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news.The European Medicines Agency (EMA) has validated X4 Pharmaceuticals’ marketing authorisation application (MAA) submitted for mavorixafor, aimed at treating WHIM syndrome, a rare primary immunodeficiency.
The once-daily oral therapy is a small-molecule antagonist of CXCR4 receptor. Credit: CDC on Unsplash.
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The application is currently being evaluated by the EMA’s Committee for Medicinal Products for Human Use (CHMP).
Mavorixafor would become the first drug for this syndrome in the European region, if EMA approves it.
Earlier, the therapy received orphan status from EMA for this condition.
X4 Pharmaceuticals CEO and president Paula Ragan said: “Making mavorixafor available to those in the European Union living with WHIM syndrome is a top priority for X4 and this submission demonstrates our continued ability to deliver on our key milestones and generate growth.
“With our MAA now validated for review by the EMA, we expect to enable our recently announced European partner, Norgine, to provide this much-needed treatment to patients as rapidly as possible should it be approved. We look forward to working alongside the EMA as they assess our application.”
The US Food and Drug Administration (FDA) approved the oral mavorixafor under the name of XOLREMDI, in April last year.
This one-time-a-day therapy is intended for treating the syndrome in individuals aged 12 years and above to boost the circulating mature neutrophils and lymphocytes.
The oral therapy is a small-molecule antagonist of the C-X-C chemokine receptor type 4 (CXCR4).
In the global Phase III 4WHIM trial of the company, mavorixafor has shown a decrease in the infection severity, duration, and rate in subjects with this syndrome.
It was also generally ‘well’ tolerated without any treatment-related serious adverse events reported. Discontinuations for safety events were also not observed.
The trial met its primary endpoint as well as a key secondary endpoint.
An exclusive licensing and supply agreement was announced by X4 earlier this month, with Norgine, for commercialising the drug in Australia, New Zealand, and Europe.
