X4 Pharmaceuticals Enters Distribution and Commercialization Agreement for WHIM Syndrome Therapy in Middle East

Rare Daily Staff

X4 Pharmaceuticals entered into an agreement with Taiba Rare to distribute and commercialize the company’s Xolremdi, a drug used to treat WHIM syndrome, a rare immunodeficiency.

The deal gives Taiba Rare exclusive rights to Xolremdi in select Middle East countries, including Saudi Arabia, the United Arab Emirates, Qatar, Oman, Kuwait, Bahrain, and Egypt, following any approvals in the region.

WHIM syndrome is a chronic neutropenic disorder caused by CXCR4 receptor dysfunction that impaired white blood cells mobilization from the bone marrow into peripheral circulation. WHIM syndrome is named for its four classic manifestations: warts, hypogammaglobulinemia, infections, and myelokathexis (accumulation of neutrophils in the bone marrow), although only a minority of patients experience all four manifestations. People with WHIM syndrome characteristically have low blood levels of neutrophils (neutropenia) and lymphocytes (lymphopenia), and as a result, experience serious and/or frequent infections.

The U.S. Food and Drug Administration approved Xolremdi in April 2024 for the treatment of patients 12 years of age and older with WHIM syndrome. Xolremdi is a selective CXCR4 receptor antagonist used orally once daily to increase the number of circulating mature neutrophils and lymphocytes. The European Medicines Agency’s Committee for Medicinal Products for Human Use is currently reviewing a marketing authorization application for the therapy.

Taiba will lead the distribution, promotion, marketing, and sales of Xolremdi within its territory, working jointly with X4 on key strategic decisions. Local regulatory filings for Xolremdi approval will be based on X4’s registration dossier submitted to the FDA. Pending any regulatory approvals in the region, Taiba is expected to be able to provide Xolremdi to WHIM patients through a compassionate use program that allows physicians to prescribe medicines approved in other countries to local patients with no other treatment options. The companies did not release financial terms of the agreement.