Press Releases June 8, 2026 07:30 AM

Zevra Therapeutics Announces Orange Book Listing of U.S. Patent Strengthening Exclusivity for MIPLYFFA

Zevra Therapeutics secures FDA Orange Book patent listing for MIPLYFFA, extending market exclusivity through 2041

By Leila Farooq
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Zevra Therapeutics has successfully submitted a U.S. patent for its drug MIPLYFFA to the FDA's Orange Book, strengthening its intellectual property protection with exclusivity extending to 2041. This development solidifies Zevra's position in treating Niemann-Pick disease type C and enhances the long-term commercial value of MIPLYFFA. A patent term extension is also pending, potentially prolonging exclusivity beyond 2029.

Zevra Therapeutics Announces Orange Book Listing of U.S. Patent Strengthening Exclusivity for MIPLYFFA
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Key Points

  • Zevra submitted U.S. Patent No. 11,707,456 of MIPLYFFA for FDA Orange Book listing, with expiration in 2041.
  • MIPLYFFA treats Niemann-Pick disease type C, an ultra-rare neurodegenerative disease, addressing a significant unmet medical need.
  • Patent term extension application for another patent (expiring 2029) is pending, potentially lengthening market exclusivity.
  • Impacted sectors include biotechnology, pharmaceuticals, and the rare disease treatment market.

MIPLYFFA patent submitted for Orange Book listing, with patent protection to 2041

Separately filed patent term extension remains pending with the U.S. Patent and Trademark Office

BOSTON, June 08, 2026 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on bringing life-changing therapeutics to people living with rare diseases, today announced that it has submitted U.S. Patent No. 11,707,456 for MIPLYFFA® (arimoclomol) to the U.S. Food and Drug Administration for listing in the publication Approved Drug Products With Therapeutic Equivalence Evaluations, commonly known as the Orange Book. The patent expires on November 19, 2041.

“We are pleased to further strengthen our intellectual property protection for MIPLYFFA with the listing of this patent in the FDA’s Orange Book,” said Neil F. McFarlane, Zevra's President and Chief Executive Officer. “MIPLYFFA represents an important therapeutic option for people living with Niemann-Pick disease type C (NPC), an ultra-rare, progressive, and devastating neurodegenerative disease with significant unmet need. This listing reflects our continued focus on maintaining and enhancing the intellectual property portfolio supporting MIPLYFFA as we work to maximize its long-term value and support patients and families affected by NPC.”

The Orange Book lists patents and regulatory exclusivities associated with brand-name drugs, which dictates when generic competitors can legally enter the market.

The Company has also previously applied for a patent term extension for U.S. Patent No. 11,045,460 which currently expires in 2029 and is awaiting a determination from the U.S. Patent and Trademark Office.

About Zevra Therapeutics, Inc.

Zevra Therapeutics, Inc. is a commercial-stage company with a late-stage pipeline committed to redefining what is possible in bringing life-changing therapies to people living with rare diseases. The Company is focused on broadening access through geographic expansion opportunities, progressing its pipeline toward key milestones, and delivering meaningful therapeutics. The commercialization of its lead product, marketed in the U.S. for Niemann-Pick disease type C (NPC), a rare, progressive neurodegenerative disease, provides a strong corporate foundation and validates its ability to advance therapies from development to market. Zevra's vision is realized through disciplined execution of its strategic plan and core values — patient centricity, integrity, accountability, innovation, and courage — which guide its efforts to deliver long-term value.

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

Caution 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 are based on information currently available to Zevra and its current plans or expectations. They are subject to several known and unknown uncertainties, risks, 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. 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, 2025, filed with the Securities and Exchange Commission on March 9, 2026 and Zevra’s Quarterly Report on Form 10-Q for the quarter ended March 31, 2026, filed on May 6, 2026, 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.

Investor Contact

Nichol Ochsner 
+1 (732) 754-2545 
[email protected]  

Media Contact

Julie Downs
+1 (508) 246-3230 
[email protected]


Risks

  • Pending approval of patent term extension introduces uncertainty around the exact length of exclusivity.
  • Market adoption and commercial success of MIPLYFFA depend on effective patient access and competitive landscape.
  • Regulatory risks and potential future patent challenges could impact exclusivity and revenues.

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