Zevra Therapeutics Stock (ZVRA) Great Growth and Undervalue in Q1 2025

Zevra Therapeutics (ZVRA) continues to build momentum in Q1 2025, demonstrating exceptional growth and signaling significant upside potential. Amid a surge in quarterly revenue up nearly 500% year‑over‑year and improved losses, analysts are rapidly raising earnings and price targets. In this article we will dive into Zevra Therapeutics recent earnings, stock performance & valuation, growth potential, and the risks investor should consider.

About Zevra Therapeutics

Founded in 2006 and based in Celebration, Florida, Zevra Therapeutics (formerly KemPharm) is a commercial-stage biopharmaceutical company focused on rare diseases. It operates with a data-driven strategy and currently markets two approved therapies: MIPLYFFA® for Niemann-Pick disease type C, approved in September 2024 following the Orphazyme acquisition, and OLPRUVA® for urea cycle disorders, acquired through Acer Therapeutics in 2023. Zevra is also advancing a Phase 3 trial for celiprolol in vascular Ehlers-Danlos syndrome, targeting sustainable growth in underserved markets with limited treatment options.

Zevra Therapeutics Financial Performance

Zevra Therapeutics delivered impressive financial results in Q1 2025, highlighting strong growth and improving fundamentals. Revenue for the quarter surged to $20.4 million, a substantial 495.65% increase compared to $3.43 million in Q1 2024, driven by early commercial success of its approved therapies. The company also significantly reduced its net loss, with earnings per share improving from –$0.40 in Q1 2024 to –$0.06 in Q1 2025, an 85% reduction. On a trailing twelve-month (TTM) basis, revenue grew 46.47% year-over-year, reaching $40.59 million, while TTM EPS declined to –$1.86 from –$1.33, reflecting a 39.85% increase in net loss primarily due to continued investments in commercialization and R&D. Despite a negative net profit margin of –226.64%, Zevra maintained a strong gross margin of 78.84%, showing strong product-level profitability. However, return on assets and equity remain deeply negative at –27.73% and –204.78%, respectively, indicating inefficiencies still being addressed. The company’s debt-to-equity ratio stands at 1.51, suggesting moderate leverage.

If we take a look at the recent quarterly financial performance, Zevra is operating at a significant net loss, but the recent Q1 2025 results suggest the potential for breakeven. This could be a turning point for Zevra Therapeutics toward becoming profitable in the future.

Zevra Therapeutics Fiscal 2025 Financial Forecast

Looking ahead, analysts forecast strong growth, projecting 2025 revenue to reach $93.17 million up 294.6% from $23.61 million in 2024 and EPS to swing to $1.09 from a loss of –$2.28 in 2024, a 147.81% improvement. Analysts have issued a “Strong Buy” rating, with an average price target of $21.71, implying 141.22% upside from current levels, and a high-end target of $25, representing a potential gain of 177.78%.

ZVRA Stock Price Performance and Valuation

At the time this article was written, Zevra stock was trading at $9 per share. Zevra Therapeutics' stock has delivered strong performance over the past year, with its share price doubling, marking a 100% increase that significantly outperformed the S&P 500’s 10.5% gain in the same period. However, over the past five years, the stock has risen 85.9%, slightly underperforming the S&P 500’s 100% increase. 

In terms of valuation, the stock currently trades at a price-to-sales (TTM) ratio of 10.99, reflecting the company’s early-stage commercial phase. More importantly, its forward-looking valuation appears attractive, with a forward price-to-sales ratio of 5.31 and a forward price-to-earnings ratio of just 8.28. These metrics suggest that the market has not yet fully priced in the company’s expected profitability and revenue surge in 2025.

Zevra Therapeutics Growth Potential

Zevra Therapeutics growth prospect remains strong, driven by several factors.

