Drugs

Pharming Group’s Joenja Gets Expanded Approval

FDA has granted Pharming Group expanded approval for Joenja (leniolisib) tablets for treating activated phosphoinositide 3-kinase delta syndrome (APDS) in children 4-11 years who weigh at least 27 kg. Joenja was first approved in 2023 for the same indication in patients 12 years and older at a dose of 70 mg orally twice a day. FDA says children 4-11 years take a weight-based dosage (40, 50, or 70 mg) orally twice a day.

APDS is a rare genetic disorder that impairs immune system cells. “These cells are necessary to recognize and attack foreign invaders, such as viruses and bacteria, to prevent infection,” FDA says. “People with APDS develop recurrent infections, particularly in the sinuses, ears, and respiratory tract. They also develop enlarged lymph nodes, tonsils, spleen, and other organs that can cause obstruction in the airway and gastrointestinal tract. Individuals with this condition often have low numbers of white blood cells, including red cells and platelets. Patients with APDS are more prone to develop blood cell cancers, like lymphoma.”

APDS is caused by mutations in the PIK3CD or PIK3R1 genes that encode the phosphoinositide-3 kinase delta (PI3K delta) protein, which is important for the normal development and function of white blood cells in the body, the agency says.

Approval was based on data from a single-arm, open-label study (Study LE 3301) that involved eight patients who received the recommended Joenja dosage based on body weight. “The pharmacokinetic data demonstrated no clinically significant difference between patients younger or older than 12 years,” FDA says.

Gupta Raises Concerns About Overton’s Independence, Ideology

FDA commissioner nominee Heidi Overton’s responses on vaccines, contraception and abortion medication during her 9/24 Senate confirmation hearing raise concerns that ideology could influence her decision-making if she takes over the agency, MSNBC senior medical analyst Dr. Vin Gupta said Friday. Gupta was particularly critical of Overton’

Overton Confirmation Remains Uncertain After Senate Hearing

FDA commissioner nominee Heidi Overton emerged from her Senate confirmation hearing Thursday (see earlier story) without resolving questions about whether she has enough Republican support to advance — and with a rapidly narrowing window for confirmation before the midterm elections. The Senate Health, Education, Labor and Pensions Committee hearing provided few

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA has classified Boston Scientific's recall of its Imager II Angiographic Catheters as Class I because a manufacturing problem could cause catheter tips to degrade and detach, potentially resulting in life-threatening complications. The agency announced the Class I classification 9/25, updating an Early Alert it issued

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome