The first targeted therapy to treat pain crises in people with sickle cell disease presents a “welcome” new option that payers likely will embrace, a PBM head tells AIS Health. While the drug’s manufacturer cites “positive” early discussions with payers on it, some experts note the lifetime treatment — via a monthly intravenous infusion — is costly: around $100,000 annually.

On Nov. 15, the FDA approved Novartis’ Adakveo (crizanlizumab-tmca), a treatment to fight the underlying cause and reduce the frequency of vaso-occlusive crisis, described as a common and painful complication of sickle cell disease. It is approved for patients ages 16 and older with the genetic blood disorder.

Hydroxyurea, a drug approved by the FDA in 1998, is now generic, costs about $1,000 a year, and is approved for children, the New York Times reported on Dec. 7. The two newcomers are Adakveo and Global Blood Therapeutics’ Oxbryta (voxelotor), a daily pill granted accelerated approval by the FDA 10 days after Adakveo’s approval. This led one expert to tell the news outlet that insurers likely will want to begin with hydroxyurea as the front-line therapy.

Yet Mesfin Tegenu, R.Ph., president of PerformRx, LLC, says that “options for patients with sickle cell disease have been very limited up to this point, so the approval of Adakveo is a welcome addition in the treatment of this debilitating disease.”

Eric Althoff, a Novartis spokesperson, says the company anticipates that health plans will see a value proposition with Adakveo. “Early discussions with payers are positive,” Althoff says. “In fact, a number of payers have already added Adakveo to medical policy including state Medicaid [programs].” Florida and Alabama’s Medicaid programs have agreed to cover Adakveo, Reuters reported on Dec. 20.