PHAR Pharming Group

$PHAR The FDA has approved Joenja® for children ages 4 to 11 with APDS who weigh at least 27 kg, ahead of the October PDUFA date. Joenja is now the first FDA-approved treatment for this pediatric APDS population in the U.S. The newly approved doses are expected to become available in October through Pharming’s existing U.S. distribution and patient-support infrastructure. This approval represents another important step in expanding Joenja’s reach to younger patients and supporting its continued growth. A separate application for children ages 4 to 11 weighing between 13 kg and less than 27 kg was submitted to the FDA on July 30 and is under review. Pharming has identified ~60 eligible patients in total between 4 to 11 in the US, representing an attractive revenue opportunity.

$PHAR Another Pharming PR narrative that sounds broader than the actual approval. Joenja is now approved for children 4–11, but only if they weigh at least 27 kg. That weight corresponds roughly to the average weight of a 9-year-old, meaning a significant part of the 4–11 age group is still excluded. Yet the headline suggests a full 4–11 approval. Is this transparency, or simply another attempt to make the news sound bigger than it is?

$PHAR Pharming FDA approves Pharming's Joenja® as first treatment for children with APDS in the U.S. Leiden, the Netherlands, September 11, 2026: Pharming (Euronext Amsterdam: PHARM/Nasdaq: PHAR), a global biotechnology company focused on rare immune and genetic diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved its supplemental New Drug Application (sNDA) for 40 mg and 50 mg twice-daily dosing of Joenja® (leniolisib), an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, as a treatment for children aged 4 to 11 years weighing at least 27 kg with activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency

$PHAR Pharming IR has not answered a single critical question or comment from investors. Are you actually capable of answering them, or has the narrative become so big that doing so would inevitably mean acknowledging the setbacks behind it? If the story is as strong as presented, why not simply answer with facts and numbers?

$PHAR Pharvaris’ call this morning should be a wake-up call for Pharming. Deucrictibant showed an 87% reduction in HAE attacks in type 1/2 patients and is being positioned as an oral treatment with injectable-like efficacy. That directly challenges Ruconest’s key advantage: strong acute efficacy. Pharvaris is targeting patients who abandoned ORLADEYO because of insufficient efficacy — reportedly around 1,900 patients — while offering oral convenience. With a prophylaxis filing planned for H1 2027, deucrictibant could potentially cover both prevention and acute treatment. Ruconest may be proven, but it is not protected. An oral drug offering comparable efficacy without injections could directly erode its competitive position.

$PHAR FDA approved expanded use of Joenja for children ages 4–11 with APDS. First FDA-approved treatment for this pediatric APDS group in the U.S. Fresh FDA catalyst worth watching Monday.

$PHAR Joehoe Monitor, gelezen? Dit mooie nieuws kwam tegen je verwachting in zeker en ook nog eens eerder. Wat een zege voor al die kinderen die met deze ziekte kampen hè.

$PHAR Pharming wins approval of Joenja for younger age group. The US FDA has approved Pharming Group's (PHAR) Joenja (leniolisib) for children between the ages of four and 11 with a kind of rare genetic immune system disorder.