Welcome to our dedicated page for Zevra Therapeutics news (Ticker: ZVRA), a resource for investors and traders seeking the latest updates and insights on Zevra Therapeutics stock.
Zevra Therapeutics reports developments as a commercial-stage biopharmaceutical company focused on therapies for rare diseases. Recurring updates center on MIPLYFFA (arimoclomol), its U.S.-approved treatment for Niemann-Pick disease type C, including commercialization, patient access, regulatory activity and diagnostic-support initiatives tied to NPC.
Zevra news also covers financial results, pipeline programs in rare diseases such as vascular Ehlers-Danlos syndrome, and corporate actions that shape its portfolio. Recent company updates include the completed sale of the serdexmethylphenidate, or SDX, portfolio, including AZSTARYS and KP1077, as well as governance and executive-compensation announcements under Nasdaq rules.
Zevra Therapeutics (NasdaqGS: ZVRA) reported Q2 2026 net revenue of $39.7 million, up 53% from Q2 2025, driven by $30.2 million from MIPLYFFA, $0.2 million from OLPRUVA, $9.0 million in global Expanded Access Program reimbursements and $0.3 million in AZSTARYS-related royalties and reimbursements.
Cost of product revenue was $1.5 million and operating expenses were $21.0 million. Zevra reported net income of $8.8 million, or $0.14 per share; adjusted net income was estimated at $15.2 million, or $0.25 per diluted share. Cash, cash equivalents and investments totaled $260.2 million.
MIPLYFFA progress included 14 new U.S. enrollment forms in Q2 (184 total since launch), 132 patients in the global EAP, extended Orange Book IP to 2041 and new safety/effectiveness publications. The Phase 3 DiSCOVER trial of celiprolol in VEDS reached 66 enrolled patients with three confirmed events.
Zevra Therapeutics (NasdaqGS: ZVRA) will present at the Canaccord Genuity 46th Annual Growth Conference in Boston, MA, on Tuesday, August 11, 2026, at 11:00 a.m. ET. Management will also hold one-on-one meetings with registered attendees, and a live webcast will be available via the Investor Relations "Events & Presentations" page on Zevra’s website.
Zevra Therapeutics (NasdaqGS: ZVRA) reported publication of four-year real-world data from the U.S. Early Access Program evaluating MIPLYFFA (arimoclomol) in Niemann-Pick disease type C (NPC) in Molecular Genetics and Metabolism. The longitudinal study followed 109 participants across 14 U.S. sites, including 53 adults, with mean arimoclomol exposure of 820 days.
Disease severity, measured by the 5-domain and rescored 4-domain NPC Clinical Severity Scales, remained relatively stable over time, and safety findings were consistent with MIPLYFFA’s established profile. The data add to more than five years of global clinical experience in over 270 NPC patients.
Zevra Therapeutics (NasdaqGS: ZVRA) reported that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has adopted a negative opinion on its Marketing Authorization Application for arimoclomol to treat Niemann-Pick disease type C (NPC) in the European Union.
According to the company, Zevra plans to request a re-examination of the CHMP opinion and submit a detailed justification. Zevra continues to provide arimoclomol to eligible patients via its global Expanded Access Program, which also generates real-world evidence. The company notes that updated international NPC management guidelines highlight arimoclomol as an important treatment option.
Zevra Therapeutics (NasdaqGS: ZVRA) will release its Q2 2026 corporate and financial results on Wednesday, August 5, 2026, via news release after market close, followed by a conference call and audio webcast at 4:30 p.m. ET.
According to Zevra, the live webcast and a 90-day replay will be available on the “Events & Presentations” page in the Investor Relations section of investors.zevra.com. Investors can also join via telephone using U.S. dial-in (800) 274-8461, international dial-in +1 (203) 518-9814, and Conference ID ZVRAQ226.
Zevra Therapeutics (Nasdaq: ZVRA) reported positive data for MIPLYFFA (arimoclomol) from a phase 2/3 open-label pediatric substudy in infants with Niemann-Pick disease type C.
The 5‑patient study (ages 12–<24 months) found arimoclomol, added to miglustat, was generally well tolerated with no new safety signals and pharmacokinetics consistent with older pediatric groups.
Zevra Therapeutics (NasdaqGS: ZVRA) announced that Chief Medical Officer Adrian Quartel will step down for personal reasons effective July 3, 2026. He will remain a consultant through December 31, 2026 while Zevra searches for a new CMO and manages the transition.
Zevra reports it remains on track with key regulatory milestones, including an EMA review of its arimoclomol MAA for Niemann-Pick disease type C and an ongoing Phase 3 celiprolol trial in Vascular Ehlers-Danlos Syndrome, with further FDA discussions planned in the second half of 2026.
Zevra Therapeutics (NasdaqGS: ZVRA) submitted U.S. Patent No. 11,707,456 for MIPLYFFA (arimoclomol) to the FDA for Orange Book listing. The patent expires November 19, 2041, aiming to strengthen exclusivity for treating Niemann-Pick disease type C.
A separate patent term extension request for U.S. Patent No. 11,045,460, currently expiring in 2029, remains pending with the U.S. Patent and Trademark Office.
Zevra Therapeutics (NasdaqGS: ZVRA) granted stock options to four new employees under its 2023 Employment Inducement Award Plan, consistent with Nasdaq Rule 5635(c)(4).
The options cover an aggregate of 33,000 shares, vesting over four years with 25% after one year and the balance in three equal annual installments.
Zevra Therapeutics (NasdaqGS: ZVRA) will present at the H.C. Wainwright BioConnect Investor Conference in New York on May 19, 2026, at 9:00 a.m. ET.
Executive leadership will present and hold one-on-one meetings, with a live webcast available via the Investor Relations section of Zevra’s website.