Drug Pipeline News

The FTD will allow the development and review timeline of nipocalimab to accelerate. It is granted to drugs with the potential to treat severe conditions and provide unmet vital medical needs.

TNX-2900 was previously granted Orphan Drug designation by the federal agency in 2022 for the treatment of PWS.

The initial dose of pemivibart is 4500 mg, which is administered as a single intravenous infusion. A repeat 4500 mg dose should be administered every 3 months if ongoing protection is needed, stated the FDA.

The decision makes givinostat the first nonsteroidal drug approved to treat patients with all genetic variants of DMD.

Administration of the HIV-1 specific, non-nucleoside reverse transcriptase inhibitor (NNRTI) to the indicated population can be done via 25 mg oral tablets or new 2.5 mg oral tablets, developed to help administration and weight-adjusted dosing for children.

The decision makes atidarsagene autotemcel the first FDA-approved gene therapy for MLD, a rare genetic disease that affects the brain and nervous system.

The approval is based on data from a phase 3, multicenter, randomized trial that evaluated patients aged 8 to 17 years with HeFH who had LDL-C levels of 130 mg / dL or greater who were being treated with lipid-lowering medications.

Understand how to naviate the current shortage of pediatric ADHD medications.

The federal agency approved Perflutren Lipid Microsphere following positive data from 3 pediatric clinical trials.

A poster presentation at AAAAI from the phase 3 PEOPLE Study revealed DBV712 treatment in children with peanut allergy has a favorable safety and tolerability profile.

The Rare Pediatric Disease Designation joins the Fast Track and Orphan Designations that have already been granted to AOC 1044.

A Prescription Drug User Fee Act (PDUFA) target action date of June 21, 2024, has been set by the FDA.

ARS Pharma plans to submit data from the repeat dose study of neffy to the FDA early in the second quarter of 2024, as part of its response to the CRL issued by the federal agency for the previously submitted New Drug Application (NDA).

The approval makes omalizumab the first and only FDA-approved medicine to reduce allergic reactions in patients with one or more food allergies.

The Orphan Drug Designation follows the receipt of Rare Pediatric Disease Designation from the FDA in January 2024.

Lawrence Eichenfield, MD, highlights the sNDA submission of tapinarof cream, 1% for the topical treatment of atopic dermatitis in children aged 2 years and older.

The sNDA submission follows additional positive topline data that was presented in Janurary 2024, highlighting an open-label, long-term extension study evaluating tapinarof cream, 1%.

The twice-daily oral corticosteroid is indicated for 12 weeks of treatment.

Vivian Hernandez-Truillo, MD, FAAP, FAAAAI, FACAAI; and Theresa Bingemann, MD, provide reaction and commentary regarding recently FDA-approved dupilumab to treat EoE in pediatric patients aged 1 to 11 years.

In this Contemporary Pediatrics Q+A interview, Weily Soong, MD, breaks down FDA-approved tralokinumab-ldrm for patients aged 12 to 17 years with moderate-to-severe atopic dermatitis.

The approval makes dupilumab the first and only treatment specifically indicated for eosinophilic esophagitis (EoE) patients aged 1 to 11 years that weigh at least 33 lbs (15 kg).

Review some of the top stories from the Contemporary Pediatrics website over the last week, and catch up on anything you may have missed.

Compared to placebo, linaclotide demonstrated a statistically significant and clinically meaningful improvement in spontaneous bowel movement(s) (SBM) frequency rate, and was subsequently approved by the FDA in June 2023.

MediWound Ltd is seeking to expand the label for NexoBrid, already approved for eschar removal in adult patients, to the pediatric indication after the FDA accepted a supplemental Biologics Application for this patient population.

Marstacimab reduced the annualized bleeding rate (ABR) by 35% and nearly 92%, respectively, compared to routine prophylaxis and on-demand treatment in hemophilia A and B patients without inhibitors, according to Pfizer.

Berdazimer gel, 10.3% demonstrated positive, topline data in the phase 3 randomized clinical B-SIMPLE4 trial (NCT04535531) that featured a total of 891 patients aged 6 months or older who had 3 to 709 raised molluscum contagiosum lesions.

Biopharmaceutical company, Invivyd, made the request to the FDA for its investigational antibody, VYD222.

Birch triterpenes (FILSUVEZ; Chiesi Global Rare Diseases) is the first approved treatment for wounds associated with junctional epidermolysis bullosa (JEB), according to the developer.

Seborrheic dermatitis patients aged 9 years and older can now use roflumilast foam 0.3% following its approval by the FDA.