Arimoclomol

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 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 nochsner@zevra.com Media Contact Julie Downs +1 (508) 246-3230 jdowns@zevra.com

Zevra’s efforts to boost U.S. NPC diagnosis rates are starting to pay off, McFarlane said As its ultra-rare disease med Miplyffa strengthens its foothold in the U.S., Zevra Therapeutics’ CEO believes the launch has the potential to convert the American treatment landscape for Niemann-Pick disease type C (NPC) into one resembling Europe’s, where most patients are diagnosed and Johnson & Johnson’s enzyme inhibitor Zavesca (miglustat) has been present as a treatment option since 2009. Last month, Zevra reported that Miplyffa—greenlit by the FDA alongside Zavesca to treat NPC in the fall of 2024—rounded up quarterly net revenues of $24.6 million. For the full year prior, Miplyffa lassoed $87.4 million, contributing to a companywide 2025 revenue haul of $106.5 million.In the first three months of 2026, Zevra received nine new Miplyffa prescription enrollment forms for NPC, bringing its total tally to 170 since launch. With multiple commercial products now on deck and another late-stage program in the hopper, Zevra is well positioned to thrive in the rare disease ecosystem over the long run, Neil McFarlane, the company’s CEO, said in a recent interview with Fierce Pharma. Much of the company’s current focus naturally revolves around Miplyffa, with McFarlane stressing that Zevra is leaning heavily on the roughly 40 centers of excellence, including teaching institutions and children’s hospitals, that it’s leveraging to help patients with NPC receive diagnoses and treatment.Working in close conjunction with multidisciplinary specialists at those centers is paramount to a successful product rollout in the rare disease space, the CEO said.“In ultra-rare disease, nine times out of ten, a patient goes through a very challenging diagnostic odyssey, but they end up at one of those centers of excellence,” McFarlane explained. He continued, “The alignment of all those parts that come together is key, and that’s kind of what we put together here—the opportunity to be a partner of choice in the rare disease community from each one of those touchpoints.” With the FDA nod for Miplyffa in late 2024, patients in the U.S. received the first purpose-approved therapy to treat neurological manifestations of NPC, which prevents the body from moving and using cholesterol and other lipid cells. The condition causes a buildup of cholesterol and other lipids in the liver, spleen or lungs, leading to issues with speech, cognition, swallowing, movement and fine motor skills.  Digging into the product’s launch, McFarlane reiterated Zevra’s estimate that there are some 900 addressable NPC patients in the U.S., with somewhere around 300 to 350 of those patients presently identified and diagnosed. In the initial stage of Miplyffa’s rollout, Zevra focused on swiftly ensuring supply for patients in its expanded access program and those who had taken part in clinical trials, among other groups, McFarlane said. Now, the company is focusing on reaching other diagnosed patients by spreading the word about Miplyffa’s profile as a new disease-modifying agent, while attempting to increase remaining patient diagnoses in parallel. Zevra’s efforts to boost diagnosis rates are starting to pay off, McFarlane said, adding that the trends in recent quarters have “given us a lot more confidence” in the company’s ability to build out the U.S. NPC market. “Q2 we saw some newly diagnosed patients through our efforts,” he said. “Q3, we saw some more. Q4 we saw a lot more, and then also in Q1.“That is how we believe that we’re going to be able to build a market from the three hundred to three-fifty to the nine hundred,” the CEO said, adding that Zevra hopes to engender a U.S. NPC market that more closely resembles Europe’s, where there are some 1,100 patients, most of whom have been diagnosed. European patients are “not in the diagnostic odyssey: They’ve been to a reference center, but it’s also on the back of miglustat, that’s been approved there for well over a decade,” McFarlane said, referring to Johnson & Johnson’s oral med Zavesca. Now, as Zevra aims to bring Miplyffa online in Europe as well, where the drug is undergoing a review by the European Medicines Agency, the company’s “go-to-market strategy is still open,” McFarlane admitted. Zevra will need to await a regulatory declaration to know where it stands on the pricing front, which will drive its decision to pursue the launch itself through some form of “hybrid” model or “outsource this to a multinational,” the CEO explained. That said, the company’s global expanded access program for Miplyffa has already garnered some 122 patients, so demand outside of the U.S. exists, McFarlane figures.  Reflecting on the broader rare disease landscape, connections and patient empowerment remain essential to Zevra’s efforts, McFarlane noted. “A lot of times, rare diseases—and inclusive of some ones that we work with—they’re started by a patient advocacy group with someone who has a disease that’s never been characterized, that is then a product or medicines developed, and then you work through the development and the regulatory approval, and then you get to the launch and the commercialization, payers, physician education, all that—but it’s a full circle,” McFarlane said, stressing that “you have to start with advocacy in this space.”