X4 Pharmaceuticals Gets FDA Approval for Xolremdi Capsules

By Chris Wack

X4 Pharmaceuticals said that the Food and Drug Administration approved Xolremdi capsules for use in patients 12 years of age and older with WHIM syndrome to increase the number of circulating mature neutrophils and lymphocytes.

The company said Xolremdi is the first therapy specifically indicated in patients with WHIM syndrome, an ultra-rare and difficult-to-diagnose primary immunodeficiency in which the body's immune system does not function properly, and has trouble fighting infections. WHIM refers to Warts, hypogammaglobulinemia, immunodeficiency, myelokathexis.

The FDA granted Breakthrough Therapy Designation to Xolremdi in WHIM syndrome and evaluated the New Drug Application under Priority Review, a designation for therapies that have the potential to provide significant improvement in the treatment, diagnosis, or prevention of serious conditions.

With the FDA approval of Xolremdi, X4 has received a Rare Pediatric Disease Priority Review Voucher that can be used to obtain priority review for a subsequent application or sold to another drug sponsor.

Write to Chris Wack at [e-mail]

(END) Dow Jones Newswires

04-29-24 0706ET