Radar on Specialty Pharmacy

March 10: The FDA approved Acadia Pharmaceuticals Inc.’s Daybue (trofinetide) for the treatment of Rett syndrome in people at least 2 years old. The drug is the only FDA-approved treatment for the complex, rare neurodevelopmental disorder, which is estimated to affect 6,000 to 9,000 people in the U.S. The agency gave the drug fast track status and orphan drug designation; it also gave the company a rare pediatric disease priority review voucher. Dosing of the oral solution is weight based and can be given orally or via gastrostomy tube. The company has not disclosed the agent’s price, but analyst estimates are between $400,000 and $600,000 per year. Acadia said it expects the agent to be available by the end of April.

March 13: The FDA expanded the patient population of Mirum Pharmaceuticals, Inc.’s Livmarli (maralixibat) to include the treatment of cholestatic pruritus in people with Alagille syndrome who are at least 3 months old. The agency initially approved the oral solution on Sept. 29, 2021. The starting dose is 190 mcg/kg once daily and then increased to 380 mcg/kg after one week. Drugs.com lists the price of 9.5 mg/mL for 30 milliliters as more than $53,712.

A little more than a year after Rep. James Comer (R-Ky.), then-ranking member of the House Committee on Oversight and Reform, released a report on PBMs’ business practices and their negative impact on drug prices, patients’ health and market competition, the now-Chairman Comer revealed that he is launching an investigation into those practices. The move continues to keep pressure on the entities over some of their practices and lack of transparency amid concerns around prescription drug prices.

On March 1, Comer sent letters to senior officials at the Office of Personnel Management (OPM), Defense Health Agency (DHA) and CMS, as well as to the three largest PBMs — Cigna Corp.’s Express Scripts, CVS Health Corp.’s Caremark and UnitedHealth Group’s Optum Rx — requesting a trove of documents and communications.

The U.S. biosimilars market is a dynamic and ever-evolving space, and a recent report shows growing acceptance of the medications among providers. Last year saw a handful of milestones related to these agents, and this year, particularly with the launches of biosimilars of AbbVie Inc.’s Humira (adalimumab), is likely to continue the trend, according to a recent report from Cardinal Health.

Titled 2023 Biosimilars Report: tracking market expansion and sustainability amidst a shifting industry, the report is based on responses to web-based surveys conducted in 2022 from both community- and hospital-based practices. Providers were from “therapeutic areas with the most potential for disruption in the year ahead.” Cardinal Health polled more than 100 rheumatologists in July and August, 125 dermatologists in September, 70 gastroenterologists in September and October, and 60 retina specialists in September and October.

The FDA recently approved a new HIV drug for a small patient population desperately in need of treatments. And the twice-yearly medication’s annual price came below the level that respondents to a Zitter Insights poll said they would consider a good value. The medication comes with both potential advantages and disadvantages for patients, providers and payers, say industry experts.

On Dec. 22, the FDA approved Gilead Sciences, Inc.’s Sunlenca (lenacapavir) for the treatment, in combination with other antiretroviral(s) (ARVs), of HIV-1 infection in heavily treatment-experienced adults with multidrug resistant HIV-1 infection who are failing their current antiretroviral regimen due to resistance, intolerance or safety considerations. The agency gave the first-in-class capsid inhibitor priority review, fast track and breakthrough therapy designations.