Radar on Specialty Pharmacy

During the prior administration of Donald Trump, the then-president talked often about taking on pharmaceutical companies and PBMs over high spending on prescription drugs but did not accomplish all he set out to do. Since his reelection, PBMs in particular have been in his crosshairs, as he criticized the “rich as hell” and “horrible” middlemen, which he vowed to “knock out.” And with a potential cabinet of many newcomers to Capitol Hill — some of whom have openly criticized how HHS, including the FDA, is run — questions remain about the upcoming administration’s impact on health care, including how it could impact the specialty drug space. For our annual series of outlook stories on the year ahead, industry experts weigh in on the Trump effect. (Editor’s note: These comments have been edited for length and clarity.) 

Dec. 12: The FDA granted another indication to Dermavant Sciences, Inc. subsidiary Organon’s Vtama (tapinarof) for the treatment of atopic dermatitis in people at least 2 years old. The agency first approved the aryl hydrocarbon receptor agonist on May 23, 2022. The cream is applied to affected areas once daily. Drugs.com lists the price of 60 grams as more than $1,531.

Dec. 13: The FDA approved Checkpoint Therapeutics, Inc.’s Unloxcyt (cosibelimab-ipdl) for the treatment of adults with metastatic cutaneous squamous cell carcinoma or locally advanced CSCC who are not candidates for curative surgery or curative radiation. It is the first programmed death-ligand 1 (PD-L1) inhibitor approved for the second most common form of skin cancer. The agency’s review used the Assessment Aid. Dosing is 1,200 mg via a 30-minute intravenous infusion every three weeks. Checkpoint told Fierce Pharma that it will commercialize Unloxcyt “through one or more potential partnerships or other type of corporate development transaction to enable a potential launch in 2025.”

Yorvipath (palopegteriparatide) is now available in the U.S., Ascendis Pharma revealed Dec. 19. The FDA approved the prodrug of parathyroid hormone on Aug. 9, 2024, for the treatment of adults with hypoparathyroidism. The manufacturer says it is the first FDA-approved therapy for this indication. The company’s U.S. Ascendis Signature Access Program has a dedicated Yorvipath team to assist patients, caregivers and physicians.

The FDA issued a Drug Safety Communication about Intercept Pharmaceuticals, Inc.’s Ocaliva (obeticholic acid), the agency said on Dec. 12. The farnesoid X receptor agonist is indicated for the treatment of adult patients with primary biliary cholangitis (PBC) without cirrhosis or with compensated cirrhosis who do not have evidence of portal hypertension, either in combination with ursodeoxycholic acid with an inadequate response to UDCA or as monotherapy in patients unable to tolerate UDCA. The FDA said it had found “cases of serious liver injury” in people being treated for PBC who did not have cirrhosis. In evaluating liver safety in postmarket clinical trials involving people appropriate for treatment, the agency discovered that “the risk of both liver transplant and death were higher in patients receiving Ocaliva compared with those receiving placebo.” The agency first granted the drug accelerated approval on May 27, 2016. The FDA says that “frequent liver test monitoring is necessary to identify worsening liver function and ensure appropriate discontinuation of Ocaliva.” It noted that it had previously communicated similar risks and will continue to monitor the issue.