Pharming Group N.V. Stock Price, News & Analysis

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.