By David Bautz, PhD
READ THE FULL XFOR RESEARCH REPORT
Enterprise Replace
XOLREMDI Permitted by the FDA
On April 29, 2024, X4 Prescription drugs, Inc. (NASDAQ:XFOR) introduced that the U.S. Meals and Drug Administration (FDA) has authorised XOLREMDI (mavorixafor) to be used in sufferers 12 years of age and older with WHIM syndrome (warts, hypogammaglobulinemia, infections, and Myelokathexis). That is the primary remedy particularly indicated for WHIM syndrome. Together with the approval of XOLREMDI, X4 additionally obtained a Uncommon Pediatric Illness Precedence Overview Voucher (PRV). We anticipate the corporate monetizing this asset within the close to future as PRVs are sometimes promoting for about $100 million.
Through the convention name to announce the approval of XOLREMDI, X4 administration offered a top level view of the corporate’s commercialization technique, which incorporates doctor schooling (together with offering diagnostic assist for affected person testing), establishing XOLREMDI as the usual of care in WHIM syndrome, and making certain broad entry by means of a collection of affected person assist providers (X4Connect).
With reference to pricing, X4 introduced an annual wholesale acquisition price (WAC) of $496,400 for sufferers >50 kg (400 mg day by day) and $372,300 for sufferers ≤50 kg (300 mg day by day; assuming full affected person compliance). The corporate has partnered with PantheRx Uncommon, a specialty pharmacy that may course of and fill XOLREMDI prescriptions and supply info on remedy, potential uncomfortable side effects, and ensure remedy dosing.
We don\’t anticipate a bolus of sufferers prepared to start remedy straight away, however as a substitute the corporate will deal with growing doctor and payer consciousness of XOLREMDI throughout the remainder of 2024 such that gross sales can start to ramp up in 2025/2026. The corporate has assembled a gross sales power of roughly two dozen representatives who collectively have over 250 years of expertise in industrial launches. When a affected person begins remedy, the X4Connect program shall be utilized to assist them navigate insurance coverage protection, any prior authorization necessities, and introduce them to monetary help applications.
Conclusion
We’re very glad to see the approval of XOLREMDI and are assured that X4 has a robust plan in place to make sure a profitable industrial launch of the drug. We assume that the PRV shall be monetized within the close to future and are assured that it is going to be offered in-line with earlier PRVs (~$100 million). Apart from the industrial launch of XOLREMDI, we proceed to anticipate Part 2 power neutropenia information within the first half of 2024. We now have adjusted our mannequin to account for the truth that XOLREMDI has been authorised, which has triggered a slight improve in our valuation to $5.50 per share.
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