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Pharming Group N.V. (Symbol: PHAR) is a prominent global biopharmaceutical company committed to revolutionizing the lives of patients suffering from rare, debilitating, and life-threatening diseases. With its headquarters in Leiden, Netherlands, Pharming Group is at the forefront of developing and commercializing an innovative portfolio of protein replacement therapies and precision medicines.
The company's flagship product, Ruconest, is a crucial protein replacement therapy designed to address hereditary angioedema (HAE), a rare genetic condition. Pharming generates significant revenue from the United States, reflecting its strong market presence.
Recently, Pharming achieved a major milestone with the enrollment of the first patient in its Phase III clinical trial in Japan for evaluating leniolisib, a treatment for activated phosphoinositide 3-kinase delta syndrome (APDS) in both adult and pediatric patients aged 12 years and older. This trial aims to assess the safety, tolerability, and efficacy of leniolisib, potentially offering a new treatment avenue for APDS patients in Japan.
Pharming's dedication to addressing unmet medical needs extends beyond Japan. The company successfully launched leniolisib in the US under the brand name Joenja®, following its FDA approval in March 2023. This makes Joenja® the first and only targeted treatment for APDS in adult and pediatric patients aged 12 and older. Pharming plans further regulatory submissions in Europe, the UK, Canada, Australia, and additional markets.
APDS, a rare primary immunodeficiency, is caused by genetic variants impacting the immune system, leading to severe infections and immune dysregulation. Pharming's focus on genetic testing for a definitive diagnosis underscores its commitment to precision medicine and patient-centric care.
Beyond leniolisib, Pharming is actively engaged in early to late-stage development of a diversified portfolio, including small molecules, biologics, and gene therapies. The company operates on a global scale, serving patients in over 30 markets across North America, Europe, the Middle East, Africa, and Asia-Pacific.
Pharming Group continues to forge strong partnerships and collaborate with leading healthcare institutions and regulatory bodies worldwide. This strategy not only accelerates research and development but also ensures timely access to life-changing therapies for patients with rare diseases.
For the latest updates and detailed information about Pharming Group's ongoing projects, financial performance, and strategic initiatives, visit their official website at www.pharming.com or follow them on LinkedIn.
Pharming Group N.V. (Euronext Amsterdam: PHARM, Nasdaq: PHAR) has announced the first commercial shipments of Joenja® (leniolisib) to patients in the United States. Joenja® is the first and only approved treatment for activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS), a rare primary immunodeficiency affecting adults and children aged 12 and older. The commencement of these shipments triggers a $10 million milestone payment to Novartis, under a 2019 licensing agreement. Joenja® was recently approved by the FDA, and the company aims to make it widely available across the U.S. Additionally, leniolisib is undergoing further regulatory reviews in Europe, the UK, Canada, Australia, and Japan, with ongoing clinical trials for younger patients suffering from APDS.
Pharming Group N.V. announced participation in the Guggenheim Healthcare Talks focused on Genomic Medicines and Rare Disease Days, scheduled for April 3-4, 2023, in New York. CEO Sijmen de Vries and CCO Stephen Toor will feature in a fireside chat on April 3 at 15:20 ET. Interested investors can access a live webcast and replay on Pharming's website. Additionally, they will be available for one-on-one meetings during the conference. Pharming is dedicated to developing treatments for rare diseases and operates globally, serving patients in over 30 markets.
Pharming Group N.V. has announced that the US Food and Drug Administration (FDA) has approved Joenja® (leniolisib) as the first and only treatment for activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in patients aged 12 and older. This milestone treatment, expected to launch in early April, was developed under priority review due to its potential to improve outcomes for patients with this rare disease. Joenja® demonstrated significant clinical efficacy in a Phase II/III trial. Pharming will incur milestone payments of approximately $10.5 million to Novartis upon approval and additional sales-based payments up to $190 million.
Pharming Group N.V. has announced the enrollment of the first patient in its Phase III clinical trial (NCT05438407) for leniolisib, aimed at treating children with activated phosphoinositide 3-kinase delta syndrome (APDS), a rare genetic disorder. The trial will assess leniolisib's safety, efficacy, and tolerance in approximately 15 children aged 4 to 11 years across the U.S., Europe, and Japan. Primary efficacy endpoints include reducing lymph node size and increasing naïve B cell counts after 12 weeks. A subsequent trial for younger children (aged 1-6) is planned for Q3 2023. Leniolisib has shown promise in prior studies, and regulatory reviews are ongoing for broader approval.
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