Zevra's Expanded-Access Reimbursements Fall as MIPLYFFA Rises

The Tip Desk · November 10, 2026 · 3 min read

Third-quarter net revenue was $37.8 million, including $34.0 million from MIPLYFFA, as global Expanded Access Program reimbursements dropped to $3.6 million.

Zevra Therapeutics, Inc. (ZVRA), a commercial-stage company focused on rare-disease therapies, reported third-quarter net revenue of $37.8 million, up 45% from $26.1 million a year earlier after a 53% year-over-year increase in the second quarter.

MIPLYFFA (arimoclomol), the company’s approved treatment for Niemann-Pick disease type C, generated $34.0 million of that total, up from $30.2 million in the second quarter. OLPRUVA contributed $0.2 million. Global Expanded Access Program net reimbursements were $3.6 million, down from $9.0 million in the prior quarter. The $0.3 million of AZSTARYS past-due royalties and other reimbursements reported in the second quarter did not appear in the third-quarter mix.

The company received 13 U.S. MIPLYFFA prescription enrollment forms in the quarter, bringing the cumulative total since launch to 197, after 14 forms in the second quarter. Global EAP enrollment stood at 134 patients as of September 30, 2026, versus 132 as of June 30. In the European Union, Zevra requested a re-examination of its Marketing Authorisation Application for arimoclomol in NPC and said it is awaiting the outcome. After publishing pediatric data, the company met with the FDA to discuss a potential supplemental New Drug Application for MIPLYFFA in infants under 24 months and anticipates filing in the first quarter of 2027.

Net income was $19.2 million, or $0.31 a share basic and $0.26 diluted, compared with $8.8 million, or $0.14 a share, in the second quarter. The company recognized $5.1 million of noncash income from fair-value adjustments on warrant and contingent value rights liability in the third quarter. Income from operations was $11.6 million.

Cost of product revenue, excluding non-cash intangible amortization, was $1.8 million. Operating expenses were $24.1 million, including $2.7 million of non-cash stock compensation. Research and development expense was $4.9 million, which the company said was due primarily to ongoing investment in arimoclomol efforts and the celiprolol development program. Selling, general and administrative expense was $19.1 million, which the company attributed primarily to commercial and infrastructure-related costs.

Cash, cash equivalents and investments were $270.9 million as of September 30, 2026, versus $260.2 million at June 30. The company said that, based on its current operating forecast, available financial resources are sufficient to execute on its strategic priorities. Common shares outstanding were 59,439,959; fully diluted shares were 68,968,909.

In the event-driven Phase 3 DiSCOVER trial of celiprolol for vascular Ehlers-Danlos syndrome, Zevra enrolled six patients in the quarter, bringing the total to 72 with four confirmed events, after four patients in the second quarter. The company said it recently engaged the FDA for a Type C meeting to discuss a potential submission strategy for celiprolol.

President and Chief Executive Officer Neil F. McFarlane said the company remained focused on reaching more people with NPC, advancing celiprolol, and pursuing European access to arimoclomol, while continuing to make arimoclomol available through the global Expanded Access Program.