Pharming's Joenja® sNDA Rejected by FDA
Written by Emily J. Thompson, Senior Investment Analyst
Updated: 1h ago
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Source: Newsfilter
- FDA Response: The FDA issued a Complete Response Letter (CRL) to Pharming's supplemental New Drug Application (sNDA) for Joenja® (leniolisib), citing concerns about potential underexposure in lower-weight pediatric patients, which impacts the drug's market entry for children aged 4 to 11 years.
- Production Method Issues: The FDA also identified issues with one of the analytical methods used in production batch testing, requesting additional data and clarification, which not only delays the approval process but may also affect Pharming's competitive position in the pediatric immunodeficiency treatment market.
- Clinical Data Support: Despite these challenges, Pharming plans to work closely with the FDA to address the clinical pharmacology and batch testing methodology issues, intending to request a Type A meeting with the FDA to determine the best path forward for resubmission, demonstrating the company's commitment to pediatric patients with APDS.
- Market Outlook: Joenja's FDA approval for patients aged 12 and older remains unaffected by this regulatory action, and with no approved treatments for children under 12 with APDS globally, the drug still holds potential growth opportunities in the future market.
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Analyst Views on PHAR
Wall Street analysts forecast PHAR stock price to rise over the next 12 months. According to Wall Street analysts, the average 1-year price target for PHAR is 39.50 USD with a low forecast of 37.00 USD and a high forecast of 42.00 USD. However, analyst price targets are subjective and often lag stock prices, so investors should focus on the objective reasons behind analyst rating changes, which better reflect the company's fundamentals.
2 Analyst Rating
2 Buy
0 Hold
0 Sell
Moderate Buy
Current: 20.690
Low
37.00
Averages
39.50
High
42.00
Current: 20.690
Low
37.00
Averages
39.50
High
42.00
About PHAR
Pharming Group NV is a biopharmaceutical company based in the Netherlands. The Company is engaged in the development, production and commercialization of human therapeutic proteins to be used in the therapies. The Company's product portfolio is aimed at treatments for genetic disorders, blood-related disorders, infectious and inflammatory diseases, tissue and bone damage and surgical/traumatic bleeding. Its core product, RUCONEST (conestat alfa) is a recombinant human C1 esterase inhibitor approved for the treatment of acute Hereditary Angioedema (HAE) attacks in patients in Europe, the Unites States, Israel and South Korea. The product is available on a named-patient basis in other territories where it has not yet obtained marketing authorization.
About the author
Emily J. Thompson
Emily J. Thompson, a Chartered Financial Analyst (CFA) with 12 years in investment research, graduated with honors from the Wharton School. Specializing in industrial and technology stocks, she provides in-depth analysis for Intellectia’s earnings and market brief reports.
Pharming's Joenja® sNDA Rejected by FDA
- FDA Response: The FDA issued a Complete Response Letter (CRL) to Pharming's supplemental New Drug Application (sNDA) for Joenja® (leniolisib), citing concerns about potential underexposure in lower-weight pediatric patients, which impacts the drug's market entry for children aged 4 to 11 years.
- Production Method Issues: The FDA also identified issues with one of the analytical methods used in production batch testing, requesting additional data and clarification, which not only delays the approval process but may also affect Pharming's competitive position in the pediatric immunodeficiency treatment market.
- Clinical Data Support: Despite these challenges, Pharming plans to work closely with the FDA to address the clinical pharmacology and batch testing methodology issues, intending to request a Type A meeting with the FDA to determine the best path forward for resubmission, demonstrating the company's commitment to pediatric patients with APDS.
- Market Outlook: Joenja's FDA approval for patients aged 12 and older remains unaffected by this regulatory action, and with no approved treatments for children under 12 with APDS globally, the drug still holds potential growth opportunities in the future market.
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Pharming Reports $376M Preliminary 2025 Revenue, Exceeding Guidance by 27%
- Significant Revenue Growth: Pharming anticipates approximately $376 million in revenue for 2025, exceeding the revised guidance of $365-$375 million by 27%, reflecting strong market demand for RUCONEST and Joenja.
- Strong RUCONEST Performance: RUCONEST generated $231.2 million in revenue during the first nine months of 2025, representing a 34% increase from the previous year, which enhances the company's financial stability in a competitive hereditary angioedema market.
- Rising Demand for Joenja: Joenja contributed $38.4 million in revenue, up 20% year-over-year, indicating successful new drug promotion and increasing acceptance in the U.S. market, which is expected to further drive future revenue growth.
- Positive Future Outlook: Pharming expects continued revenue growth in 2026 and plans to provide detailed updates on its clinical pipeline and financial guidance during the Investor Day on February 3, 2026, demonstrating the company's commitment to long-term value creation.
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