Radar on Specialty Pharmacy

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.

Cellular therapies made meaningful achievements in 2024, with two of them involving a pair of chimeric antigen receptor T cell (CAR-T) therapies that gained FDA approval for use in earlier lines of setting for the treatment of relapsed or refractory multiple myeloma (RRMM). According to a recent survey, some payers already have been making exceptions to allow for earlier use of CAR-Ts than what is indicated on their labels.

On April 4, 2024, the FDA approved Abecma (idecabtagene vicleucel) from Bristol Myers Squibb and 2seventy bio, Inc. for the treatment of adults with triple-class exposed RRMM after at least two lines of therapy. The following day, the FDA approved Carvykti (ciltacabtagene autoleucel) from the Janssen Pharmaceutical Companies of Johnson & Johnson and Legend Biotech USA, Inc. for the treatment of adults with RRMM who have received at least one prior line of therapy. Both agents had previously been approved for the treatment of RRMM after at least four lines of therapy.

Krystal Biotech, Inc.’s Vyjuvek (beremagene geperpavec-svdt) soon may have a gene therapy competitor, and the FDA soon may approve the first biosimilar of Roche Group member Genentech USA, Inc. and Novartis Pharmaceuticals Corp.’s Xolair (omalizumab). That's according to AscellaHealth LLC’s latest breakdown of insights into new, pending and upcoming launches in specialty and rare disease, biosimilars and cell and gene therapies (CGTs).

AscellaHealth partners with life sciences companies to offer customizable solutions to support the specialty pharmaceutical industry. Its Specialty and Rare Pipeline Digest: Q4 2024 takes an expansive look at the specialty industry, from the launches of the biosimilars of Stelara (ustekinumab) from Johnson & Johnson Innovative Medicine to the new atopic dermatitis indication for Galderma Laboratories, L.P.’s Nemluvio (nemolizumab-ilto).