Get caught up with Contemporary Pediatrics! This list helps you navigate our top stories from last week, all in one place.
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Thank you for visiting the Contemporary Pediatrics® website. Take a look at some of our top stories from last week (Monday, November 11, to Friday, November 15, 2024), and click on each link to read and watch anything you may have missed.
Kebilidi, which is administered via 4 infusions in 1 surgical session into a large structure in the brain involved in motor control, should be administered in a medical center that specializes in pediatric stereotactic neurosurgery.
"AADC deficiency can cause a range of debilitating symptoms, including life-threatening complications,” said Nicole Verdun, MD, director of the Office of Therapeutic Products in CBER, in a press release. “Today’s approval represents important progress in the advancement and availability of safe and effective treatments for debilitating genetic disorders.”
Click here for full approval details.
A resubmission Biologics License Application for prademagene zamikeracel (pz-cel) as a potential treatment for recessive dystrophic epidermolysis bullosa (RDEB) has been accepted by the FDA, according to a press release from Abeona Therapeutics.
With this acceptance, the federal agency has assigned a target action date of April 25, 2025, for an approval decision.
Pz-cel, an investigational autologous, COL7A1 gene-corrected epidermal sheet, was granted FDA Priority Review on November 28, 2023, with an original target action date of May 25, 2024.2 However, following a Complete Response Letter (CRL) that was issued in April 2024, any potential approval decision was delayed.
Click here for clinical trial data and more FDA pipeline details.
The update, published in Pediatrics and Circulation, is based on 7 systematic reviews completed by the International Liaison Committee on Resuscitation task force, used to generate treatment recommendations and good practice statements. The reviews included the following drowning topics, according to the update published in Pediatrics1:
Click here for the AAP and AHA guidelines and recommendations.
Drug-resistant epilepsy remains a critical challenge in pediatric care, with far-reaching implications for patient outcomes. “If a patient fails two medications, the likelihood that the next medication will result in seizure freedom is less than 5%,” stated H. Westley Phillips, MD, pediatric neurosurgeon-scientist and epilepsy researcher at Stanford Medicine Children’s Health, in this Contemporary Pediatrics interview. As Epilepsy Awareness Month brings this issue to the forefront, Phillips explained how primary care providers can help identify these patients for early referral and advanced interventions.
“We have very recent data demonstrating that the time from diagnosis of epilepsy to surgical evaluation can be upwards of 16 years,” Phillips emphasized. This delay often prevents patients from accessing effective options like epilepsy surgery.
To facilitate early detection, Phillips encouraged pediatricians to maintain a high index of suspicion during routine visits. He explained, “A quick referral to the pediatric neurologist if there are concerns about seizures can help diagnose a seizure that may not look like a stereotypical, generalized tonic-clonic seizure, but something more focal.”
Click here for the full video interview discussion.
“You are America’s ADHD providers,” said Rakesh Jain, MD, MPH, clinical professor of psychiatry at Texas Tech University School of Medicine, in a video interview with Contemporary Pediatrics. “I know you didn’t sign up for it, but ADHD is in your practice, and 80% to 90% of all patients with ADHD in America are being taken care of by you.”
Jain highlighted the approval of clonidine hydrochloride (ONYDA XR, Tris Pharma), a new extended-release liquid formulation of clonidine. “We’ve got once-a-day formulation that’s liquid and can be used as a monotherapy in appropriate patients, or if your patient’s currently on a stimulant, we don’t have to settle for suboptimal control of symptoms,” he explained. “We certainly can, in appropriate situations, consider augmentation therapy.”
