Radar on Specialty Pharmacy

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).

From identifying eligible patients and training providers to seeking reimbursement and collecting data, cell and gene therapies (CGTs) present unique challenges to the health care system. One company has launched a new offering with an eye on simplifying one aspect of the process.

Cardinal Health recently unveiled its Advanced Therapy Connect, a unified ordering portal for CGTs. Providers have had to log in to different product platforms with distinct reporting structures and procedures for invoicing and reimbursement. But the new solution allows providers to order all in-network CGTs within a single platform, helping decrease errors from manual inputs and optimizing access to the agents.

Patient nonadherence to therapy can be an issue for people with cancer, despite the potential for poor prognosis and remission, several studies have found. A recent study from Walgreen Co. is reinforcing the idea that supporting cancer patients and giving them tools to help remain adherent can benefit both patients and payers.

Walgreens’ Connected Care Oncology (CC-Onc) is a patient-centered clinical management program that offers 21 oral hematological cancer therapies. The program utilizes a high-touch, personalized approach to boost adherence and improve outcomes. Researchers sought to find “significant associations” between discontinuation rates for those oral oncolytics and total medical costs, hospitalizations and inpatient length of stay, according to a recent poster presentation.

Jan. 15: The FDA approved Eli Lilly and Co.’s Omvoh (mirikizumab-mrkz) for the treatment of adults with moderately to severely active Crohn’s disease. The agency first approved the interleukin-23p19 (IL-23p19) inhibitor on Oct. 26, 2023. Dosing for the newest indication starts with 900 mg via a 90-minute-plus intravenous infusion at weeks zero, four and eight; maintenance dosing is 300 mg via subcutaneous injection at week 12 and then every four weeks. The agent’s list price is $9,593.22 for one 300mg/15ml infusion and $10,878.70 for two 100mg injections.

Jan. 15: The FDA approved two new vial sizes of Incyte Corp. and Syndax Pharmaceuticals’ Niktimvo (axatilimab-csfr) for the treatment of chronic graft-versus-host disease (GVHD) after the failure of at least two lines of systemic therapy in adults and pediatric patients weighing at least 40 kg. The agency first approved the colony stimulating factor-1 receptor (CSF-1R)-blocking antibody on Aug. 14, 2024, as a 50 mg single-dose vial, and it now also has 9 mg and 22 mg vials. The recommended dose is 0.3 mg/kg via a 30-minute intravenous infusion every two weeks. At the time of the initial approval, the company said that it was waiting to launch until those smaller vial sizes were approved. All three sizes are now available. The price of a 9 mg vial is $4,725, and the price of the 22 mg vial is $11,550.