TM Owner PTC Therapeutics
Kebilidi 2.8 x 10^11 vg/0.5ml Single-dose vial (suspension for intraputaminal infusion)
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Indications & Clinical Uses
- Kebilidi 2.8 x 10^11 vector genomes per 0.5 mL (eladocagene exuparvovec-tneq) is a one-time adeno-associated virus serotype 2 gene therapy for aromatic L-amino acid decarboxylase (AADC) deficiency in adults and children.
- It delivers a working copy of the DDC gene so that brain cells can make the enzyme needed to produce dopamine.
- Kebilidi is not an ordinary injection: the total dose of 1.8 x 10^11 vector genomes is infused directly into the putamen of the brain as four separate 0.08 mL infusions, two into each side, during a single stereotactic neurosurgical procedure carried out under general anaesthetic in a specialist centre.
- Children must have reached skull maturity, confirmed by brain imaging; the youngest patient studied was 16 months old.
- US approval in 2024 was accelerated, based on gross motor milestone gains at 48 weeks.
- The identical active substance is authorised in the European Union under a different brand name, Upstaza, for patients aged 18 months and older.
Side Effects
- In the studies supporting Kebilidi the commonest reactions, reported in at least 15% of patients, were dyskinesia, meaning involuntary movements, in about 77%, fever in about 38%, low blood pressure in about 31%, anaemia in about 31%, and increased saliva, low potassium, low phosphate, low magnesium, difficulty sleeping and complications of the procedure itself.
- Dyskinesia usually appears within the first three months after treatment and is managed by the specialist team.
- Serious complications of the neurosurgical procedure have included respiratory and cardiac arrest within 24 hours of the operation, so intensive monitoring follows the surgery.
- An immune response against the AAV2 vector can develop.
- Kebilidi does not carry a boxed warning.
- Tell the team urgently about breathing difficulty, seizures, fever, worsening involuntary movements, confusion or any change in feeding or alertness after treatment.
Precautions & Warnings
- Kebilidi is given once only, in a hospital with neurosurgical expertise, and cannot be repeated, so the decision is made by a specialist multidisciplinary team.
- Brain imaging must confirm skull maturity before surgery.
- The procedure carries the usual risks of stereotactic brain surgery and general anaesthesia, and patients are watched closely for at least 24 hours afterwards for breathing and heart complications, then monitored for dyskinesia over the following months.
- Antibodies to the AAV2 vector may form.
- Existing treatments for AADC deficiency symptoms should only be adjusted or stopped on the specialist's advice.
- The vials are stored and transported frozen at minus 65 degrees Celsius or below; once thawed the suspension is used within 6 hours and never refrozen.
- Preparation and handling follow local rules for genetically modified material.
Avoid Interactions
- No formal interaction studies have been carried out with Kebilidi, so the team must be told about every medicine, herbal product and supplement being taken, including those for AADC deficiency symptoms.
- Because the therapy is delivered during brain surgery under general anaesthetic, medicines that increase bleeding risk, such as antiplatelet drugs, anticoagulants and high-dose non-steroidal anti-inflammatory drugs, matter most and are managed by the surgical team before and after the operation.
- Dopaminergic and other symptomatic treatments may need to be changed as the child responds, but only under specialist supervision, because stopping them suddenly can be harmful.
- Kebilidi must not be diluted or mixed with any other product, must not be given intravenously, and is infused only through the authorised cannula at the specified slow rate.
- No live vaccine restriction is stated on the label; check timing with the team.
References
Frequently Asked Questions
Kebilidi (eladocagene exuparvovec-tneq) is a one-time gene therapy for aromatic L-amino acid decarboxylase (AADC) deficiency in adults and children. It delivers a working DDC gene to brain cells so they can make dopamine. US approval was accelerated, based on gross motor milestone gains at 48 weeks.
Kebilidi is infused directly into the putamen of the brain during a single stereotactic neurosurgery under general anaesthetic. The 1.8 x 10^11 vector genome dose is split into four 0.08 mL infusions, two per side, each given over about 27 minutes. It is never given into a vein.
The commonest side effects of Kebilidi are involuntary movements (dyskinesia), affecting about 77% of patients, fever, low blood pressure, anaemia, extra saliva and low potassium, phosphate or magnesium. Dyskinesia usually starts within three months. Report breathing trouble, seizures or reduced alertness urgently.
Before Kebilidi surgery, avoid medicines that raise bleeding risk unless the team agrees, including antiplatelet drugs, anticoagulants and high-dose anti-inflammatory painkillers. Do not stop or change existing AADC treatments without specialist advice. This eladocagene exuparvovec suspension is never mixed, diluted or given into a vein.
Kebilidi is given in one operating-room session: the surgeon uses stereotactic guidance to place a cannula into each putamen and infuses the gene therapy very slowly. Monitoring in hospital continues for at least 24 hours afterwards, because breathing and heart complications have occurred in that window.
Yes. Kebilidi and Upstaza are the same active substance, eladocagene exuparvovec, sold under different brand names: Kebilidi in the United States and Upstaza in the European Union, where it is approved from 18 months of age. US treatment requires confirmed skull maturity on brain imaging.
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