Two new drugs to treat plaque psoriasis are adding to the topical options available to treat the condition. Both payers and dermatologists have expressed interest in the agents, according to Zitter Insights, and the class remains a high priority for payer management.

On May 23, the FDA approved Roivant Sciences subsidiary Dermavant Sciences, Inc.’s Vtama (tapinarof) cream for the topical treatment of plaque psoriasis in adults, regardless of disease severity. The company said the agent is the first and only FDA-approved steroid-free topical medication in its class. Dosing of the aryl hydrocarbon receptor agonist is once daily on affected areas, and the drug has no restrictions on length of use. The price for one tube of the drug is $1,325.

In its latest drug pipeline report, UnitedHealth Group-owned PBM Optum Rx takes a look at two high-priced gene therapies that are slated for FDA approval soon. But those are far from the only gene therapies that payers should be watching, an Optum Rx executive tells AIS Health.

Both drugs highlighted in Optum’s report, Zynteglo (betibeglogene autotemcel) and Skysona (elivaldogene autotemcel), are manufactured by bluebird bio, Inc., a Massachusetts-based biotech company. An FDA advisory panel in June recommended both drugs for approval. Currently, only two gene therapies are on the U.S. market: Luxturna and Zolgensma.

July 14: The FDA expanded the patient population of Biocodex, Inc.’s Diacomit (stiripentol) for the treatment of seizures associated with Dravet syndrome in people between the ages of 6 months to 2 years and weighing at least 7 kg who are taking clobazam. The agency first approved the treatment on Aug. 20, 2018. The drug is available as a capsule and an oral suspension. Dosing is 50 mg/kg/day for both routes of administration. Drugs.com lists the price of 60 250 mg capsules and 60 250 mg powder for reconstitution as more than $1,589.

July 14: The FDA expanded the label of Pfizer Inc.’s Xalkori (crizotinib) to include the treatment of people at least 1 year old with unresectable, recurrent or refractory inflammatory anaplastic lymphoma kinase (ALK)-positive myofibroblastic tumors. The agency initially approved the kinase inhibitor on Aug. 26, 2011. The FDA gave the agent orphan drug designation and granted the application priority review; that review used the Assessment Aid, a voluntary submission from the applicant to assist the FDA in its analysis. Dosing for the newest indication of the capsule in adults is 250 mg twice daily. The recommended pediatric dosage is 280 mg.m2 twice daily based on body surface area. Drugs.com lists the price of 60 250 mg capsules as more than $20,657.

Spending in the U.S. on medicines increased by 12% in 2021, primarily because of COVID-19 vaccines and therapies, according to the IQVIA Institute’s annual report on drug trends. The report also found that “Despite an increase in overall spending, costs per prescription on average are flat or slightly declining. Prescription drug use reached a record 194Bn daily doses in 2021 as new prescription starts for both chronic and acute care recovered from the slowdown recorded in 2020…[but] spending on medicines is expected to return to pre-pandemic growth trend lines by 2023.”

A federal appeals court on July 25 held that a Pfizer Inc. coupon program violated the federal Anti-Kickback Statute. The Second Circuit Court of Appeals ruled that Pfizer offering copay assistance to Medicare beneficiaries was illegal under the statue. AHIP applauded the ruling, arguing that “By upholding these protections, the courts have taken an important step to protect Americans from what would otherwise be an unchecked multi-billion-dollar price tag that would make coverage and care less affordable for everyone.”

A recent FDA approval of a label expansion put the three marketed therapies for spinal muscular atrophy (SMA) on equal footing for the youngest patients. A recent survey found that many payers are covering sequential use of the costly agents, including a gene therapy.

The FDA initially approved Evrysdi (risdiplam) from Roche Group member Genentech USA, Inc. on Aug. 7, 2020, for the treatment of SMA in people at least 2 months old. On May 30, 2022, the agency expanded the drug’s label to include the treatment of babies less than 2 months old. The medication is an oral solution administered by mouth or feeding tube and can be administered by a patient or caregiver at home after a recommended consultation with a health care professional prior to the first dose.

