Radar on Specialty Pharmacy

As expected, multiple biosimilars of Stelara (ustekinumab) from Johnson & Johnson Innovative Medicine (formerly Janssen Biotech, Inc.) launched onto the U.S. market in February, offering significant discounts off the reference drug’s list price.

As of mid-March, the FDA has approved seven biosimilars of Stelara for all of the reference drug’s indications: the treatment of adults and pediatric patients at least 6 years old with moderate-to-severe plaque psoriasis who are candidates for phototherapy or systemic therapy, adults and pediatric patients at least 6 years old with active psoriatic arthritis, adults with moderately to severely active Crohn’s disease and adults with moderately to severely active ulcerative colitis.

The FDA recently approved a new antibody-drug conjugate (ADC) for the treatment of the most common form of breast cancer. The treatment offers a new option for a disease with less-than-impressive survival rates. While payers expect it to have some impact in their management of the condition, oncologists said they anticipate it having more of an impact on their prescribing, according to a Zitter Insights survey.

On Jan. 17, the FDA approved Daiichi Sankyo, Inc. and AstraZeneca’s Datroway (datopotamab deruxtecan-dlnk or Dato DXd) for the treatment of adults with unresectable or metastatic hormone receptor positive (HR+), human epidermal growth factor receptor 2 negative (HER2-) breast cancer who have received endocrine-based therapy and chemotherapy for unresectable or metastatic disease. The agency gave the drug breakthrough therapy designation and priority review, which used the Assessment Aid for the Trop-2-directed antibody and topoisomerase inhibitor conjugate. The recommended dose is 6 mg/kg via intravenous infusion every three weeks, and the price of one 100 mg single-dose vial is $4,891.07.

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.