Norgine's marketing authorisation of XOLREMDI® marks a significant advancement in treating WHIM syndrome, benefiting patients across the EU.

Norgine's recent EU marketing authorization for XOLREMDI® marks a significant step for WHIM syndrome patients, opening new treatment options.

Norgine has received EU marketing authorisation for XOLREMDI® (mavorixafor), the first treatment for WHIM syndrome, enhancing care for affected individuals.

X4 Pharmaceuticals (Nasdaq:XFOR) reported financial results for the quarter ended June 30, 2026 and detailed progress across its rare hematology portfolio. Revenue was $8.8 million for the quarter and $11.5 million for the first half of 2026, including $2.4 million and $4.8 million in net U.S. XOLREMDI® product sales and $6.4 million and $6.7 million in license and other revenue, largely from its Norgine agreement.Research and development expenses declined to $15.1 million in the quarter, while general and administrative expenses fell to $8.5 million. Net loss narrowed to $16.2 million, or $(0.13) per share, for the quarter. Cash, cash equivalents, and marketable securities totaled approximately $208 million at June 30, 2026, which the company said provides a cash runway into 2029. Operationally, X4 transitioned management of its global Phase 3 4WARD chronic neutropenia trial to a premier CRO, continued enrolling patients at over 110 sites, and expects an FDA meeting update and enrollment completion timing by the end of the third quarter of 2026. The European Commission granted marketing authorization for XOLREMDI for WHIM syndrome, with commercialization in Europe led by partner Norgine and up to an additional €221 million in potential milestones plus escalating double-digit royalties.

Patients in the EU with the ultra-rare disease WHIM syndrome now have their first approved treatment, Norgine's Xolremdi.

With the approval of X4 Pharmaceuticals’ first product mavorixafor, there’s a new commercial rare disease player in town. | Following a 10-year development journey, the FDA on Monday approved X4 Pharm...

Reported Wednesday, Apr 29 - 9:37am EDT (TheFly subscription required to read more.)

Reported Wednesday, Feb 19 - 7:39am EST (TheFly subscription required to read more.)

Norgine announces that the European Commission has granted marketing authorization for XOLREMDI®, the first treatment for WHIM syndrome, marking a pivotal moment for patients.

Key approval by the European Commission for Norgine's XOLREMDI® signifies a breakthrough for patients with WHIM syndrome, providing much-needed therapy.

X4 Pharmaceuticals (Nasdaq: XFOR) reported Q1 2026 results and a corporate update on May 6, 2026. Key items: EU marketing authorization for XOLREMDI (mavorixafor) for WHIM syndrome, continued enrollment progress in the global 4WARD Phase 3 chronic neutropenia trial, and a $233.7M cash balance providing runway through 2028. The Norgine license could yield up to €226M in milestones plus escalating royalties; Q1 revenue was $2.7M versus $28.8M prior year, and net loss was $(20.2)M.

X4 Pharmaceuticals (Nasdaq: XFOR) announced the EMA CHMP has issued a positive opinion recommending marketing authorization under exceptional circumstances for mavorixafor (XOLREMDI) to treat WHIM syndrome in the EU. The European Commission is expected to issue a final decision in Q2 2026.CHMP support is based on the Phase 3 4WHIM trial showing statistically significant increases in ANC and ALC thresholds, ~40% reduction in total infection score, and a 60% reduction in annualized infection rate versus placebo. X4 has a commercialization license with Norgine and may receive up to €226 million plus escalating royalties.

X4 Pharmaceuticals (XFOR) reported Q1 2025 financial results and corporate updates. The company's 4WARD Phase 3 trial for chronic neutropenia is progressing well, with full enrollment expected in Q3/Q4 2025 and top-line data in 2H 2026. X4 reported net revenue of $28.8 million, including $27.9 million from the Norgine agreement and $0.9 million from XOLREMDI product sales. Since its May 2024 launch, XOLREMDI has generated $3.5 million in U.S. sales. The company ended Q1 with $87.7 million in cash, sufficient to fund operations into 1H 2026. Key developments include EMA acceptance of mavorixafor's MAA for WHIM syndrome, new international partnerships with Norgine Pharma UK and taiba rare, and a strategic restructuring expected to reduce annual spending by $30-35 million. The company recently completed a 1-for-30 reverse stock split.

X4 Pharmaceuticals (XFOR) reported its Q4 and full year 2024 financial results, highlighting significant progress in commercializing XOLREMDI® for WHIM syndrome and advancing clinical trials. Key financial metrics include $2.6 million in XOLREMDI® net revenues since its May 2024 launch, and $102.8 million in cash position as of December 31, 2024.The company's Phase 3 4WARD trial for chronic neutropenia has reached ~90% site activation, with full enrollment expected in Q3/Q4 2025 and top-line data anticipated in 2H 2026. Notable achievements include the sale of a priority review voucher for $105 million and strategic partnerships in EU/ANZ and MENA regions, with Norgine Pharma providing a €28.5 million upfront payment.Financial results show R&D expenses of $81.6 million and SG&A expenses of $61.5 million for 2024. The company implemented a strategic restructuring in February 2025, expected to reduce annual spending by $30-35 million. X4 projects sufficient funding into first half of 2026.

X4 Pharmaceuticals' mavorixafor gains EMA validation for WHIM syndrome review following US approval. Could become Europe's first treatment for rare immunodeficiency disorder.