ZEVRA THERAPEUTICS (ZVRA) Company Report

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

Transcript

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.

Jan 15, 2026, 5:45 PM

Presentation

Zevra Therapeutics Discusses Niemann-Pick Disease Type C Launch and European MAA

ZVRA

Product Launch

New Projects/Investments

Revenue Acceleration/Inflection

Zevra Therapeutics is a commercial-stage rare disease company with two commercial products, a late-stage Phase 3 development program, and royalties from an ADHD program, supported by a robust balance sheet of approximately $230 million in capital.
The company's Niemann-Pick disease type C (NPC) product, Miplyffa, has achieved 137 prescription enrollment forms in the U.S., covering about 40% of the diagnosed patient population (300-350 patients) since its launch less than a year ago.
Zevra has filed a Marketing Authorization Application (MAA) for Miplyffa in Europe, which is currently under review with responses expected by the end of 2025, targeting an estimated 1,100 NPC patients in the region.
Efforts in disease state awareness and genetic testing are leading to the diagnosis of new NPC patients, expanding the estimated U.S. market opportunity beyond the initial 300-350 diagnosed patients, potentially up to 900 patients.
In Europe, Zevra has 92 patients in an Expanded Access Program (EAP), including 30 patients in France where the AAP program generates approximately $10 million annually in pre-commercial revenue.

Nov 12, 2025, 4:30 PM

Transcript

Zevra Pharmaceuticals Discusses Strong Niemann Pick Disease Type C Launch and European Expansion

ZVRA

Product Launch

New Projects/Investments

Revenue Acceleration/Inflection

Zevra Pharmaceuticals is a commercial-stage rare disease company with two commercial products and a late-stage Phase III development program in vascular Ehlers Danlos syndrome, supported by a balance sheet with approximately $230 million in capital.
The company's U.S. launch of its Niemann Pick disease type C (NPC) treatment has garnered 137 prescription enrollment forms, representing about 40% of the diagnosed patient population (300-350 patients), and is now identifying newly diagnosed patients beyond initial estimates.
Zevra is expanding its NPC treatment into Europe, having filed a marketing authorization application (MAA) that has been validated and is on a 120-day clock for responses by the end of 2025 for an estimated 1,100 prevalent patients.
In Europe, Zevra has 92 patients enrolled in an Expanded Access Program (EAP), with 30 patients in France contributing approximately $10 million in annual pre-commercial revenue.
The company reports high adherence and persistency rates for its NPC treatment, MyPlayfa, with physicians and payers favoring the agent due to strong long-term data.

Nov 12, 2025, 4:30 PM

Transcript

Zevra Therapeutics Provides Update on Niemann-Pick Disease Type C (NPC) Program and European Expansion

ZVRA

Product Launch

New Projects/Investments

Zevra Therapeutics is a commercial-stage rare disease company with two commercial products for urea cycle disorders and Niemann-Pick disease type C (NPC), a late-stage Phase III program in vascular Ehlers-Danlos Syndrome, and royalties from an ADHD program, supported by a robust balance sheet of approximately $230 million in capital.
For its Niemann-Pick disease type C (NPC) product, Miplyffa, the company has secured 137 prescription enrollment forms in the U.S. since its commercial launch at the end of November last year, representing about 40% of the diagnosed patient population of 300-350 patients, with 8 new forms added in the last quarter.
The company filed a Marketing Authorization Application (MAA) in Europe for Miplyffa at the end of July (2025), with responses expected by the end of 2025, targeting a prevalent patient population of approximately 1,100 patients.
Market research indicates strong positive perception of Miplyffa, with patients reporting improvements in gait and swallow, physicians preferring it based on long-term data, and high adherence and persistency rates. The company also noted $10 million in annual pre-commercial revenue from its European Expanded Access Program (EAP) in France.

Nov 12, 2025, 4:30 PM

Transcript

ZVRA Reports Q3 2025 Financial Results with Strong Miplyf Performance

ZVRA

Earnings

Product Launch

Revenue Acceleration/Inflection

Zevra reported net revenue of $26.1 million for Q3 2025, with Miplyf contributing $22.4 million, and significantly reduced its net loss to $500,000 ($0.01 per share) compared to a $33.2 million net loss in Q3 2024.
Miplyf, a treatment for Niemann-Pick Disease Type C, added 8 new prescription enrollment forms in Q3 2025, reaching a total of 137 since its launch one year ago, now covering approximately 40% of the diagnosed U.S. patient population.
The company is pursuing European expansion for Miplyf, with a Marketing Authorization Application under review by the European Medicines Agency and 92 patients enrolled in its European Expanded Access Program. As of September 30, 2025, Zevra's cash, cash equivalents, and investments increased to $230.4 million, with total debt at approximately $61.3 million.

Nov 5, 2025, 9:30 PM

Transcript

Zevra Therapeutics Reports Strong Q3 2025 Financial Results and Strategic Updates

ZVRA

Earnings

Revenue Acceleration/Inflection

New Projects/Investments

Zevra Therapeutics reported net revenue of $26.1 million for Q3 2025, a significant increase from $3.7 million in Q3 2024, primarily driven by MIPLYFFA net revenue of $22.4 million.
The company achieved a net loss of $(0.5) million, or $(0.01) per basic and diluted share, for Q3 2025, a substantial improvement compared to a net loss of $(33.2) million in Q3 2024.
As of September 30, 2025, Zevra's cash, cash equivalents, and securities totaled $230.4 million.
Zevra has decided to scale back sales and marketing efforts for OLPRUVA while evaluating options, despite market access reaching 81% of covered lives.
A Marketing Authorisation Application for arimoclomol (MIPLYFFA) for the treatment of Niemann-Pick disease type C is currently under review by the European Medicines Agency (EMA).

Nov 5, 2025, 9:08 PM

8-K

Zevra Therapeutics Reports Q3 2025 Financials and Provides Business Update

ZVRA

Earnings

Product Launch

New Projects/Investments

As of September 30, 2025, Zevra Therapeutics reported a strong financial position with $230.4 million in cash, cash equivalents, and investments, alongside $61.3 million in debt.
For Q3 2025, the company generated net revenue of $26.1 million, which included $22.4 million from MIPLYFFA and $100,000 from OLPRUVA, resulting in a net loss of $(0.5) million for the quarter.
Zevra has two commercial-stage rare disease products in the U.S.: MIPLYFFA® for Niemann-Pick Disease Type C, which received FDA approval on September 20, 2024, and OLPRUVA® for Urea Cycle Disorders, approved on December 22, 2022.
The company's advanced clinical development pipeline includes arimoclomol for NPC, with an MAA filing under review by the EMA, and celiprolol in an ongoing Phase 3 trial for Vascular Ehlers-Danlos Syndrome (VEDS).