Radar on Specialty Pharmacy

CMS revealed its public engagement events for the second round of Medicare drug price negotiations on Feb. 21. The agency will hold two livestreamed town halls for all selected drugs on April 30, as well as 15 private patient-focused roundtables, one for each drug and organized alphabetically, from April 16 through April 29.

ImmunityBio, Inc. said on Feb. 19 that the FDA has authorized it to provide next-generation recombinant Bacillus Calmette-Guerin (rBCG) to the U.S. via an expanded access program to treat people with bladder cancer. The agent, which has been developed by the Serum Institute of India, has undergone Phase II clinical trials in Europe for the treatment of non-muscle invasive bladder cancer (NMIBC). BCG has been the standard of care for people with NMIBC since 1977, but it has been in short supply for several years. Merck & Co., Inc. became the sole supplier of BCG in the U.S. in 2012, and the current shortage began in 2019, when the company began allocating the available supply. Merck has said it is constructing a new manufacturing facility in Durham, North Carolina, to expand its production capacity for BCG.

While the “Big Three” PBMs’ private-label subsidiaries have drawn recent attention for their agreements with pharma companies to offer their own branded biosimilars, one of these planned arrangements has landed a manufacturer in hot water.

Johnson & Johnson and its Janssen Biotech, Inc. subsidiary — now known as Johnson & Johnson Innovative Medicine — on Feb. 24 filed a lawsuit (Case 2:25-cv-01439) against Samsung Bioepis Co. Ltd. over its attempts to sell a private-label biosimilar of J&J’s interleukin-12/IL-23 antagonist Stelara (ustekinumab) via an unidentified PBM private-label subsidiary in addition to Samsung’s Pyzchiva (ustekinumab-ttwe) biosimilar.

Amgen Inc. recently revealed that it had settled its patent infringement litigation against Celltrion Inc. over its denosumab biosimilar candidates that could allow them to launch onto the U.S. market this summer. The arrangement means that two different companies may be launching biosimilars of Amgen’s receptor activator of nuclear factor-kappa B ligand (RANKL) inhibitors Prolia and Xgeva on consecutive days.

The decision (No. 1:24-cv-06497-CPO-EAP), filed Jan. 23 in the U.S. District Court for the District of New Jersey, noted that the parties agreed that Celltrion’s products infringe on 29 Amgen patents. The court issued an injunction preventing Celltrion “from making, using, offering to sell, or selling” the biosimilars in the U.S. until June 1, 2025.

Since the FDA approved the first cell therapy, the chimeric antigen receptor T cell (CAR-T) agent Kymriah (tisagenlecleucel) from Novartis Pharmaceuticals Corp. on Aug. 30, 2017, several similar cellular agents have followed, and 2024 was a particularly noteworthy year. Accomplishments included approvals of two new types of cell therapies for treating solid tumors and the first new CAR-T since 2022. But with research booming, a “radical change” is needed to pay for these advances.

Such advancements come with high costs, however, and payers will be challenged to find novel strategies to enhance affordability and access, market experts tell AIS Health, a division of MMIT. While value-based agreements are one such strategy, payers are having issues with providers staying in the agreements.

Chimeric antigen receptor T cell (CAR-T) therapies had quite the year in 2024, with the first approval of a new agent in nearly three years, as well as a handful of expanded indications, including two that moved the moved the agents into an earlier line of treatment.

On Nov. 8, 2024, the FDA approved Autolus Therapeutics plc’s Aucatzyl (obecabtagene autoleucel) for the treatment of adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia. That marked the first new CAR-T since the Feb. 28, 2022, approval of Carvykti (ciltacabtagene autoleucel) from the Janssen Pharmaceutical Companies of Johnson & Johnson and Legend Biotech USA, Inc.  While Aucatzyl shares similar black box warnings about cytokine release syndrome (CRS), neurologic toxicities and secondary hematological malignancies as the other CAR-Ts, Autolus noted that the new therapy is the first such agent approved without a Risk Evaluation Mitigation Strategy (REMS) program.