An innovation for patients
with AADC deficiency
KEBILIDI is the FIRST AND ONLY gene therapy approved for patients with aromatic L-amino acid decarboxylase (AADC) deficiency1
KEBILIDI is a gene therapy indicated for the treatment of adult and pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency.
This indication is approved under accelerated approval based on a change from baseline in gross motor milestone achievement at 48 weeks post-treatment. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory clinical trial.
KEBILIDI is a one-time therapy that is delivered into the putamen, a part of the brain where the AADC enzyme is made
In AADC deficiency, a genetic variation causes a shortage in AADC enzyme production.
KEBILIDI delivers a working copy of the DDC gene to help cells produce the AADC enzyme and neurotransmitters your body needs, such as dopamine.
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Complete the form below, and a PTC Therapeutics team member will contact you.
Do you have a patient or loved one with AADC deficiency who may be eligible for KEBILIDI? Are you seeking more information? Would you like to receive updates about KEBILIDI? Would you like to speak to a member of the PTC Therapeutics team?
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Important Safety Information
What is KEBILIDI™ used for?
KEBILIDI is a gene therapy indicated for the treatment of adult and pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency.
What Warnings should I know about KEBILIDI?
Procedural complications can occur with administration of KEBILIDI, which can include stopping of breathing or stopping of the heart within 24 hours of the procedure and during post-surgical care. Additional procedure related adverse events include cerebrospinal fluid leak, bleeding within the brain, inflammation of brain tissue, damage of brain tissue, and infection.
Uncontrolled movement was reported after administration of KEBILIDI. All events were reported within 3 months of administration and 2 events required hospitalization. Uncontrolled movements in the face, arm, leg, or entire body should be reported right away to a healthcare provider.
You should not use KEBILIDI if:
Skull maturity has not been achieved when assessed by neuroimaging, since skull maturity is needed for administration of KEBILIDI.
What are the side effects of KEBILIDI?
The most common side effects of KEBILIDI are uncontrolled movement, fever, low blood pressure, low red blood cell count, excess saliva production, low blood potassium, low blood phosphate, sleeplessness, low blood magnesium, and procedural complications, including stopping of breathing and stopping of the heart.
To report an adverse event, please call 1-866-562-4620 or email at usmedinfo@ptcbio.com. You may also report side effects to FDA at 1-800-FDA-1088 or at www.fda.gov/medwatch.
Please see www.KEBILIDI.com for the full Prescribing Information for additional information.