June 6: The FDA expanded the label of Roche Group member Genentech USA, Inc.’s CellCept (mycophenolate mofetil) to include, in combination with other immunosuppressants, prophylaxis of organ rejection in people at least 3 months old who have received an allogenic heart transplant or an allogenic liver transplant. The agency first approved the drug on May 3, 1995. Dosing for the newest uses is based on body surface area and indication. The drug is available as a capsule, tablet, oral suspension and intravenous injectable. Website GoodRx.com lists the price of 60 500 mg tablets as more than $1,070.

June 7: The FDA granted another indication to Sanofi and Regeneron Pharmaceuticals, Inc.’s Dupixent (dupilumab) for the treatment of moderate-to-severe atopic dermatitis in people between the ages of 6 months and 5 years whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable. The agency initially approved the subcutaneous injectable on March 28, 2017. The FDA gave the new indication priority review. Dosing for people weighing 5 kg to less than 15 kg is 200 mg every four weeks; for those weighing 15 kg to less than 30 kg, dosing is 300 mg every four weeks. The drug’s list price, regardless of dose, is $3,384.83 per carton, which includes either two prefilled pens or two prefilled syringes.

A new drug to treat plaque psoriasis is the first topical novel chemical entity launched in the U.S. for the condition in 25 years. Both payers and dermatologists have expressed interest in the agent, according to Zitter Insights.

On May 23, the FDA approved Roivant Sciences subsidiary Dermavant Sciences, Inc.’s Vtama (tapinarof) cream for the topical treatment of plaque psoriasis in adults, regardless of disease severity. The company says the agent is the first and only FDA-approved steroid-free topical medication in its class. Dosing of the aryl hydrocarbon receptor agonist is once daily on affected areas, and the drug has no restrictions on length of use. The price for one tube of the drug is $1,325.

The FDA recently gave an additional indication to AbbVie Inc.’s Rinvoq (upadacitinib) in ulcerative colitis, broadening that therapeutic class even more. And while a study revealed some concerns around another agent with a similar mechanism of action, payers and gastroenterologists last year expressed interest in Rinvoq over other late-stage pipeline agents.

On March 16, the FDA expanded the label of Rinvoq to include the treatment of adults with moderately to severely active ulcerative colitis who have had an inadequate response or intolerance to at least one tumor necrosis factor (TNF) blocker. The agency initially approved the Janus kinase (JAK) inhibitor on Aug. 16, 2019. The recommended starting dose for the tablet is 45 mg once daily for eight weeks, followed by a maintenance dose of 15 mg once daily. The wholesale acquisition cost for a 30-day supply is $5,671.26.

May 10: The FDA granted full approval to Eli Lilly and Co.’s Olumiant (baricitinib) for the treatment of COVID-19 in hospitalized adults requiring supplemental oxygen, noninvasive or invasive mechanical ventilation, or extracorporeal membrane oxygenation. The agency first approved the Janus kinase (JAK) inhibitor on May 31, 2018. The drug has been available for COVID treatment since Nov. 19, 2020, under Emergency Use Authorization (EUA). The EUA remains in place for hospitalized people between the ages of 2 and 18 years old who require various degrees of oxygen support. The recommended dose of the tablet for the newest use is 4 mg once daily for 14 days or until hospital discharge, whichever occurs first. Alternative modes of administration via oral dispersion, gastrostomy tube, nasogastric tube or orogastric tube are available. The drug’s list price for a 30-day supply of 2 mg tablets is $2,497.20.

Through 2020, CMS spent $68 billion on 38 drugs that were granted accelerated approval from the FDA between 2012 and 2017, with spending after conversion to standard approval accounting for 75% of overall spending, according to a JAMA Health Forum study. However, only 34% of these drugs had a confirmatory trial evaluating a clinical outcome as a primary end point and more than $40 billion was spent for drugs evaluated using surrogate end points. Clinical trials for one drug that converted to standard approval (pembrolizumab) and three that remained unconverted (atezolizumab, durvalumab and olaratumab) for their original indications failed to confirm benefit for primary efficacy end points, while these drugs cost CMS $14 billion in total through 2020. The researchers concluded that “persistent evidentiary gaps should prompt payers to limit spending on promising drugs with unproven benefits.