Pfizer Inc.’s bivalent prefusion vaccine for protecting newborns from severe respiratory syncytial virus (RSV) hit one of its two primary endpoints in its phase III study, which was good enough for the company to stop enrollment and plan to submit a BLA to the U.S. FDA by year-end. RSV affects about 5,000 young people weekly in the U.S. and can be deadly for newborns. PF-06928316 is one of six RSV vaccines in active phase III development globally. On Sept. 20, Astrazeneca plc and Sanofi SA saw their RSV prevention drug, Beyfortus (nirsevimab), approved by EU regulators, creating a potential approval and launch runway in 2023. Also in the pack is GSK plc, whose RSV vaccine in older people posted strong phase III numbers, too.

HI-Bio advances with $120M financing for immune-mediated disease therapies

Human Immunology Biosciences Inc. (HI-Bio), a company developing targeted therapies for severe immune-mediated diseases, has secured $120 million in financing. Its initial pipeline is built around two clinical-stage immunology assets in-licensed from Morphosys AG in June 2022. Incubated by Arch Venture Partners and cross-Atlantic investor Monograph Capital, the company has now added Jeito Capital, institutional investors and family offices to its backers. It's Jeito’s first investment in the U.S.

China bans GSK from participating in volume-based tenders following failed GMP inspection

Shanghai’s State Medical Products Administration announced that GSK plc has been banned from participating in volume-based procurement tenders until April 29, 2024, after failing a good manufacturing practices (GMP) inspection at a contract manufacturing plant in Poland that makes products for China.

US FDA updates its advice on expanded access

Reflecting statutory and regulatory requirements added to expanded access over the past five years, the U.S. FDA is issuing a revised draft question-and-answer (Q&A) guidance on expanded access to investigational drugs. One of several guidances recently issued, the draft incorporates requirements from the 21^st Century Cures Act and the 2017 FDA Reauthorization Act that took effect after the current final Q&A guidance was issued in 2017. Other guidances the agency is publishing this week include a draft on evaluating pubertal growth in pediatric trials and a small entity compliance guide on the regulation of human cells, tissues, and cellular and tissue-based products.

Newco news: Replay launches first HSV-focused firm, Eudora, with eye on retinal disease

“Big genomics” specialist Replay Holdings LLC has unveiled the first of four satellite genomic medicine companies it is forming to apply its high capacity herpes simplex viral (HSV) vector to next generation gene therapies. Eudora Therapeutics will specialize in inherited retinal eye diseases. It arrives on the scene with programs targeted at retinitis pigmentosa, Stargardt disease and Usher syndrome type 1B.

October lags other months for FDA approvals and regulatory activity

Despite a busy September, U.S. FDA approvals and global regulatory news fell in October to the lowest point this year. So far in 2022, the FDA has approved 127 drugs and biologics, including supplemental filings. This is 25% less than each of the last two years. The last time approvals were lower than this year was 2016 when there were 121. The 28 new molecular entities (NMEs) approved this year are also at their lowest point since 2016, which had 19 by the end of October. Most recently, approved NMEs include Astrazeneca plc’s Imjudo (tremelimumab, a CTLA4 antibody for hepatocellular carcinoma, and Janssen Pharmaceutical Co.’s Tecvayli (teclistamab), a bispecific antibody for relapsed or refractory multiple myeloma.

Also in the news

Acelrx, Actinium, Alzamend Neuro, Amicus, Antengene, Ardelyx, BMS, Cerecin, Cytodyn, Disc Medicine, Dragonfly, Dyne, Eucure, First Wave, Innocare, Instil, Kyowa Kirin, Ligand, Memo, Merck, Mosaic, Nicox, Nuvalent, Omniab, Ono, Orsobio, Pharmather, Pharmazz, Phathom, Santhera, Turnstone Biologics, United, Vaccitech