FDA Approves Zevra’s MIPLYFFA™ for Niemann-Pick Disease Type C

XOMA Royalty Corporation, a biotechnology royalty aggregator, announced that the FDA has approved MIPLYFFA™ (arimoclomol) for the treatment of Niemann-Pick disease type C (NPC). This drug is to be used in combination with miglustat and is suitable for adult and pediatric patients aged two years and older. This marks the first time a therapy has been authorized for NPC in the U.S., a serious neurodegenerative disease.

Owen Hughes, CEO of XOMA Royalty, highlighted the significance of this approval, noting that it provides NPC patients with the first FDA-approved treatment for this debilitating condition. He emphasized that clinical data suggests MIPLYFFA™ combined with miglustat may improve patient outcomes and slow disease progression.

In addition to becoming a vital therapy for NPC patients, MIPLYFFA™ is also the sixth commercial asset in XOMA Royalty’s expanding portfolio. Earlier, XOMA Royalty had announced a $5 million upfront payment to LadRx and agreed to share future event-based milestones, acquiring a mid-single digit royalty on the commercial sales of arimoclomol. This agreement also includes up to $52.6 million in potential milestone payments from Zevra Therapeutics.

XOMA Royalty Corporation specializes in acquiring future economic rights associated with both pre-commercial therapeutic candidates and established commercial assets. These assets are typically licensed to pharmaceutical or biotechnology firms. By purchasing these future economic rights, XOMA Royalty offers non-dilutive, non-recourse funding to sellers, allowing them to advance their internal drug candidates or support general corporate needs.

The company boasts a broad and growing portfolio consisting of over 70 assets. Each asset grants the right to receive potential future revenues stemming from the development of an underlying therapeutic candidate. XOMA Royalty’s diverse portfolio includes both commercialized drugs and investigational compounds. Their commercial assets now include VABYSMO® (faricimab-svoa), OJEMDA™ (tovorafenib), MIPLYFFA™ (arimoclomol), XACIATO™ (clindamycin phosphate vaginal gel 2%), IXINITY® [coagulation factor IX (recombinant)], and DSUVIA® (sufentanil sublingual tablet).

The corporation plays a distinctive role in the biotech industry by aiding biotech companies in their mission to improve human health. By providing the necessary funding, XOMA Royalty enables these companies to further develop their therapeutic candidates without diluting ownership or incurring debts.

Overall, the approval of MIPLYFFA™ represents a significant milestone not only for NPC patients but also for XOMA Royalty’s business strategy of aggregating biotech royalties. The company continues to focus on expanding its portfolio of milestone and royalty rights, with a clear aim to generate substantial revenues over time through its partnerships and licensed technologies.