FDA Declines Joenja Pediatric Approval for APDS Treatment
Written by Emily J. Thompson, Senior Investment Analyst
Updated: 6d ago
0mins
Should l Buy PHAR?
Source: NASDAQ.COM
- FDA Rejection: Pharming Group's Joenja drug for treating activated phosphoinositide 3-kinase delta syndrome (APDS) in children aged 4 to 11 has been declined by the FDA, despite existing approval for patients aged 12 and older, indicating regulatory hurdles that may hinder market expansion.
- Safety Concerns: The FDA's complete response letter raised issues regarding potential underexposure in lower-weight pediatric patients, requesting additional pediatric pharmacokinetic data to reassess dosing, which could delay market entry and impact revenue forecasts for the company.
- Clinical Response Strategy: Pharming Group intends to work closely with the FDA to address the clinical pharmacology and batch-testing methodology issues outlined in the letter, demonstrating confidence in the resubmission process that may expedite future approval timelines.
- Market Performance: Since its U.S. launch in 2023, Joenja generated $15.1 million in revenue in Q3 2025, reflecting a 35% increase year-over-year, indicating strong demand in the adult and adolescent markets, although the rejection of the pediatric indication may pose challenges for future growth.
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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: 16.040
Low
37.00
Averages
39.50
High
42.00
Current: 16.040
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 Group Under Investigation for Securities Fraud Claims
- FDA Response Letter: On February 1, 2026, Pharming received a Complete Response Letter from the FDA requesting additional pediatric pharmacokinetic data to confirm drug exposure levels in lower weight children, potentially delaying the approval of Joenja® and impacting future revenues.
- Significant Stock Drop: Following the FDA announcement, Pharming's American Depositary Receipt (ADR) price fell by $3.495, or 17.07%, to close at $16.975 per ADR on February 2, 2026, reflecting market concerns about the company's future prospects.
- Legal Investigation Initiated: Pomerantz LLP is investigating whether Pharming and its executives engaged in securities fraud or other unlawful business practices, which could lead to investor losses and further exacerbate market skepticism regarding the company's governance.
- Historical Context: Pomerantz LLP, recognized as a leading firm in securities class action litigation, has recovered millions for victims, indicating that this investigation could trigger broader legal actions that may affect Pharming's reputation and shareholder confidence.
See More
Pharming Announces 2026 Financial Guidance and Investor Day
- Financial Guidance Outlook: Pharming expects total revenues for 2026 to range between $405 million and $425 million, reflecting an 8% to 13% growth rate, indicating strong ongoing growth in its rare disease commercial products and bolstering investor confidence in future developments.
- Increased R&D Spending: The total operating expenses are projected to be between $330 million and $335 million in 2026, primarily driven by increased R&D expenditures related to ongoing clinical trials for leniolisib and napazimone (KL1333), showcasing the company's commitment to innovative drug development.
- Pipeline Progress: Pharming is advancing two major clinical-stage programs targeting primary immunodeficiencies with immune dysregulation and mtDNA-driven mitochondrial disease, with top-line data from both trials expected in the second half of 2026, potentially opening new market opportunities for the company.
- Investor Day Event: Today's Investor Day will focus on Pharming's scientific strategy and clinical plans, expected to attract attention from industry experts and further enhance the company's market position and brand influence in the rare disease sector.
See More
Pharming Group Announces 2026 Financial Guidance and Investor Day Highlights
- Financial Outlook: Pharming anticipates total revenues between $405 million and $425 million for 2026, reflecting an 8% to 13% growth driven by robust performance from Joenja® and RUCONEST®, indicating strong market expansion potential in rare diseases.
- R&D Investment: The company expects total operating expenses to range from $330 million to $335 million in 2026, primarily due to increased R&D costs associated with ongoing clinical trials for leniolisib and napazimone (KL1333), underscoring its commitment to innovative drug development.
- Pipeline Progress: Pharming is advancing two key programs, leniolisib for primary immunodeficiencies with immune dysregulation and napazimone (KL1333) for mtDNA-driven mitochondrial disease, both of which aim to address significant unmet medical needs and enhance the company's competitive position in the market.
