Pharming Group NV is a holding company, which engages in the development of pharmaceutical products for the treatment of rare diseases and unmet medical needs. The firm offers RUCONEST, which is a recombinant human C1-esterase inhibitor for the treatment of acute hereditary angioedema. It operates through the following segments: RUCONEST?, Joenja?, Europe, and Rest of the World. The company was founded on November 11, 1988, and is headquartered in Leiden, the Netherlands.
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药物批准
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监管机构
3
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成立时间
1988
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- The FDA approved Pharming's supplemental New Drug Application for 40 mg and 50 mg twice-daily leniolisib in children aged 4 to 11 weighing at least 27 kg with APDS. - Joenja becomes the first FDA-approved treatment for this younger pediatric APDS population, expanding an indication previously limited to patients 12 years and older. - Approval was supported by a multinational, open-label, single-arm Phase III study showing reduced lymphadenopathy and increased naive B cells over 12 weeks. - Pharming submitted a separate application on July 30 seeking approval of lower doses for children weighing 13 kg to less than 27 kg.
- More than 300 million people worldwide live with one of over 7,000 identified rare diseases, yet diagnostic delays average four to five years and can stretch to nine years in the US. - Biotech leaders argue that meaningful innovation requires embedding patient voices into trial design, endpoints, and real-world evidence rather than relying on clinical data alone. - The FALCON study in primary mitochondrial disease co-created endpoints with patients, prioritizing fatigue and functional strength as the outcomes that matter most. - Rare diseases impose a heavy burden, with an estimated annual cost of nearly $1 trillion in the US and elevated rates of depression (39%) and anxiety (44%) among patients.
- Mary Catchpole, 19, becomes the first European patient to receive leniolisib (Joenja), a newly approved targeted treatment for activated PI3-kinase delta syndrome (APDS), a rare inherited immune disorder. - The drug works by inhibiting an overactive enzyme that disrupts immune function, offering a potential cure for patients who previously faced lifelong infections and invasive treatments. - APDS was discovered by Cambridge researchers in 2013 with help from Catchpole's family, who lost four members to the condition before this breakthrough treatment became available. - The NHS approved leniolisib at a list price of £352,000 per year with a confidential discount, potentially benefiting up to 50 patients over age 12 in England.
- Pharming Group N.V. has announced a public cash offer to acquire Abliva AB for approximately US$66.1 million, strengthening its pipeline. - Abliva's lead product, KL1333, is in a pivotal clinical trial for primary mitochondrial disease (PMD) driven by mitochondrial DNA mutations. - KL1333 has shown positive clinical effects and has received Fast Track and Orphan Drug designations, targeting over 30,000 patients. - The acquisition, funded by Pharming's existing cash, aims for a U.S. launch of KL1333 in 2028, pending regulatory approvals.
- Pharming's leniolisib demonstrated positive topline results in a Phase 3 trial for children aged 4-11 with Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS). - The study showed improvements in lymphoproliferation and immunophenotype correction, consistent with results from trials in older patients. - Pharming plans to submit global regulatory filings in 2025 to seek approval of leniolisib for treating APDS in younger pediatric patients. - All 21 patients completed the 12-week treatment, with adverse events reported as mild to moderate and no drug-related serious adverse events.
- The MHRA has approved Joenja (leniolisib) as the first medicine for Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS) in the UK. - APDS is a rare, inherited immune disorder affecting approximately 30-40 people in the UK, characterized by recurrent infections and abnormal immune function. - Leniolisib demonstrated a statistically significant reduction in lymph node size and normalization of immunophenotype in a 12-week placebo-controlled trial. - The approval was expedited through the International Recognition Procedure (IRP), leveraging prior FDA approval to provide quicker access to UK patients.
