AllianceRx Walgreens Prime announced that it has access to a series of limited-distribution drugs (LDDs) for its specialty patients, including the recently approved Symdeko (tezacaftor/ivacaftor and ivacaftor) for cystic fibrosis (CF).
Symdeko, manufactured by Vertex Pharmaceuticals, is designed to treat CF in people ages 12 and older who have two copies of the F508del mutation or one mutation responsive to the medication. It comes at hefty cost, however: an estimated $292,000 for a year’s treatment.
The therapy was approved by the U.S. Food and Drug Administration in February, supported by results from two Phase 3 studies, EVOLVE (NCT02347657) and EXPAND (NCT02392234), involving approximately 750 CF patients.
Symdeko is a combination of VX-661 (tezacaftor) and ivacaftor (sold by Vertex as Kalydeco), two agents that together help improve the performance of the CFTR protein channel in CF patients, CFTR being the gene defective in CF.
VX-661 helps the CFTR protein move onto the cell surface so that, once there, Kalydeco can help the CFTR ion channel remain open for longer periods of time. Their combined action was shown to improve the balance of water and salts in patients’ lungs and other affected organs.
Trial results also showed that treatment significantly improved lung function and other measures when compared with placebo. Symdeko was also found to have a favorable safety profile.
An open-label Phase 3 extension study, EXTEND (NCT02565914), is currently evaluating its long-term safety and efficacy in patients who took part in earlier trials.
The the tezacaftor/ivacaftor combination is now under review by European Medicines Agency (EMA) for possible approval, with a decision expected in late 2018.
Symdeko is available at select Walgreens community-based specialty pharmacies in the U.S.
“Continually expanding on our industry-leading limited distribution drug list demonstrates our passion and commitment to making sure patients have access to the medications they need, including those that may be hard to find,” Joel Wright, chief executive officer for AllianceRx Walgreens Prime, said in a press release.
“We are pleased to offer these medications as additional options to help treat patients who have been diagnosed with these serious conditions, and continue to work with Walgreens at the community-based specialty pharmacies as well,” Wright added.