Radar on Specialty Pharmacy

The cell and gene therapy (CGT) space seems to be a bit of a mixed bag recently. While last year’s accomplishments included FDA approvals of two new first-in-class treatments for solid tumors and the first chimeric antigen receptor T cell (CAR-T) therapy since 2022, on back-to-back days last month, the news was more downbeat.

On Feb. 20, reports that Pfizer Inc. was discontinuing its hemophilia B gene therapy Beqvez (fidanacogene elaparvovec) globally were first publicized and later confirmed by the drugmaker. The FDA had approved the single-dose $3.5 million treatment less than a year prior, on April 25, 2024.

As CMS undergoes its pricing negotiations on the second round of drugs under the Medicare Drug Price Negotiation Program, a recent report reveals that manufacturer concerns over the program’s potential threat to drug development may be warranted. Researchers at the IQVIA Institute for Human Data Science analyzed initial drug approvals and expansions that followed and found that due to some aspects of the program, clinical development after a drug’s initial approval may indeed be impacted, according to a new report, titled Proliferation of Innovation Over Time: Frequency, Timing and Clinical Value of Expansions Post-Initial Approval.

Genentech USA, Inc., a member of the Roche Group, provided funding for the report.

Feb. 11: The FDA approved a new tablet formulation of Roche Group subsidiary Genentech USA, Inc.’s Evrysdi (risdiplam) for the treatment of spinal muscular atrophy in people at least 2 years old who weigh at least 20 kg. The agency first approved the survival of motor neuron 2 (SMN2) splicing modifier on Aug. 7, 2020, as an oral solution; that formulation remains available. Dosing for the tablet is 5 mg once daily. Drugs.com lists the price of 60 mg of oral powder (0.75 mg/mL solution) as more than $14,062.

Feb. 12: The FDA expanded the label of Astellas Pharma Inc.’s Izervay (avacincaptad pegol) to remove the 12-month limit on treatment duration for the treatment of geographic atrophy secondary to age-related macular degeneration (AMD). The agency first approved the complement C5 inhibitor on Aug. 4, 2023. Dosing is 2 mg administered by intravitreal injection to each affected eye once monthly without a limitation on duration of dosing. The drug’s list price per single-dose vial is $2,100.

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.