• Strong MIPLYFFA Performance
  MIPLYFFA, Zevra's flagship product for Niemann-Pick Disease Type C (NPC), has demonstrated exceptional commercial traction since its launch. The drug achieved 122 totalprescription enrollment forms by the end of Q1 2025, with 13 new enrollments during the quarter. Market access has reached 38% of covered lives, which is in line with expectations at this stage of launch.
  The success of MIPLYFFA is particularly significant as it represents the first FDA-approved treatment for NPC, positioning Zevra as the market leader in this rare disease space. Early adoption has exceeded company expectations, with U.S. sales reaching $17.1 million in Q1 2025, surpassing both analyst and consensus estimates. Analysts have revised their sales projections upward, with estimates for MIPLYFFA reaching $80 million for 2025 and $117 million for 2026.
• Market Opportunity and Competitive Position
  The NPC market presents substantial growth potential, with the global Niemann-Pick Disease Type C drugs market valued at USD 708.75 million in 2024 and projected to reach USD 2,024.61 million by 2032, representing a CAGR of 14.02%. The market is expected to exhibit a CAGR of 17.05% during 2025-2035 across the 7 major markets.
  Zevra's first-to-market advantage with MIPLYFFA provides significant competitive benefits in this rare disease space, potentially establishing the drug as the standard of care. The company is among the key sponsors in the NPC market alongside IntraBio Ltd., Cyclo Therapeutics Inc., and Azafaros.
• European Expansion
  Zevra Therapeutics is advancing its international growth with plans to file MIPLYFFA’s Marketing Authorization Application (MAA) with the European Medicines Agency in the second half of 2025, staying on schedule. The European Niemann-Pick Disease Type C (NPC) market is well-established, with existing treatments already in place, which could help speed up MIPLYFFA’s commercialization once approved. This established market familiarity among healthcare providers and patients is expected to support a smoother and faster adoption process.
  Additionally, Zevra continues to support patient access to MIPLYFFA through its global Expanded Access Program, helping address unmet medical needs in Niemann-Pick Disease Type C (NPC) while gathering valuable real-world insights. This initiative complements Zevra’s regulatory strategy and supports its commercial readiness. Combined with its planned MAA filing in Europe, these efforts position Zevra for a strong entry and potential growth in the European NPC market.

Risks to Consider

While Zevra Therapeutics presents a compelling investment case, we should be mindful of potential risks.

• Market Expansion Challenges in Europe
  Zevra intends to submit a regulatory application for MIPLYFFA in Europe but faces challenges such as navigating varied regulatory frameworks, setting up distribution networks, and adapting to diverse healthcare and reimbursement systems across European countries. These complexities may cause delays or complications in market entry, potentially hindering growth projections and limiting the company’s ability to establish a strong international presence in the European rare disease market.
• Regulatory and Clinical Trial Risks
  Upcoming regulatory decisions, including the FDA PDUFA date for Arimoclomol and the European marketing authorization for MIPLYFFA, represent critical binary events for Zevra. These milestones carry significant risk, as negative outcomes or delays could substantially impact the company’s future revenue streams and overall valuation. Successful approvals are essential for commercial growth, while setbacks may hinder market access and investor confidence, affecting Zevra’s financial stability and long-term prospects.
• Dependence on Rare Disease Market Dynamics
  Zevra’s focus on niche rare disease markets exposes it to volatility from small patient populations, uncertain reimbursement environments, and competitive pressures. The company’s success heavily depends on effective market education and accurate diagnosis rates, which directly affect product demand. These factors introduce variability and unpredictability into revenue forecasts, making financial outcomes more uncertain and challenging to project consistently in the specialized rare disease sector.

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Conclusion

Zevra Therapeutics presents a compelling investment opportunity backed by strong Q1 2025 performance, rapid revenue growth, and a clear path toward profitability. With two FDA-approved rare disease therapies now in early commercialization, a promising clinical pipeline, and robust analyst projections forecasting nearly $100 million in revenue and positive EPS by year-end, the company is well-positioned for continued success. Despite some financial risks typical of emerging biopharma firms, Zevra’s attractive valuation metrics, solid gross margins, and substantial upside potential make it a stock worth considering for investors seeking exposure to high-growth opportunities in the rare disease space.