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FDA Approves PTC Therapeutics' Kebilidi, First Gene Therapy with Direct Brain Administration for AADC Deficiency

• The FDA granted accelerated approval to PTC Therapeutics' Kebilidi (eladocagene exuparvovec-tneq) for AADC deficiency, marking the first gene therapy approved in the U.S. for direct brain administration. • Kebilidi, a gene replacement therapy, is indicated for both children and adults with AADC deficiency, addressing the full spectrum of disease severity by restoring dopamine synthesis. • Clinical trials demonstrated significant gross motor function improvement in 8 of 12 treated pediatric patients, a milestone not typically observed in untreated patients with severe AADC deficiency. • The approval was based on safety and efficacy data from the PTC-AADC-GT-002 trial, with confirmatory evidence expected from long-term follow-up studies.

The FDA has granted accelerated approval to PTC Therapeutics' Kebilidi (eladocagene exuparvovec-tneq), a gene therapy for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency. This marks the first gene therapy approved in the U.S. that involves direct administration to the brain. The approval is a significant advancement for patients with this rare genetic disorder, which impairs motor function due to the inability to synthesize dopamine.

Kebilidi: A Gene Replacement Therapy

Kebilidi is a gene replacement therapy administered directly into the putamen of the brain via a stereotactic neurosurgical procedure. The treatment involves four infusions in a single surgical session, targeting a key structure involved in motor control. By delivering a functional gene, Kebilidi aims to restore dopamine synthesis in patients with AADC deficiency.
AADC deficiency is a rare genetic disorder that prevents the synthesis of dopamine, a crucial neurotransmitter for motor function. Individuals with this deficiency often experience delays in motor skills such as head control, sitting, standing, and walking, as well as hypotonia (weak muscle tone) and developmental delays.

Clinical Trial Efficacy

The safety and efficacy of Kebilidi were demonstrated in an open-label, single-arm clinical study involving 13 pediatric patients. Results showed that the gene therapy led to de novo synthesis of dopamine, followed by progressive acquisition of motor development milestones. Specifically, gross motor function improvement was observed in 8 of 12 treated patients, a result not typically seen in untreated patients with severe AADC deficiency.
The approval was based on the safety and clinical efficacy findings from the ongoing global gene therapy clinical trial (PTC-AADC-GT-002). Continued long-term follow-up of treated patients will provide further confirmatory evidence.

Regulatory and Market Impact

With the approval, a rare disease priority review voucher was granted, which PTC Therapeutics plans to monetize. Barclays has maintained an Equal-Weight rating for PTC Therapeutics, raising the price target from $43 to $45.
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FDA Approves PTC Therapeutics' Gene Therapy Kebilidi for AADC Deficiency

The FDA has approved eladocagene exuparvovec-tneq (Kebilidi), a gene therapy developed by PTC Therapeutics, for treating AADC deficiency in adult and pediatric patients.
Kebilidi is the first FDA-approved gene therapy for AADC deficiency, a rare genetic disorder affecting neurotransmitter production and causing severe developmental delays.

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.
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The FDA has approved Kebilidi (eladocagene exuparvovec), a gene therapy by PTC Therapeutics, for aromatic L-amino acid decarboxylase (AADC) deficiency, a rare genetic disorder.
Kebilidi is the first gene therapy approved in the U.S. that involves direct administration into the brain, offering a novel approach to treating AADC deficiency.

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The FDA has granted accelerated approval to Kebilidi (eladocagene exuparvovec-tneq), a gene therapy for AADC deficiency, marking the first such treatment approved in the U.S.
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FDA Approves PTC Therapeutics' KEBILIDI, a Gene Therapy for AADC Deficiency

The FDA has granted accelerated approval to KEBILIDI (eladocagene exuparvovec-tneq), a gene therapy for AADC deficiency, marking the first FDA-approved gene therapy directly administered to the brain.
KEBILIDI is indicated for both children and adults with AADC deficiency, addressing the full spectrum of disease severity and offering a new treatment option for this rare genetic disorder.

FDA Approves PTC Therapeutics' Gene Therapy KEBILIDI for AADC Deficiency

The FDA has granted accelerated approval to KEBILIDI (eladocagene exuparvovec-tneq), a gene therapy for AADC deficiency, marking the first FDA-approved gene therapy directly administered to the brain.
KEBILIDI, developed by PTC Therapeutics, is indicated for both children and adults with AADC deficiency, addressing a critical unmet need across the spectrum of disease severity.

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]
FDA Grants Conditional Approval For PTC Therapeutics' Gene Therapy As First Ever...
finance.yahoo.com · Nov 8, 2024

FDA approved PTC Therapeutics' Kebilidi, the first U.S. gene therapy directly administered to the brain for AADC deficie...

[2]
FDA Grants Conditional Approval For PTC Therapeutics' Gene Therapy As First Ever With ...
finance.yahoo.com · Nov 14, 2024

FDA grants conditional approval to PTC Therapeutics' gene therapy for AADC deficiency, the first to be directly administ...

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