X4 Pharmaceuticals announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending marketing authorization for its oral CXCR4 antagonist, mavorixafor (XOLREMDI), for the treatment of WHIM syndrome in the European Union. The opinion was adopted on February 26, 2026, and the company disclosed the decision to the public on February 27, 2026.
The positive CHMP opinion is a critical regulatory milestone that could bring the first and only therapy for WHIM syndrome to European patients. The European Commission will review the recommendation, and a final approval decision is expected in the second quarter of 2026.
Under the existing licensing and supply agreement with Norgine, X4 is eligible to receive up to €226 million in milestone payments and ongoing royalties once the drug is approved in the EU. The partnership gives X4 a non‑dilutive funding source that supports its broader strategy to focus resources on the Phase 3 chronic neutropenia trial while maintaining a revenue stream from WHIM syndrome sales.
X4’s financial position remains modest. In the third quarter of 2025, the company reported an earnings per share of –$0.69 and revenue of $1.77 million, both below expectations. Cash and cash equivalents stood at $87.7 million as of March 31 2025, providing a runway into the first half of 2026. A restructuring in February 2025, which included a 30 % workforce reduction and the closure of its Vienna facility, was undertaken to reduce annual spending by $30–$35 million and to concentrate resources on the chronic neutropenia program.
The company’s chief executive, Paula Ragan, said, “This strategic partnership is a significant milestone for X4 as we seek to maximize the global potential of mavorixafor and bring in funding for our ongoing global, Phase 3 trial in chronic neutropenia.” She added that the partnership would allow X4 to “focus on executing the 4WARD trial while building WHIM communities through our U.S. commercial presence.”
The chronic neutropenia program, which targets a larger patient population than WHIM syndrome, is advancing through a pivotal Phase 3 trial (4WARD). Enrollment is expected to be completed by the third or fourth quarter of 2025, with top‑line data anticipated in the second half of 2026. The EMA orphan drug designation for WHIM syndrome, granted in July 2019, further supports the drug’s development pathway.
The positive CHMP opinion also reinforces X4’s position in the rare‑disease market and provides a foundation for future growth. By securing a potential EU launch, X4 can expand its commercial footprint beyond the United States, where XOLREMDI is already approved, and generate additional revenue streams that will help sustain its pipeline investments.
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