile acid portfolio — which included this drug, chenodiol.

Chenodiol has been approved already for decades to treat gallstones. This means that patients with this disease have already been able to access chenodiol off-label for years, FiercePharma points out.

from AXIOS:

Shortage over for Novo’s Ozempic, Wegovy

By Maya Goldman

Illustration: Shoshana Gordon/Axios

Makers of copycat weight-loss drugs and digital health companies that sell them are bracing for upheaval now that the FDA has declared Novo Nordisk’s blockbusters Ozempic and Wegovy are no longer in shortage.Why it matters: The announcement on Friday effectively ends an FDA policy that allowed drug compounders to make less expensive off-brand versions of the semaglutide injections when the brand-name products are in short supply.And it may force patients to pay more to stay on their regimens.State of play: To provide a wind-down period, compounders have until at least April 22 to stop selling copycat versions before the FDA starts enforcing the cutoff.Where it stands: Compounders and pharmacies knew the semaglutide shortage would end at some point but are questioning whether now is the appropriate time to declare it over.Noom, a digital health company that prescribes compound semaglutide, said it believes patients are still having trouble accessing the branded Ozempic and Wegovy.Digital health company Hims & Hers said it will continue selling compounded semaglutide as it seeks to “personalize the delivery of existing, clinically studied medications,” CEO Andrew Dudum wrote in a post on X Friday.