- Investor Day Event: The company will showcase the scientific rationale and clinical strategy behind its pipeline at today's Investor Day, which is expected to attract investor interest and further solidify its market position in the rare disease sector.
See More
FDA Issues Complete Response Letter to Pharming for Joenja Pediatric Application
- Regulatory Impact: The FDA issued a Complete Response Letter to Pharming Group regarding its supplemental New Drug Application for Joenja (leniolisib) in children aged 4 to 11, resulting in a 16.76% drop in premarket trading to $17.03, indicating negative market sentiment towards the news.
- Dosage Concerns: The FDA raised issues about potential underexposure in lower-weight pediatric patients, requesting additional pediatric pharmacokinetic data to reassess proposed doses, which could significantly impact the drug's market potential if not resolved.
- Testing Method Deficiencies: The FDA identified issues with one of the analytical methods used for production batch testing, asking Pharming for further data and clarification, which may delay the drug's market entry and increase R&D costs.
- Meeting Plans and Market Response: Pharming intends to request a Type A meeting with the FDA; although Joenja was approved for patients aged 12 and older in March 2023, this regulatory action could hinder its promotional strategy in the pediatric market, affecting future revenue expectations.
See More
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- Disappointing Delivery Numbers: Xpeng Inc reported delivering 20,011 vehicles in January 2026, a figure that, while substantial, fell short of market expectations, leading to a decline in investor confidence.
- Stock Price Reaction: Following the disappointing delivery results, Xpeng's stock dropped 5.6% in pre-market trading to $16.97, reflecting concerns about the company's future performance among investors.
- Overall Market Trend: U.S. stock futures were lower, with Dow futures falling around 100 points, indicating a cautious market sentiment that could further impact investor attitudes towards Xpeng.
- Industry Impact: The delivery figures from Xpeng may negatively affect overall confidence in the electric vehicle market, especially in an increasingly competitive landscape, prompting investors to reassess their strategies.
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- Significant Revenue Growth: DarkIris Inc reported a year-over-year revenue increase of 27.3% for FY25, reaching $10.08 million compared to $7.92 million in FY24, indicating strong market performance and growth potential.
- Stock Price Surge: Following the positive earnings report, DarkIris shares jumped 104.2% in pre-market trading to $0.68, reflecting investor optimism about the company's future prospects.
- Positive Market Reaction: The release of this earnings report has significantly increased market attention on DarkIris, enhancing investor confidence in its continued growth and potentially attracting more capital inflow.
- Optimistic Industry Outlook: The robust performance of DarkIris not only strengthens its market position but may also provide more opportunities for future financing and expansion plans, further solidifying its competitive edge in the industry.
See More
Pharming Group Under Investigation for Securities Fraud Claims
- FDA Response Letter: On February 1, 2026, Pharming received a Complete Response Letter from the FDA requesting additional pediatric pharmacokinetic data to confirm drug exposure levels in lower weight children, potentially delaying the approval of Joenja® and impacting future revenues.
- Significant Stock Drop: Following the FDA announcement, Pharming's American Depositary Receipt (ADR) price fell by $3.495, or 17.07%, to close at $16.975 per ADR on February 2, 2026, reflecting market concerns about the company's future prospects.
- Legal Investigation Initiated: Pomerantz LLP is investigating whether Pharming and its executives engaged in securities fraud or other unlawful business practices, which could lead to investor losses and further exacerbate market skepticism regarding the company's governance.
- Historical Context: Pomerantz LLP, recognized as a leading firm in securities class action litigation, has recovered millions for victims, indicating that this investigation could trigger broader legal actions that may affect Pharming's reputation and shareholder confidence.
See More
Pharming Announces 2026 Financial Guidance and Investor Day
- Financial Guidance Outlook: Pharming expects total revenues for 2026 to range between $405 million and $425 million, reflecting an 8% to 13% growth rate, indicating strong ongoing growth in its rare disease commercial products and bolstering investor confidence in future developments.
- Increased R&D Spending: The total operating expenses are projected to be between $330 million and $335 million in 2026, primarily driven by increased R&D expenditures related to ongoing clinical trials for leniolisib and napazimone (KL1333), showcasing the company's commitment to innovative drug development.
