FDA Grants Conditional Approval For PTC Therapeutics' Gene Therapy As First Ever With Direct Administration In Brain

On Thursday, the FDA granted accelerated approval to PTC Therapeutics, Inc.’s PTCT gene therapy for AADC deficiency, the first-ever gene therapy approved in the U.S. that is directly administered to the brain.

The gene therapy, which will be marketed in the United States with the brand name Kebilidi (eladocagene exuparvovec-tneq), is indicated for the treatment of children and adults with aromatic L-amino acid decarboxylase (AADC) deficiency, including the full spectrum of disease severity.

Launch preparations are well underway, with centers already identified and surgeons trained in the procedure to deliver the gene therapy. 

Also Read: PTC Therapeutics Says FDA Lifts Clinical Hold On Investigational Study In Patients With Rare Weak Nerve Cell Disorder

AADC deficiency is a rare genetic disorder that results in the inability to synthesize dopamine, a neurotransmitter essential for motor function.

Affected individuals may experience symptoms such as delays in gross motor function (head control, sitting, standing, and walking), hypotonia (weak muscle tone), and developmental and cognitive delays. 

Kebilidi is a gene replacement therapy that is directly administered to the putamen of the brain through a stereotactic neurosurgical procedure.

Kebilidi is administered via four infusions in one surgical session into a large structure in the brain involved in motor control.

Clinical trial results demonstrate that following gene therapy, de novo synthesis of dopamine occurs, followed by the progressive acquisition of motor development milestones. 

The safety and effectiveness of Kebilidi were demonstrated in an open-label, single-arm clinical study in 13 pediatric patients.

The efficacy of Kebilidi was demonstrated based on gross motor function improvement in 8 of 12 treated patients, which has not been reported in untreated patients with the severe presentation of AADC deficiency. 

Kebilidi received accelerated approval based on the safety and clinical efficacy findings in the ongoing global gene therapy clinical trial (PTC-AADC-GT-002). 

Confirmatory evidence will be provided from the long-term follow-up of patients already treated in the study.

Along with the approval, a rare disease priority review voucher was granted. The company plans to monetize the voucher.

Barclays maintains PTC Therapeutics with an Equal-Weight rating, raising the price target from $43 to $45.

Price Action: PTCT stock is down 2.41% at $42.97 at last check Thursday.

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