ZOverview
Zevra Therapeutics develops and commercializes therapies for rare diseases, especially rare CNS disorders, by acquiring assets and pursuing in-house development. Its products include OLPRUVA for urea cycle disorders and azstarys, a once-daily ADHD treatment marketed in the U.S. with Corium for commercialization, plus a development pipeline like arimoclomol for Niemann-Pick disease type C. The company mixes internal R&D with strategic acquisitions and engages with regulators, patients, and advocacy groups to target underserved populations. Its goal is to build a differentiated portfolio of therapies that deliver sustainable value for patients and stakeholders.
Work here?Claim your company
About Zevra Therapeutics
Industries
Company Size
51-200
Company Stage
IPO
Headquarters
Boston, MA
Founded
2006
Get referred to Zevra Therapeutics
See people who can refer or advise you
Simplify's take
What believers are saying
- Q2 2026 revenue hit $39.7 million, up 53% year over year.
- Cash reached $260.2 million June 30, 2026, after debt repayment and asset sale.
- Q2 2026 prescription enrollment reached 184, with 69% covered-life access.
What critics are saying
- EMA rejected arimoclomol in 2026; re-examination can still fail again.
- MIPLYFFA drove $30.2 million of Q2 2026 revenue, creating single-product dependence.
- If Europe stays closed, Zevra loses its biggest near-term expansion pathway.
What makes Zevra Therapeutics unique
- MIPLYFFA won FDA approval September 20, 2024, for Niemann-Pick disease type C.
- Zevra owns orphan and Orange Book protection for MIPLYFFA through 2041.
- The 270-patient NPC dataset gives Zevra unmatched long-term real-world evidence.
Help us improve and share your feedback! Did you find this helpful?
Funding
Total Funding
$291.8M
Above
Industry Average
Funded Over
9 Rounds
Benefits
- Wellness Program
- Remote Work Options
Stock Price
Growth & Insights and Company News
6 month growth
1%1 year growth
5%2 year growth
-3%Zevra Therapeutics reported third-quarter earnings of $19.2 million on Tuesday, beating Wall Street expectations. The Boston-based specialty pharmaceutical company posted net income of 26 cents per share. Adjusted for non-recurring gains, earnings came to 18 cents per share, against an average analyst estimate of 14 cents. Revenue was $37.8 million, ahead of the $36.7 million three analysts surveyed by Zacks Investment Research had expected.
Zevra Therapeutics Q3 earnings call highlights. Zevra Therapeutics (NASDAQ:ZVRA) reported third-quarter 2026 net revenue of $37.8 million, up 45% from $26.1 million a year earlier, driven primarily by U.S. sales of MIPLYFFA for Niemann-Pick disease type C, or NPC. Net U.S. product revenue from MIPLYFFA reached $34 million in the quarter, rising 12.6% sequentially and 52% from the prior-year period, according to Chief Financial Officer Justin Renz. The company also reported $3.6 million in net reimbursements from its global expanded-access program and $0.2 million from OLPRUVA. Zevra recorded net income of $19.2 million, or $0.26 per diluted share, compared with a net loss of $0.5 million, or $0.01 per share, in the third quarter of 2025. The results included a $5.5 million favorable non-cash mark-to-market credit related to outstanding common-stock warrants. MIPLYFFA launch and patient identification. President and Chief Executive Officer Neil McFarlane said MIPLYFFA, the first approved therapy for NPC, received 13 prescription enrollment forms during the third quarter, bringing cumulative enrollment forms since launch to 197. The company estimates that about 900 people in the United States may be living with NPC, although only 300 to 350 had historically been diagnosed based on a 2021 study. Management said it is increasingly seeing newly diagnosed patients and believes the addressable diagnosed population may be larger than its earlier estimates. Chief Commercial Officer Josh Schafer said third-quarter prescriptions included a mix of adult and pediatric patients, as well as a meaningful proportion of newly diagnosed patients. "We're seeing a pretty stable number of new patients coming in every quarter," Schafer said. He added that analysis of medical claims data, along with increased disease awareness, suggests more patients may have NPC than previously estimated. The company said it has modestly expanded its customer-facing commercial team and broadened outreach to community practices beyond centers of excellence. Schafer said the flexible