Having the rare disease pulmonary arterial hypertension increases the risk of blood clots in the ... [+] lungs' small arteries. The Food and Drug Administration approved Winrevair last week. The drug is indicated for a rare and deadly cardiovascular disorder called pulmonary arterial hypertension. PAH has long been challenging to treat , as STAT News reports. As such, Winrevair represents a potentially important breakthrough. The approval of Winrevair (sotatercept) comes on the heels of the FDA’s licensing of another PAH treatment, a tablet called Opsynvi which combines macitentan and tadalafil. Winrevair could be disease-modifying—it decreases the likelihood of worsening clinical events—as it inhibits a growth factor that is overproduced by people with PAH. This leads to thickening of the walls of blood vessels, which in turn can cause high blood pressure and a heavy strain on the heart due to constriction of lung arteries. The New
England Journal of Medicine published Phase 3 trial data which showed adults with PAH who were given sotatercept achieved greater improvement in six-minute walk distance,* experienced better overall functioning and had a significantly (84%) reduced risk for clinical deterioration or death at 24 weeks compared with the placebo group. Here, a person’s functioning is measured on a World Health Organization-designated scale of “functional classes.” There are four such classes that are used to rate how ill PAH patients are. The higher the class the more severe the disease and the greater the urgency for the patient to be accurately diagnosed and started on therapy. PAH affects around 40,000 people in the U.S. and 43% of patients die within five years of diagnosis. Up to 50% of PAH patients may be eligible for Winrevair. The drug is expected to be used as a second- or third-line therapy, but eventually may have first-line potential , according to FirstWord Pharma . Winrevair should arrive on (specialty) pharmacy shelves by late April, Healio reports . The therapeutic is injected once every three weeks and can be self-administered by patients. It remains an open question whether people prescribed sotatercept will need to still take the cocktail of drugs that help ease symptoms. The drug’s sponsor, Merck, will sell Winrevair for a list price of approximately $14,000 per vial or roughly $240,000 a year, depending on a person’s weight. This figure does not account for certain discounts to payers which will be factored into the net price. The high annual list price puts Winrevair well above commonly cited cost-effectiveness thresholds, even when accounting for the benefits if confers. The cost-effectiveness watchdog, the Institute for Clinical and Economic Review, considered the use of sotatercept added to existing therapy among adults with PAH versus existing therapy alone and concluded that if Winrevair had a net price of between roughly $18,000 and $35,000 a year, it would meet typical thresholds. Since it’s highly unlikely the net yearly price of Winrevair will be close to the $18,000 to $35,000 range, this may be considered problematic by payers and pharmacy benefit managers. To reduce their financial exposure, it’s plausible they will impose a set of utilization tools, including placement of the drug in a relatively high patient cost-share tier, extensive use of prior authorization and possibly step edits (a cheaper drug may have to be tried first prior to Winrevair being covered). They could also exclude from coverage certain patients assigned to a low or less severe WHO functional class. Finally, payers may apply duration limits while assessing dose titration, side effects and disease progression. For those who take the drug and are on Medicare, Pink Sheet noted that the soon-to-be implemented $2,000 out-of-pocket cost-sharing cap will help lower their financial burden. This cap comes into force in 2025 as part of the Inflation Reduction Act’s redesign of Medicare’s outpatient (Part D) pharmacy benefit. At the same time, however, as part of the Part D overhaul in 2025 pharmaceutical manufacturers will face a 20% mandatory price discount in the so-called catastrophic or high-cost phase of the Medicare drug benefit. In addition, given that payers will soon be responsible for managing a much higher share of the cost of pharmaceuticals, they will probably impose utilization restrictions that may limit access. The newly approved Winrevair is a promising therapy for PAH patients. Its high price and correspondingly limited cost-effectiveness could, however, restrain its uptake. * The six-minute walk test assesses a person’s ability to walk farther in six minutes than they could before they started taking the drug.