X4 PHARMACEUTIC. DL-,001

The European Commission has approved XOLREMDI (mavorixafor) from X4 Pharmaceuticals for once‑daily oral treatment of patients aged 12 and older with WHIM syndrome. The approval was granted under exceptional circumstances and is based on the Phase 3 4WHIM study; mavorixafor is thus the first and only approved therapy for WHIM in the US and the EU. Norgine will lead commercialization in Europe, Australia and New Zealand following regulatory approvals; under the licensing agreement X4 can receive up to €226 million in milestone payments plus tiered royalties. » Plus sur de.investing.com

At the Leerink Global Healthcare Conference, X4 Pharmaceuticals presented a strategic refocus on chronic neutropenia and the regulatory Phase‑3 study 4WARD for mavorixafor. Recruitment for the study is expected to be completed by the end of Q3 2026, with top‑line data anticipated in 2027 and commercialization for 2028 fully funded. The company reports significant cost reductions, personnel additions from CTI BioPharma, and positions mavorixafor as an oral alternative to G‑CSF for a U.S. market estimated at ~15,000 symptomatic patients. » Plus sur de.investing.com

At the Guggenheim Summit, X4 Pharmaceuticals outlined its priorities: focus on the Phase‑3 4WARD study for mavorixafor, operating cost cuts and workforce reductions to consolidate resources. The oral drug is expected to offer advantages over G‑CSF; the addressable market is seen at about 15,000 patients, with roughly 5,000 patients to be treated initially. Commercialization for WHIM syndrome has been postponed; the pricing strategy foresees a reduced but still premium level versus the current list price of about USD 500,000. Approval is targeted for 2028. » Plus sur de.investing.com