ZEVRA THERAPEUTICS (ZVRA) Company Report

Zevra Therapeutics reported Q4 total revenue of $106.5 million for MIPLYFFA, representing 30% quarter-over-quarter growth and exceeding the $100 million threshold.
MIPLYFFA has achieved 40%-50% market share in its first full year of launch, with 161 patient enrollment forms since launch, and the company is actively working to increase diagnosis rates for Niemann-Pick disease type C.
The company is focused on geographic expansion for MIPLYFFA, with global Expanded Access Program (EAP) patients reaching 113 and significant penetration in the European market.
Zevra is advancing its pipeline, with Celiprolol continuing patient enrollment for vascular Ehlers-Danlos syndrome and efforts underway to accelerate its clinical development.
Zevra has the capital to operate independently of capital markets to fund its U.S. launch, European expansion, and the Celiprolol program through launch.

1 day ago

Transcript

Zevra Therapeutics Reports Strong MIPLYFFA Performance and Outlines Growth Strategy

ZVRA

Revenue Acceleration/Inflection

Product Launch

New Projects/Investments

Zevra Therapeutics reported Q4 revenue of $106.5 million for MIPLYFFA, marking a 30% quarter-over-quarter growth and achieving 40-50% market share in its first full year of launch.
The company is actively working to increase the diagnosed patient population for Niemann-Pick disease type C (NPC) in the U.S., where an estimated 900 patients have the disease but only 300-350 are diagnosed, through genetic testing and awareness campaigns.
Zevra is pursuing geographic expansion for MIPLYFFA, with 113 global Expanded Access Program (EAP) patients (primarily in Europe) and is evaluating go-to-market strategies for Europe pending label approval.
Enrollment for Celiprolol in vascular Ehlers-Danlos syndrome trials is progressing, targeting a 7,500-patient U.S. market, with efforts to accelerate clinical development and regulatory options.
The company is financially independent of capital markets, possessing sufficient cash to fund the U.S. launch, potential European launch, and the Celiprolol program through launch.

1 day ago

Transcript

Zevra Therapeutics Discusses Strong MIPLYFFA Performance and Pipeline Progress

ZVRA

Revenue Acceleration/Inflection

Product Launch

New Projects/Investments

Zevra Therapeutics reported $106.5 million in total revenue for MIPLYFFA's first full year of launch, achieving 30% quarter-over-quarter growth in Q4.
MIPLYFFA has garnered 161 patient enrollment forms since launch, securing 40%-50% market share among the 300-350 diagnosed Niemann-Pick disease type C (NPC) patients in the U.S., with ongoing efforts to diagnose more of the 900 estimated prevalence.
The company is expanding MIPLYFFA's global access, with 113 global EAP patients and significant penetration in Europe, including pre-commercial revenue in France.
Zevra is also advancing Celiprolol for vascular Ehlers-Danlos syndrome (vEDS), a condition with 7,500 patients in the U.S., and is exploring regulatory pathways to accelerate its clinical development.
Zevra states it has the capital to independently fund its U.S. launch, European expansion, and the Celiprolol program through launch, without reliance on capital markets.

1 day ago

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Zevra Therapeutics Reports Strong Q4 and Full Year 2025 Financial Results, Driven by MIPLYFFA Sales

ZVRA

Earnings

Revenue Acceleration/Inflection

CFO Change

Zevra Therapeutics reported net revenue of $34.1 million for Q4 2025 and $106.5 million for the full year 2025, with MIPLYFFA sales contributing $26.4 million in Q4 and $87.4 million for the full year.
The company achieved net income of $12.2 million ($0.19 diluted EPS) in Q4 2025 and $83.2 million ($1.35 diluted EPS) for the full year 2025, a significant improvement from net losses in 2024.
MIPLYFFA demonstrated strong commercial momentum with 52 prescription enrollment forms in 2025, bringing total enrollments to 161 since launch, and secured 68% total covered lives in the U.S. market.
As of December 31, 2025, Zevra held $238.9 million in cash, equivalents, and investments and had approximately $61.9 million in total debt.
Justin Renz was appointed as the new Chief Financial Officer, effective on the day of the call, and the company is advancing its pipeline with 52 patients enrolled in the celiprolol study.

3 days ago

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Zevra Therapeutics Reports Q4 and Full Year 2025 Financial Results and Appoints New CFO

ZVRA

Earnings

CFO Change

Revenue Acceleration/Inflection

Zevra Therapeutics reported full year 2025 net revenue of $106.5 million, with MIPLYFFA sales contributing $87.4 million. For Q4 2025, total net revenue was $34.1 million, including $26.4 million from MIPLYFFA sales.
The company achieved net income of $83.2 million or $1.35 per diluted share for the full year 2025, and $12.2 million or $0.19 per diluted share for Q4 2025.
MIPLYFFA saw 52 prescription enrollment forms in the full year 2025, bringing the total to 161 enrollments since launch. The global Expanded Access Program (EAP) had 113 patients enrolled at the end of 2025.
Justin Renz was appointed as the new Chief Financial Officer, with his official start date on March 9, 2026.
As of December 31, 2025, cash, cash equivalents, and investments totaled $238.9 million, with total debt at approximately $61.9 million.