- Pipeline Progress: Pharming is advancing two major clinical-stage programs targeting primary immunodeficiencies with immune dysregulation and mtDNA-driven mitochondrial disease, with top-line data from both trials expected in the second half of 2026, potentially opening new market opportunities for the company.
- Investor Day Event: Today's Investor Day will focus on Pharming's scientific strategy and clinical plans, expected to attract attention from industry experts and further enhance the company's market position and brand influence in the rare disease sector.
See More
Pharming Group Announces 2026 Financial Guidance and Investor Day Highlights
- Financial Outlook: Pharming anticipates total revenues between $405 million and $425 million for 2026, reflecting an 8% to 13% growth driven by robust performance from Joenja® and RUCONEST®, indicating strong market expansion potential in rare diseases.
- R&D Investment: The company expects total operating expenses to range from $330 million to $335 million in 2026, primarily due to increased R&D costs associated with ongoing clinical trials for leniolisib and napazimone (KL1333), underscoring its commitment to innovative drug development.
- Pipeline Progress: Pharming is advancing two key programs, leniolisib for primary immunodeficiencies with immune dysregulation and napazimone (KL1333) for mtDNA-driven mitochondrial disease, both of which aim to address significant unmet medical needs and enhance the company's competitive position in the market.
- Investor Day Event: The company will showcase the scientific rationale and clinical strategy behind its pipeline at today's Investor Day, which is expected to attract investor interest and further solidify its market position in the rare disease sector.
See More
FDA Issues Complete Response Letter to Pharming for Joenja Pediatric Application
- Regulatory Impact: The FDA issued a Complete Response Letter to Pharming Group regarding its supplemental New Drug Application for Joenja (leniolisib) in children aged 4 to 11, resulting in a 16.76% drop in premarket trading to $17.03, indicating negative market sentiment towards the news.
- Dosage Concerns: The FDA raised issues about potential underexposure in lower-weight pediatric patients, requesting additional pediatric pharmacokinetic data to reassess proposed doses, which could significantly impact the drug's market potential if not resolved.
- Testing Method Deficiencies: The FDA identified issues with one of the analytical methods used for production batch testing, asking Pharming for further data and clarification, which may delay the drug's market entry and increase R&D costs.
- Meeting Plans and Market Response: Pharming intends to request a Type A meeting with the FDA; although Joenja was approved for patients aged 12 and older in March 2023, this regulatory action could hinder its promotional strategy in the pediatric market, affecting future revenue expectations.
See More
Xpeng Inc Reports Disappointing Delivery Numbers, Stock Drops
- Disappointing Delivery Numbers: Xpeng Inc reported delivering 20,011 vehicles in January 2026, a figure that, while substantial, fell short of market expectations, leading to a decline in investor confidence.
- Stock Price Reaction: Following the disappointing delivery results, Xpeng's stock dropped 5.6% in pre-market trading to $16.97, reflecting concerns about the company's future performance among investors.
- Overall Market Trend: U.S. stock futures were lower, with Dow futures falling around 100 points, indicating a cautious market sentiment that could further impact investor attitudes towards Xpeng.
- Industry Impact: The delivery figures from Xpeng may negatively affect overall confidence in the electric vehicle market, especially in an increasingly competitive landscape, prompting investors to reassess their strategies.
See More
DarkIris Inc Reports 27.3% Revenue Growth, Shares Surge
- Significant Revenue Growth: DarkIris Inc reported a year-over-year revenue increase of 27.3% for FY25, reaching $10.08 million compared to $7.92 million in FY24, indicating strong market performance and growth potential.
- Stock Price Surge: Following the positive earnings report, DarkIris shares jumped 104.2% in pre-market trading to $0.68, reflecting investor optimism about the company's future prospects.
- Positive Market Reaction: The release of this earnings report has significantly increased market attention on DarkIris, enhancing investor confidence in its continued growth and potentially attracting more capital inflow.
- Optimistic Industry Outlook: The robust performance of DarkIris not only strengthens its market position but may also provide more opportunities for future financing and expansion plans, further solidifying its competitive edge in the industry.
See More
