Following positive data from competitors earlier this year, United Therapeutics Corp. rolled out impressive and unexpected phase III results of nebulized Tyvaso (treprostinil) for idiopathic pulmonary fibrosis (IPF), setting the stage for an sNDA filing with the U.S. FDA next year.  If approved for the indication, United, of Silver Spring, Md., and Research Triangle Park, N.C., could tap into what analysts say is a $4 billion-plus market.

Two days after the ouster of Susan Monarez as CDC director on Aug. 27, HHS confirmed to BioWorld that James O’Neill is serving as acting director. O’Neill was previously Deputy Secretary of Health and Human Services. He has also served as the CEO of the SENS Research Foundation, which merged with Lifespan.io in 2024. The resulting Lifespan Research Institute describes itself as “focused on the defeat of age-related disease and the extension of healthy human lifespan” on its website.

Three milestones expected to bring the reality of U.S. prescription drug price negotiations into focus are hovering on the horizon. First, the CMS is scheduled to publish its maximum fair prices (MFPs) for the round 2 selected drugs by Nov. 30. Then, on Jan. 1, the MFPs for the first round kick in, affecting not only the 10 selected drugs, but a dozen approved biosimilars referencing the three biologics in that round, 94 generics either approved or tentatively approved that reference the small molecules on the list, and perhaps other innovator drugs in the same therapeutic spaces. And by Feb. 1, CMS must publish the list of up to 15 drugs selected for negotiations for the 2028 price year. That list will be the first to include Part B drugs.

The U.S. Department of Health and Human Services reported Aug. 27 that Susan Monarez no longer occupies the post of director for the CDC, a development that arose within hours of the FDA’s approval of two COVID-19 vaccines with historically restrictive labeled indications.

Telix Pharmaceuticals Ltd. received an FDA complete response letter (CRL) a day after the Aug. 27 PDUFA date for its BLA for radiopharmaceutical renal cancer imaging agent, Zircaix (TLX250-CDx, 89Zr-DFO-girentuximab).

Twenty-two years since its formation, Bioarctic AB expects to become profitable in 2025, as milestones for marketing approvals and royalties on sales of the Alzheimer’s drug Leqembi (lecanemab) roll in, and partners sign up to use its proprietary Braintransporter drug delivery technology.

Leal Therapeutics Inc. is taking advantage of new genetic data that suggest products from metabolic pathways are involved in psychiatric and neurodegenerative diseases.

The recombinant fusion protein drug telitacicept from Remegen Co. Ltd. and Vor Bio Inc. has notched a phase III win in treating adults with IgA nephropathy. The clinical trial of the fusion protein hit the primary endpoint, reducing proteinuria, too much protein in patients’ urine, in stage A of the study in China.

Summit Therapeutics Inc.’s presentation for bispecific antibody ivonescimab in non-small-cell lung cancer could draw some extra attention at next month’s World Conference on Lung Cancer meeting thanks to promising overall survival data from partner Akeso Pharmaceuticals Inc.’s China study of the PD-1/VEGF candidate.