commercial model is intended to allow Zevra to scale its field organization as it gains more information about the market. Zevra reported that payer coverage for MIPLYFFA is currently 69%, while most remaining patients receive coverage through medical-exception pathways. The company said it continues to see high patient retention and refill rates. Its disease-awareness efforts included the Learn NPC, Read Between the Signs campaign and collaborations with genetic-testing providers. Visits to the company's disease-awareness website increased 123% sequentially, to nearly 56,000 in the third quarter from 25,000 in the second quarter, aided by the launch of an NPC Knowledge Hub for patients and caregivers. Potential pediatric label expansion. Zevra is pursuing a potential supplemental new drug application to expand the MIPLYFFA label to include infants younger than 24 months. McFarlane said the company held a Type B meeting with the FDA after publishing data from an open-label pediatric substudy of arimoclomol used with miglustat in infants ages six months to less than 24 months. The substudy evaluated safety and tolerability for up to 36 months and found arimoclomol was well tolerated, with no new safety signals observed, the company said. Based on FDA feedback, Zevra expects to file the sNDA in the first quarter of 2027. Management said the younger-than-24-month population represents a relatively small number of additional patients - estimated at roughly 5% of the total addressable market - but viewed the potential expansion as meaningful for broadening access. The company also highlighted MIPLYFFA's intellectual-property position. McFarlane said patents listed in the FDA's Orange Book extend to 2041, while U.S. orphan-drug exclusivity for NPC extends through 2031. Zevra is also pursuing a potential patent-term extension and now expects a decision from the U.S. Patent and Trademark Office in 2027. International access and European review. Zevra's global expanded-access program for arimoclomol enrolled 134 patients through compassionate-use and named-patient reimbursement programs. The company said it added an additional territory during the third quarter and continues to assess further opportunities to expand access internationally. In Europe, the re-examination of Zevra's marketing authorization application for arimoclomol is underway at the Committee for Medicinal Products for Human Use. A new rapporteur and co-rapporteur have been appointed, and an ad hoc expert group is expected to review the data. Zevra expects the CHMP's opinion in the fourth quarter. Celiprolol program and financial position. Zevra is also developing celiprolol for vascular Ehlers-Danlos syndrome, or vEDS. The Phase III DiSCOVER trial enrolled six patients during the quarter, reaching 72 total participants. The study's interim analysis is triggered at 28 events; four confirmed events had occurred through the end of the third quarter. The company held a second Type C meeting with the FDA regarding a possible submission strategy incorporating retrospective real-world data sets. Zevra is developing a statistical analysis plan and intends to seek a follow-up discussion with the agency in the first half of 2027, pending formal meeting minutes. Operating expenses were $24.1 million in the third quarter, up $3.7 million from a year earlier. Research and development expense increased to $4.9 million, reflecting investments in arimoclomol and celiprolol, while selling, general and administrative expense rose to $19.1 million due to commercial and infrastructure costs. As of Sept. 30, Zevra had $270.9 million in cash equivalents and investments, up approximately $10.7 million from June 30. Renz said the company does not anticipate meaningful increases in marketing spending next year following its modest commercial expansion. About Zevra Therapeutics (NASDAQ:ZVRA). Zevra Therapeutics, Inc is a biopharmaceutical company focused on developing and commercializing treatments for rare diseases and other conditions with significant unmet medical needs. The company combines traditional drug development with data science and patient-focused research to advance therapies for diseases that may have limited treatment options. Zevra's lead commercial product is MIPLYFFA (arimoclomol), which is approved in the United States for the treatment of neurological manifestations of Niemann-Pick disease type C in adults and children aged 2 years and older, in combination with miglustat.
