The U.S. Centers for Medicare & Medicaid Services (CMS) rolled out the negotiated costs of the second batch of drugs subject to such bargaining. CMS said the adjusted maximum fair prices would have achieved 44% lower net spending – 36% if forgiven discounts from the part D redesign of the Medicare prescription drug benefit are figured in – had they been implemented in 2024. Fifteen drugs are included.

Otsuka’s IgAN therapy gets a thumbs-up from the FDA

The priority BLA from Otsuka Pharmaceutical Co. Ltd., of Tokyo, and its U.S. subsidiary, Otsuka Pharmaceutical Development & Commercialization Inc., for sibeprenlimab to treat immunoglobulin A nephropathy (IgAN) has received accelerated approved by the U.S. FDA. The humanized monoclonal antibody, branded Voyxact, is for reducing proteinuria, too much protein in the kidneys, in adults who have primary IgAN. The therapy is designed to block action of the cytokine A proliferation inducing ligand (APRIL) for treating the rare kidney disease. A progressive autoimmune disorder, IgAN can worsen over the years and lead to kidney failure, requiring dialysis.

Despite expectations, insulin biosimilars see slow uptake in the US

The Sept. 4, 2015, at-risk launch of Sandoz Inc.’s Zarxio as the first biosimilar to hit the U.S. market came several months after the FDA had approved the filgrastim biosimilar due to a court battle over the requirements of the 2010 Biologics Price Competition and Innovation Act, which laid out the rules of the road for the new class of follow-on drugs. Ten years later, biosimilar developers are still struggling with some of those rules that were drafted by Congress in an effort to balance competition with innovation in the biologics space. Insulin biosimilars may be the hardest hit.

Vigencell’s cell therapy for lymphoma meets phase II endpoint

Vigencell Inc. plans to seek conditional approval in South Korea for VT-EBV-N, an antigen-specific killer T-cell therapy for natural killer T-cell lymphoma (NKTCL), after gaining positive top-line data from a phase II study Nov. 25. “This is the world’s first case of a NKTCL-targeting cell therapy to show efficacy in clinical trials,” Vigencell CEO Ki Pyung-suk said. “We will move quickly with regulatory processes and commercialization, both domestically and globally, to offer patients a valuable treatment option.” According to top-line findings, the study met its primary endpoint, defined as two-year disease-free survival, with statistical significance.

Myrio first to develop binders with high affinity/specificity

Myrio Therapeutics Pty Ltd. has been able to accomplish something no other company has yet been able to crack: to develop binders where both the affinity and the specificity can be increased. “We're able to do this through a proprietary technology we developed called ReD [for Retained Display],” Myrio’s CEO Graeme Wald told BioWorld, explaining that the discovery platform is essentially a cell display system that allows Myrio to rapidly develop a huge library of potential binders to pick the best candidates for a specific peptide human leukocyte antigen (pHLA) complex. From there, the binder is optimized to make sure it has the appropriate binding kinetics. These binders have repeatedly shown their utility in bispecific T-cell engagers or CAR T formats to direct cytotoxicity against pHLA-specific target tumor cells.

In vivo gene editing to halt the clock before it’s too late

A 24‑week pregnant woman fears for her unborn baby, who is developing with a sacrococcygeal teratoma so large and vascularized that it nearly surpasses the size of the fetus itself. The mass, attached to the base of the coccyx, diverts much of the blood flow and forces the tiny heart to pump with extreme effort, leading to heart failure. Faced with this threat, surgeons operate inside the uterus in an open procedure that partially exposes the baby to remove the tumor and give the baby a chance to survive until birth. According to scientists, this could be avoided. “We are now at a point, in prenatal care, where most human genetic as well as anatomic abnormalities can or will be able to be diagnosed before birth. And unfortunately, some of these diseases can cause significant health problems before or immediately after a baby is born,” Bill Peranteau, pediatric surgeon at the Children’s Hospital of Philadelphia, told the audience during the Muscle Session of the special meeting Breakthroughs in Targeted In Vivo Gene Editing.

Merck’s Keytruda and Welireg power October’s phase III oncology wins

In October 2025, BioWorld tracked 252 clinical trial updates across phases I-III. Of those, 27 phase III studies reported positive results, while four disclosed failures. In the cancer space, Merck & Co. Inc. reported positive results from two phase III trials: the Keynote-B96 study of Keytruda (pembrolizumab) in platinum-resistant recurrent ovarian cancer and Welireg (belzutifan) in advanced renal cell carcinoma.

Holiday notice

BioWorld's offices will be closed in observance of Thanksgiving in the U.S. No issues will be published Thursday, Nov. 27, or Friday, Nov. 28.

Also in the news

Ascentage, Biogen, BMS, Eisai, Faron, Geneditbio, Gilead, Innovent, Junshi, Liora, Lixte, Lunai, Novartis, Novo Nordisk, Otsuka, Pharmacyte, Psyence, Regeneron, Sanofi, Sarepta, Sprint, Tempest, Teva, Toolgen, Zetagen