3 days ago

Transcript

Zevra Therapeutics Reports Strong Q4 and Full-Year 2025 Financial Results and Key Product Updates

ZVRA

Earnings

Product Launch

CFO Change

Zevra Therapeutics reported total net revenue of $34.1 million for Q4 2025 and $106.5 million for the full year 2025, driven by MIPLYFFA sales of $26.4 million in Q4 2025 and $87.4 million for the full year 2025.
The company achieved net income of $12.2 million ($0.19 diluted EPS) in Q4 2025 and $83.2 million ($1.35 diluted EPS) for the full year 2025. As of December 31, 2025, Zevra held $238.9 million in cash equivalents and investments.
MIPLYFFA saw 24 prescription enrollment forms in Q4 2025, bringing the total for 2025 to 52 enrollment forms and 161 total enrollments since launch. The Marketing Authorisation Application (MAA) for arimoclomol (MIPLYFFA) in Europe is under review by the European Medicines Agency, with responses to a 120-day list of questions due within the 90-day clock stop period.
The Phase 3 DiSCOVER study for celiprolol enrolled 8 patients in Q4 2025, reaching a total of 52 enrolled patients out of a planned 150 by year-end. The company is engaging with the FDA to discuss regulatory options to accelerate the development program.
Justin Renz was appointed as the new Chief Financial Officer, with his official start date on March 9, 2026.

3 days ago

Transcript

Zevra Therapeutics Provides Update on MIPLYFFA Launch and Pipeline at J.P. Morgan Healthcare Conference

ZVRA

Guidance Update

New Projects/Investments

Revenue Acceleration/Inflection

Zevra Therapeutics is a commercial-stage company focused on rare diseases, with its lead product, MIPLYFFA, for Niemann-Pick disease type C (NPC).
The company generated $72.3 million in net revenue for the first nine months of 2025 and held a strong cash position of $230.4 million at the end of Q3 2025.
Zevra's MIPLYFFA launch in the U.S. achieved approximately 40% of the diagnosed NPC patient population within 12 months post-launch, with 137 patient enrollments.
Key growth drivers include a Marketing Authorization Application (MAA) for MIPLYFFA under review by the EMA and an expected 2026 ruling on a potential patent term extension for MIPLYFFA in the U.S. beyond 2031.

Jan 15, 2026, 5:45 PM

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Zevra Therapeutics Discusses Growth Drivers and MIPLYFFA Expansion

ZVRA

Product Launch

New Projects/Investments

Revenue Acceleration/Inflection

Zevra Therapeutics reported net revenue of $72.3 million for the first nine months of 2025 and exited Q3 2025 with a strong cash position of $230.4 million.
The company's lead product, MIPLYFFA, for Niemann-Pick disease type C, was FDA approved in September 2024 and has orphan drug exclusivity through 2031.
A Marketing Authorization Application (MAA) for MIPLYFFA is under review by the EMA, and a potential patent term extension beyond 2031 is expected to be determined by the U.S. Patent Office in 2026.
MIPLYFFA has achieved approximately 40% of the diagnosed patient population in the U.S. within the first 12 months post-launch, and Zevra is pursuing global access through geographic expansion and distribution agreements.

Jan 15, 2026, 5:45 PM

Transcript

Zevra Therapeutics Reports Strong Financials and Miplyffa Launch Success at J.P. Morgan Healthcare Conference

ZVRA

Revenue Acceleration/Inflection

New Projects/Investments

Product Launch

Zevra Therapeutics generated $72.3 million in net revenue for the first nine months of 2025 and exited Q3 2025 with a strong cash position of $230.4 million.
The company's lead product, Miplyffa, achieved approximately 40% penetration of the diagnosed Niemann-Pick disease type C patient population within 12 months post-launch following its FDA approval in September 2024.
Zevra is pursuing geographic expansion for Miplyffa with an MAA under review by the EMA and anticipates a 2026 determination on a patent term extension that could prolong Miplyffa's exclusivity beyond 2031.
The company's strong balance sheet was further enhanced by the $150 million monetization of a Pediatric Priority Review Voucher in 2025.
Zevra is progressing its pipeline, with 44 of 150 patients enrolled in the Phase III DiSCOVER trial for Celiprolol for vascular Ehlers-Danlos syndrome as of Q3.

Jan 15, 2026, 5:45 PM

Transcript

Zevra Therapeutics Reports Q3 2025 Financials and Provides Pipeline Updates

ZVRA

Earnings

Product Launch

New Projects/Investments

Zevra Therapeutics reported $72.3 million in net revenue for the nine-month period ending September 30, 2025, and $26.1 million for Q3 2025, resulting in a Q3 2025 net loss of $(0.5) million.
The company maintained a strong cash position, with $230.4 million in available cash, cash equivalents, and investments as of September 30, 2025.
MIPLYFFA® for Niemann-Pick Disease Type C (NPC), FDA-approved on September 20, 2024, achieved adoption in approximately 40% of diagnosed patients within its first year of launch and secured 66% payor coverage as of Q3 2025.
The Marketing Authorization Application (MAA) for arimoclomol (NPC) is under review by the EMA since July 28, 2025, and the Phase 3 DISCOVER trial for celiprolol (Vascular Ehlers-Danlos Syndrome) has 44 of 150 patients enrolled as of Q3 2025.
Zevra is pursuing a possible patent term extension for MIPLYFFA and planning geographic expansion for its products beyond the U.S. and Europe.