The FDA recently approved an ORAL Tx, Xolremdi (mavorixafor) from X4 Pharmaceuticals, indicated in patients 12 years of age and older with WHIM (warts,hypogammaglobulinemia, infections and myelokathexis) syndrome to increase the number of circulating mature neutrophils and lymphocytes. WHIM is the first therapy specifically indicated in patients with WHIM syndrome, a rare, combined primary immunodeficiency and chronic neutropenic disorder caused by CXCR4 pathway dysfunction.
People with WHIM syndrome characteristically have low blood levels of neutrophils (neutropenia) and lymphocytes (lymphopenia) and experience serious and/or frequent infections. X4 Pharmaceuticals cited that at least 1,000 people are currently diagnosed with WHIM syndrome in the U.S.
The company has set a wholesale acquisition cost for Xolremdi on an annual basis at $496,400 for patients greater than 50 kilograms and $372,300 for patients less than or equal to 50 kilograms.
In a rare event in itself, X4 announced that Xolremdi will be commercially available in the U.S. through specialty pharmacy partner PANTHERx Rare.
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X4 Pharmaceuticals Announces FDA Approval of Xolremdi (mavorixafor) Capsules, First Drug Indicated in Patients with WHIM Syndrome