Globeflex Capital L P invests $2.75 million in Zevra Therapeutics, Inc. $ZVRA. August 24, 2026 Key points. * Globeflex Capital initiated a $2.75 million position in Zevra Therapeutics, purchasing 191,904 shares, while institutional investors collectively own 35.03% of the company. * Zevra reported quarterly earnings of $0.25 per share, beating estimates of $0.11, and revenue of $39.66 million versus the $33.88 million consensus forecast. * Analysts maintain a favorable outlook, with nine Buy ratings and one Hold; the consensus rating is "Moderate Buy" with an average price target of $27.14, compared with the stock's reported $11.87 price. * Five stocks we like better than Zevra Therapeutics. Globeflex Capital L P bought a new position in shares of Zevra Therapeutics, Inc. (NASDAQ:ZVRA - Free Report) in the second quarter, according to the company in its most recent 13F filing with the Securities and Exchange Commission (SEC). The institutional investor bought 191,904 shares of the company's stock, valued at approximately $2,752,000. Globeflex Capital L P owned about 0.32% of Zevra Therapeutics as of its most recent filing with the Securities and Exchange Commission (SEC). Other institutional investors also recently bought and sold shares of the company. AIGH Capital Management LLC bought a new position in shares of Zevra Therapeutics during the 1st quarter valued at about $11,184,000. Dimensional Fund Advisors LP increased its holdings in shares of Zevra Therapeutics by 259.2% in the first quarter. Dimensional Fund Advisors LP now owns 554,802 shares of the company's stock worth $5,168,000 after purchasing an additional 400,336 shares during the last quarter. Hennion & Walsh Asset Management Inc. increased its holdings in shares of Zevra Therapeutics by 116.6% in the second quarter. Hennion & Walsh Asset Management Inc. now owns 383,706 shares of the company's stock worth $5,502,000 after purchasing an additional 206,543 shares during the last quarter. UBS Group AG lifted its position in shares of Zevra Therapeutics by 30.9% in the fourth quarter. UBS Group AG now owns 285,488 shares of the company's stock worth $2,558,000 after buying an additional 67,392 shares in the last quarter. Finally, Janney Montgomery Scott LLC lifted its position in shares of Zevra Therapeutics by 16.7% in the first quarter. Janney Montgomery Scott LLC now owns 236,834 shares of the company's stock worth $2,207,000 after buying an additional 33,951 shares in the last quarter. 35.03% of the stock is owned by hedge funds and other institutional investors. Zevra Therapeutics stock performance. Shares of Zevra Therapeutics stock opened at $11.87 on Monday. The stock has a market cap of $704.84 million, a price-to-earnings ratio of 12.90 and a beta of 0.91. The stock has a 50 day moving average price of $12.23 and a 200-day moving average price of $10.77. Zevra Therapeutics, Inc. has a one year low of $7.16 and a one year high of $15.03. Zevra Therapeutics (NASDAQ:ZVRA - Get Free Report) last released its quarterly earnings results on Wednesday, August 5th. The company reported $0.25 earnings per share for the quarter, beating the consensus estimate of $0.11 by $0.14. The company had revenue of $39.66 million for the quarter, compared to analyst estimates of $33.88 million. Zevra Therapeutics had a return on equity of 54.54% and a net margin of 42.82%. Sell-side analysts predict that Zevra Therapeutics, Inc. will post 0.72 earnings per share for the current fiscal year. Insiders place their bets. In other news, Director Corey Michael Watton sold 12,300 shares of the stock in a transaction on Monday, August 10th. The shares were sold at an average price of $11.25, for a total value of $138,375.00. Following the sale, the director owned 1,800 shares of the company's stock, valued at approximately $20,250. This represents a 87.23% decrease in their ownership of the stock. The sale was disclosed in a legal filing with the SEC, which can be accessed through this link. 3.70% of the stock is owned by company insiders. Analyst upgrades and downgrades. Several equities analysts have weighed in on ZVRA shares. Citizens Jmp raised their price target on Zevra Therapeutics from $25.00 to $28.00 and gave the company a "market outperform" rating in a report on Thursday, August 6th. Canaccord Genuity Group decreased their price objective on Zevra Therapeutics from $29.00 to $20.00 and set a "buy" rating on the stock in a report on Monday, July 27th. Maxim Group increased their target price on Zevra