Drug Pipeline News

The higher dose regimen of nusinersen comprises a more rapid loading regimen, two 50 mg doses 14 days apart, and higher maintenance regimen, 28 mg, every 4 months.

The digestive enzyme cartridge is designed to mimic pancreatic lipase function.

The lead clinical trial investigator for a phase 3 study of crinecerfont in pediatric CAH patients breaks down the December 2024 FDA approval.

Neffy was first approved in September 2024 to treat type 1 allergic reactions in patients who weigh at least 66 lbs (33 kg).

Positive interim data was reported from the ENERGY 1 trial and Expanded Access Program that featured a total of 5 infants and 1 child.

Contemporary Pediatrics' editorial advisory member Russell Libby, MD, FAAP, highlights 2024 FDA approval of epinephrine nasal spray (Neffy) in this video interview.

Needle-free treatments, such as Neffy and FluMist, offer promising alternatives for children and adults with needle phobia, ensuring access to critical care.

The indication is for patients 12 years and older hemophilia A with factor VIII inhibitors or hemophilia B with factor IX inhibitors.

The FDA has approved the first generic once-daily GLP-1 injection for the improvement of glycemic control for type 2 diabetes in patients 10 years and up.

The expanded indication is approved to reduce excess body weight and maintain reduction long-term in children 2 years and up with obesity due to BBS, POMC, or LEPR deficiency.

"Tapinarof comes in with that mixture of the short-term studies and longer-term studies intermittently, giving us a nice, effective alternative non-steroid for eczema across the ages."

The primary endpoint was non-inferiority in HbA1c levels after 26 weeks.

With the federal agency's decision, remestemcel-L-rknd has become the first FDA-approved mesenchymal stromal cell therapy.

From new topical dermatology treatments for atopic dermatitis to the first nasal spray to treat type 1 allergic reactions, these are our top FDA approvals of 2024.

Currently, golimumab is approved in adults with moderately to severely active ulcerative colitis.

The federal agency has set a target action date of June 10, 2025 for potential approval.

Arcutis seeks FDA approval for roflumilast cream 0.05% (ZORVYE) to treat mild to moderate atopic dermatitis in children 2 to 5 years.

The FDA has approved Crenessity for managing classic CAH in patients 4 years and up, reducing steroid doses while controlling hormones, offering a safer treatment option.

The halt follows a severe respiratory disease safety signal observed in a July 2024 phase 1 trial of Moderna's mRNA-1345 and mRNA-1365 vaccine candidates.

Pharming Group N.V. plans to include these findings in worldwide regulatory filings in 2025.

The submission to treat PsO is for children aged 6 years and up while the jPsA submission is to treat children aged 5 years and older.

Both monthly migraine days and headache days were reduced vs placebo in patients aged 6 to 17 years.

101-PGC-005 is Type IA prodrug of dexamethasone that targets CD206+ macrophages.

If approved, SL1009 has the potential to be the first approved medication for PDCD, and would be available as an oral solution.

Currently, there are no treatments for the serious, rare pediatric disease.