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FDA Approves Kebilidi Gene Therapy for AADC Deficiency and Accepts BLA for Pz-cel in RDEB

• The FDA has approved Kebilidi, the first gene therapy for aromatic L-amino acid decarboxylase (AADC) deficiency, administered via infusions into the brain. • A Biologics License Application (BLA) resubmission for prademagene zamikeracel (pz-cel) to treat recessive dystrophic epidermolysis bullosa (RDEB) has been accepted by the FDA, with a target action date of April 25, 2025. • Updated guidelines for resuscitation following drowning have been jointly released by the American Academy of Pediatrics (AAP) and the American Heart Association (AHA), based on systematic reviews. • Experts emphasize early identification of drug-resistant epilepsy patients in primary care to facilitate timely referral and access to advanced interventions like surgery.

The FDA has approved Kebilidi as the first gene therapy for treating aromatic L-amino acid decarboxylase (AADC) deficiency, a rare genetic disorder that can cause severe motor and developmental impairments. Additionally, the FDA has accepted the resubmitted Biologics License Application (BLA) for prademagene zamikeracel (pz-cel) for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), setting a target action date for approval in April 2025. These developments mark significant advancements in the treatment of rare and debilitating conditions.

Kebilidi Approved for AADC Deficiency

Kebilidi, a gene therapy administered through four infusions in a single surgical session directly into a key motor control structure in the brain, offers a new treatment option for patients with AADC deficiency. The therapy is intended to be administered in specialized medical centers equipped for pediatric stereotactic neurosurgery.
"AADC deficiency can cause a range of debilitating symptoms, including life-threatening complications," stated 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."

BLA Resubmission Accepted for Pz-cel in RDEB

Abeona Therapeutics announced that the FDA has accepted the resubmission of its BLA for prademagene zamikeracel (pz-cel) as a potential treatment for recessive dystrophic epidermolysis bullosa (RDEB). The FDA has assigned a target action date of April 25, 2025.
Pz-cel, an investigational autologous, COL7A1 gene-corrected epidermal sheet, had previously received Priority Review, but its approval was delayed following a Complete Response Letter (CRL) issued in April 2024.

Updated Resuscitation Guidelines Following Drowning

The American Academy of Pediatrics (AAP) and the American Heart Association (AHA) have jointly updated their guidelines for resuscitation following drowning. Published in Pediatrics and Circulation, the updated guidance is based on seven systematic reviews by the International Liaison Committee on Resuscitation task force.
The reviews covered critical topics, including:
  • In-water resuscitation versus delayed resuscitation
  • Oxygen administration following drowning
  • AED first versus CPR first in cardiac arrest after drowning
  • Chest compressions, airway, breathing versus airway, breathing, chest compressions
  • Implementation of PAD programs
  • Ventilation with equipment versus without equipment before hospital arrival following drowning
  • Compression-only CPR

Identifying Drug-Resistant Epilepsy in Primary Care

Drug-resistant epilepsy remains a significant challenge in pediatric care. H. Westley Phillips, MD, pediatric neurosurgeon-scientist and epilepsy researcher at Stanford Medicine Children’s Health, noted that “If a patient fails two medications, the likelihood that the next medication will result in seizure freedom is less than 5%.”
Phillips emphasized the importance of early detection and referral, stating, “We have very recent data demonstrating that the time from diagnosis of epilepsy to surgical evaluation can be upwards of 16 years.” He encouraged pediatricians to maintain a high index of suspicion and promptly refer patients with suspected seizures to pediatric neurologists.
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The FDA has granted accelerated approval to Kebilidi (eladocagene exuparvovec), a gene therapy for aromatic L-amino acid decarboxylase (AADC) deficiency, the first of its kind to be injected directly into the brain.
Kebilidi is designed to boost AADC enzyme production in the brain, restoring dopamine levels and improving motor function in children and adults with the rare genetic disorder.

FDA Approves Kebilidi, First Gene Therapy for AADC Deficiency

The FDA has granted accelerated approval to Kebilidi (eladocagene exuparvovec-tneq) for treating Aromatic L-Amino Acid Decarboxylase (AADC) deficiency in adults and children.
Kebilidi, an adeno-associated virus vector-based gene therapy, is the first FDA-approved treatment of its kind for this rare genetic disorder affecting neurotransmitter production.

FDA Approves Kebilidi, First Gene Therapy for AADC Deficiency

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Kebilidi, an adeno-associated virus vector-based therapy, delivers the human DDC gene directly into the putamen to enhance dopamine production.

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The FDA has approved Kebillidi (eladocagene exuparvovec-tneq) from PTC Therapeutics, a gene therapy for both adult and pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency.
Kebillidi's approval is based on a single-arm study demonstrating significant motor milestone gains in a majority of treated pediatric patients compared to untreated individuals.

FDA Approves Kebilidi, First Brain-Delivered Gene Therapy for AADC Deficiency

The FDA has approved Kebilidi (eladocagene exuparvovec-tneq), a gene therapy for AADC deficiency, marking the first brain-delivered gene therapy approved in the U.S.
Kebilidi is administered directly into the brain and replaces the mutated DDC gene with a functional version, restoring dopamine and serotonin production in both adults and children.

FDA Approves PTC Therapeutics' Kebilidi, First Gene Therapy for AADC Deficiency

The FDA has approved Kebilidi (eladocagene exuparvovec-tneq) from PTC Therapeutics, the first gene therapy for Aromatic L-amino acid decarboxylase (AADC) deficiency.
Kebilidi is an adeno-associated virus vector-based gene therapy designed for both adult and pediatric patients suffering from AADC deficiency, administered via neurosurgical infusion.

FDA Approves Kebilidi, First Gene Therapy for AADC Deficiency

The FDA has approved eladocagene exuparvovec-tneq (Kebilidi) as the first gene therapy for aromatic L-amino acid decarboxylase (AADC) deficiency in adults and pediatric patients.
Kebilidi, administered via infusions into the brain, facilitates AADC expression and dopamine production, addressing motor, attention, learning, and memory impairments.

FDA Approves Kebilidi, First Gene Therapy Directly Administered to the Brain for AADC Deficiency

The FDA has granted accelerated approval to Kebilidi (eladocagene exuparvovec-tneq), a gene therapy for both children and adults with AADC deficiency, a rare genetic disorder.
Kebilidi, developed by PTC Therapeutics, is the first gene therapy approved in the U.S. that is directly administered to the brain via a stereotactic surgical procedure.

FDA Approves PTC Therapeutics' KEBILIDI, First Gene Therapy Delivered Directly to the Brain for AADC Deficiency

The FDA has granted accelerated approval to PTC Therapeutics' KEBILIDI, a gene therapy for AADC deficiency, marking the first such therapy delivered directly to the brain.
KEBILIDI works by using an adeno-associated virus to deliver a functional DOPA decarboxylase gene, restoring dopamine production in patients with AADC deficiency.

FDA Approves Kebilidi Gene Therapy for AADC Deficiency

The FDA has granted accelerated approval to Kebilidi (eladocagene exuparvovec-tneq) for treating AADC deficiency in both children and adults.
Kebilidi, a gene replacement therapy, is administered directly into the brain to restore dopamine production, addressing the underlying genetic defect.

Reference News

[1]
Weekly report: ADHD, epilepsy in primary care, resuscitation guidance, and more
contemporarypediatrics.com · Nov 18, 2024

FDA approves Kebilidi, the first gene therapy for aromatic L-amino acid decarboxylase deficiency; FDA accepts resubmissi...

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