EMA CHMP Recommends EU Approval for WHIM Therapy, Norgine Deal Triggers €226M Milestones
EMA’s CHMP issued a positive opinion recommending exceptional-circumstances approval of mavorixafor for WHIM syndrome in the EU, with final European Commission decision expected in Q2 2026. X4 Pharmaceuticals stands to earn up to €226 million in milestones plus escalating royalties under its Norgine deal.
1. Regulatory Milestone
The EMA’s CHMP issued a positive opinion recommending exceptional-circumstances marketing authorization of mavorixafor for treatment of WHIM syndrome in the EU, with final European Commission approval expected in Q2 2026.
2. Clinical Data
The positive opinion follows the 4WHIM Phase 3 trial in 31 patients, which demonstrated significant improvements in time above threshold for absolute neutrophil counts (p<0.0001) and lymphocyte counts (p<0.0001), a 40% reduction in infection scores and a 60% decrease in annualized infection rate versus placebo.
3. Commercial Partnership
Under the January 2025 licensing and supply agreement, X4 Pharmaceuticals will earn up to €226 million in regulatory and commercial milestones plus escalating double-digit royalties on net sales in Europe, Australia and New Zealand, while partner Norgine handles market access and commercialization and X4 supplies the therapy.