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 (ZVRA) will present at three investor conferences in New York in September 2026, featuring fireside chats with its executive leadership team.
Events include the Cantor Global Healthcare Conference on September 9, the Morgan Stanley 24th Annual Global Healthcare Conference on September 14, and the H.C. Wainwright 28th Annual Global Investment Conference on September 15, with live webcasts available via Zevra’s investor relations website.
Zevra Therapeutics (NasdaqGS: ZVRA) will present two posters with new long-term real-world data on MIPLYFFA (arimoclomol) in Niemann-Pick disease type C (NPC) at the 2026 SSIEM Annual Symposium. U.S. expanded access program data showed durable disease stabilization for up to 4 years across children and adults, with NPC Clinical Severity Scale scores remaining essentially unchanged versus baseline and pivotal trial data indicating a 65% reduction in disease progression versus placebo over 12 months.
German compassionate use data from 48 patients (ages 2–63, mean exposure 3.0 years) showed no discontinuations due to arimoclomol-related side effects and reported rapid clinical deterioration in some patients after stopping treatment, supporting the importance of continued therapy. MIPLYFFA is FDA-approved in the U.S. for neurological manifestations of NPC in patients 2 years and older, used with miglustat, and has an EMA Marketing Authorization Application under review.
Zevra Therapeutics (NasdaqGS: ZVRA) granted stock options to purchase an aggregate 92,200 common shares to four new employees under its 2023 Employment Inducement Award Plan. The options vest over four years, starting with 25% after one year, in accordance with Nasdaq Rule 5635(c)(4).
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.