Zevra’s MIPLYFFA Receives FDA Approval for Use in Patients With Niemann-Pick Disease Type C


XOMA Royalty Corporation recently announced Zevra Therapeutics has received approval from the US FDA for MIPLYFFA (arimoclomol). MIPLYFFA is approved for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients two years of age and older.  It is the sixth commercial asset in XOMA Royalty’s growing royalty and milestone portfolio.

“With the approval of MIPLYFFA, NPC patients in the United States now have access to the first FDA approved therapeutic in this rare, progressive and fatal neurodegenerative disease,” said Owen Hughes, Chief Executive Officer of XOMA Royalty.  “Based on the clinical data to date, we believe MIPLYFFA plus miglustat has the potential to improve outcomes and slow disease progression for many NPC patients.”

In June 2023, XOMA Royalty announced it had paid LadRx a $5-million upfront payment plus a share of future event-based milestones to acquire a mid-single digit royalty on arimoclomol’s commercial sales and up to $52.6 million, net, in potential milestone payments from Zevra.

XOMA Royalty is a biotechnology royalty aggregator playing a distinctive role in helping biotech companies achieve their goal of improving human health.  XOMA Royalty acquires the potential future economics associated with pre-commercial therapeutic candidates and commercial assets that have been licensed to pharmaceutical or biotechnology companies.  When XOMA Royalty acquires the future economics, the seller receives non-dilutive, non-recourse funding they can use to advance their internal drug candidate(s) or for general corporate purposes.  The Company has an extensive and growing portfolio with more than 70 assets (asset defined as the right to receive potential future economics associated with the advancement of an underlying therapeutic candidate).  For more information, visit www.xoma.com.