Zevra Therapeutics Enters Agreement to Sell its Rare Pediatric Disease Priority Review Voucher for $150 Million

Rhea-AI Summary

Zevra Therapeutics (ZVRA) has entered into an agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for $150 million in gross proceeds. The transaction is expected to close within 30-45 days, subject to customary conditions.

The PRV was awarded to Zevra in September 2024 following FDA approval of MIPLYFFA™ (arimoclomol), a treatment for neurological manifestations of Niemann-Pick disease type C in patients 2 years and older. The non-dilutive capital will support the commercial launches of MIPLYFFA™ and OLPRUVA®, along with advancing the company's rare disease pipeline.

Positive

• Secured $150M non-dilutive funding through PRV sale
• Strengthens balance sheet with significant cash infusion
• Transaction expected to close within 30-45 days
• Funds support commercial launches of two products

Negative

• None.

Insights

Financial Analyst

Zevra Therapeutics' sale of its Rare Pediatric Disease Priority Review Voucher (PRV) for $150 million represents a transformative financial transaction for the company. With a current market cap of approximately $393 million, this non-dilutive capital infusion is equivalent to 38% of Zevra's entire market value – a substantial balance sheet strengthening without shareholder dilution.

PRVs are highly valuable, transferable assets in the pharmaceutical industry that entitle holders to an expedited 6-month FDA review of a future drug application instead of the standard 10-month review. Large pharmaceutical companies often acquire these vouchers to accelerate potential blockbuster drug approvals, creating a secondary market that smaller companies like Zevra can capitalize on.

This transaction demonstrates excellent capital management, as Zevra is monetizing a regulatory asset just five months after receiving FDA approval for MIPLYFFA. The $150 million proceeds will significantly extend Zevra's operational runway, likely providing sufficient capital to support both commercial launches and continued pipeline development without near-term financing concerns.

For context, Zevra now has two FDA-approved rare disease therapies (MIPLYFFA and OLPRUVA) that require commercial infrastructure investment. The PRV sale provides the financial flexibility to execute robust launches while continuing development work on their pipeline candidates – a difficult balancing act for smaller biopharma companies that often forces pipeline prioritization or dilutive financing.

This transaction represents efficient monetization of a regulatory asset at a critical growth stage, potentially accelerating Zevra's transition to a fully integrated commercial-stage rare disease company with multiple revenue streams.

CELEBRATION, Fla., Feb. 27, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today announced that it has entered into a definitive asset purchase agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for gross proceeds of $150 million upon the closing of the transaction, which is expected to take place within 30 to 45 days, subject to customary closing conditions.

LaDuane Clifton, Zevra’s Chief Financial Officer said, “This non-dilutive capital strengthens our balance sheet by adding gross cash proceeds of $150 million, supporting continued investment in our strategic priorities, which include executing the commercial launches of MIPLYFFA™ and OLPRUVA®, and advancing our pipeline of product candidates to address unmet needs within the rare disease community.”

The PRV was granted to Zevra in September 2024 following approval by the U.S. Food and Drug Administration of MIPLYFFA (arimoclomol), which is indicated for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older. The transaction is subject to customary closing conditions, including expiration of the applicable waiting period under the Hart-Scott Rodino Antitrust Improvements Act (HSR). Cantor Fitzgerald acted as Zevra’s exclusive financial advisor and Latham & Watkins LLP acted as Zevra’s legal advisor for this transaction.

About Zevra Therapeutics, Inc.

Zevra Therapeutics, Inc. is a commercial-stage company combining science, data, and patient need to create transformational therapies for rare diseases with limited or no treatment options. Our mission is to bring life-changing therapeutics to people living with rare diseases. With unique, data-driven development and commercialization strategies, the Company is overcoming complex drug development challenges to make new therapies available to the rare disease community.

For more information, please visit www.zevra.com or follow us on X and LinkedIn.

Cautionary Note Concerning Forward-Looking Statements

This press release may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include all statements that do not relate solely to historical or current facts, including without limitation statements regarding the potential benefits of any of our products or product candidates for any specific disease or at any dosage; our strategic and product development objectives; the consummation and benefits of the transaction and its impact on the Company’s balance sheet; the outcome of any required filings under the HSR; prescription enrollments; our ability to support patients as they navigate the benefits verification process to obtain either MIPLYFFA™ or OLPRUVA®; availability of and access to MIPLYFFA and OLPRUVA; and the timing of any of the foregoing. Forward-looking statements are based on information currently available to Zevra and its current plans or expectations. They are subject to several known and unknown uncertainties, risks, assumptions, and other important factors that may cause our actual results, performance, or achievements to be materially different from any future results, performance, or achievements expressed or implied by the forward-looking statements. Given these risks and uncertainties, you should not place undue reliance on these forward-looking statements. These and other important factors are described in detail in the "Risk Factors" section of Zevra’s Annual Report on Form 10-K for the year ended December 31, 2023, Zevra’s Quarterly Report on Form 10-Q for the three and nine months ended September 30, 2024, and Zevra’s other filings with the Securities and Exchange Commission]. While we may elect to update such forward-looking statements at some point in the future, except as required by law, we disclaim any obligation to do so, even if subsequent events cause our views to change. Although we believe the expectations reflected in such forward-looking statements are reasonable, we cannot assure that such expectations will prove correct. These forward-looking statements should not be relied upon as representing our views as of any date after the date of this press release.

_____________________________________________

Zevra Contact

Nichol Ochsner
+1 (732) 754-2545
nochsner@zevra.com

Russo Partners Contact

David Schull
+1 (858) 717-2310
david.schull@russopartnersllc.com

FAQ

How much is Zevra Therapeutics (ZVRA) selling its Priority Review Voucher for?

Zevra Therapeutics is selling its Rare Pediatric Disease Priority Review Voucher for $150 million in gross proceeds.

When will ZVRA receive the $150M payment for the Priority Review Voucher?

The transaction is expected to close within 30-45 days, subject to customary closing conditions.

What will ZVRA use the $150M PRV sale proceeds for?

The proceeds will support commercial launches of MIPLYFFA and OLPRUVA, and advance their pipeline of rare disease product candidates.

Why did Zevra Therapeutics (ZVRA) receive the Priority Review Voucher?

ZVRA received the PRV in September 2024 following FDA approval of MIPLYFFA for treating Niemann-Pick disease type C.

What is MIPLYFFA, ZVRA's newly approved drug?

MIPLYFFA (arimoclomol) is a treatment for neurological manifestations of Niemann-Pick disease type C in patients 2 years and older.