Therapeutics from $25.00 to $35.00 and gave the company a "buy" rating in a research report on Wednesday, June 10th. Citigroup reissued an "outperform" rating on shares of Zevra Therapeutics in a report on Thursday, August 6th. Finally, Guggenheim decreased their price target on shares of Zevra Therapeutics from $24.00 to $23.00 and set a "buy" rating on the stock in a research note on Friday, August 7th. Nine analysts have rated the stock with a Buy rating and one has assigned a Hold rating to the company. Based on data from MarketBeat, the company has a consensus rating of "Moderate Buy" and an average price target of $27.14. Discover more Space Stocks Report Dividend Screener Tool About Zevra Therapeutics. Zevra Therapeutics, Inc discovers and develops various proprietary prodrugs to treat serious medical conditions in the United States. The company develops its products through Ligand Activated Therapy platform. Its lead product candidate is KP1077, consisting of KP1077IH, which is under Phase 2 clinical trial for the treatment of idiopathic hypersomnia, and KP1077N, which is under Phase 1/2 clinical trial to treat narcolepsy. The company is also developing Celiprolol, a prodrug product candidate that is under Phase 1/2 clinical trial for the treatment of vascular Ehlers Danlos syndrome. This instant news alert was generated by narrative science technology and financial data from MarketBeat in order to provide readers with the fastest reporting and unbiased coverage. Please send any questions or comments about this story to [email protected]. Continue following MarketBeat Before you consider Zevra Therapeutics, you'll want to hear this. MarketBeat keeps track of Wall Street's top-rated and best performing research analysts and the stocks they recommend to their clients on a daily basis. MarketBeat has identified the five stocks that top analysts are quietly whispering to their clients to buy now before the broader market catches on... and Zevra Therapeutics wasn't on the list. While Zevra Therapeutics currently has a Moderate Buy rating among analysts, top-rated analysts believe these five stocks are better buys. The AI boom is creating opportunities across semiconductors, cloud computing, enterprise software, infrastructure, cybersecurity, and automation. Inside this report, you'll find 10 companies positioned to benefit as artificial intelligence moves from hype to real-world deployment and becomes a core growth driver for corporate America.
Zevra Therapeutics published long-term real-world safety and effectiveness data for arimoclomol in adults with Niemann-Pick disease type C. This marks the first extensive evaluation of arimoclomol outside clinical trials. The company is pursuing regulatory options in Europe following a negative opinion from the European Medicines Agency on arimoclomol. Zevra has requested a re-examination of its Marketing Authorization Application. The stock has been volatile, gaining 23.2% over the past week but declining 17.7% over the past month. Year-to-date returns stand at 42.4%, with the stock last closing at $11.88. Latest quarterly results showed net income of $8.75 million on sales of $39.66 million. The European regulatory review adds timing risk and uncertainty for investors.
Zevra Therapeutics reported total net revenue of $39.7 million in Q2 2026, marking a 53% increase year-over-year. The company's US sales of MIPLYFFA reached $30.2 million, up 23% sequentially and 40% year-over-year. Operating expenses decreased to $21.0 million, down $3.2 million from the previous year. The company posted net income of $8.8 million, or $0.14 per share. Cash and investments stood at $260.2 million as of 30 June 2026, up $23.4 million from the previous quarter. Since launch, MIPLYFFA has received 184 prescription enrolment forms, with 14 added in Q2. Zevra strengthened its intellectual property position for MIPLYFFA with Orange Book protection through 2041. The company continues advancing its pipeline, including the Phase III DISCOVER trial for celiprolol.
Zevra Therapeutics is Hiring for 8 Jobs on Simplify!
Find jobs on Simplify and start your career today
Don't see your dream role? Check out thousands of other roles on Simplify.Browse all jobs
About Zevra Therapeutics
Industries
Company Size
51-200
Company Stage
IPO
Headquarters
Boston, MA
Founded
2006
Zevra Therapeutics is Hiring for 8 Jobs on Simplify!
Find jobs on Simplify and start your career today
Don't see your dream role? Check out thousands of other roles on Simplify.Browse all jobs