Kebilidi

Eladocagene exuparvovec-tneq · Suspension · Intracerebral

Prescription (Rx)

Uses

(eladocagene exuparvovec-tneq) is an adeno-associated virus (AAV) vector-based 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 the change from baseline in gross motor milestone achievement at 48 weeks post treatment [see Clinical Studies ( 14 )] . Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory clinical trial. KEBILIDI is an adeno-associated virus (AAV) vector-based 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 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. ( 1 , 14 )

Dosage and administration

For single-dose intraputaminal infusion only. For single-dose intraputaminal infusion only. Recommended dose: 1.8×10 11 vector genomes (vg). ( 2.2 ) Brain imaging for stereotactic planning and intraoperative navigation should be done prior to the procedure. ( 2.4 ) Post stereotactic registration, mark the entry point on the skull. Surgical access through the skull bone and dura should be performed. ( 2.4 ) Administer a total dose of 1.8×10 11 vg (0.32 mL total volume) delivered as four 0.08 mL (0.45×10 11 vg) infusions (two sites per putamen-anterior and posterior) at a rate of 0.003 mL/minute (0.18 mL/hour) for a total of 27 minutes per site, administered in a single stereotactic surgery using a cannula that is FDA-authorized for intraparenchymal infusion. ( 2.2 , 2.4 ) 2.1 Important Dosing Information Confirm patient has AADC deficiency due to biallelic mutations in the DDC gene. Strictly observe aseptic technique during preparation and administration of KEBILIDI. KEBILIDI should be administered in a medical center which specializes in stereotactic neurosurgery. Administer KEBILIDI only using an FDA-authorized cannula for intraparenchymal infusion (i.e., ClearPoint SmartFlow Neuro Cannula Part Number NGS-NC-01-EE or NGS-NC-02-EE). Use of the syringe (i.e., connecting the syringe to the syringe pump and priming of the cannula) should begin within 6 hours of starting product thaw KEBILIDI is intended to be administered with an infusion pump capable of infusing at a rate of 0.003 mL/min. 2.2 Recommended Dose KEBILIDI is administered as four intraputaminal infusions in a single stereotactic neurosurgical procedure as per the recommended dose shown in Table 1 . Table 1: Recommended Dose of KEBILIDI Total Recommended Dose 1.8x10 11 vg (0.32 mL) Total number of infusions 4 Volume (dose) per infusion 0.08 mL (0.45x10 11 vg) Location of infusions 2 in anterior putamen, 2 in posterior putamen Infusion rate at each target point 0.003 mL/min Dose duration for infusion at each target point 27 minutes 2.3 Preparation Thawing KEBILIDI Vial Coordinate timing of KEBILIDI thaw and infusion. KEBILIDI should be used within 6 hours of starting product thaw. Infusion of KEBILIDI takes 4 hours. The maximum time from thaw to completion of infusion should be no more than 10 hours. Thaw the KEBILIDI vial upright at room temperature before use. The contents of the vial will thaw in approximately 15 minutes at room temperature. Do not thaw or warm the vial any other way. Gently invert the vial 3 times. Do not shake the vial. Inspect the fully thawed KEBILIDI vial after mixing. KEBILIDI should be inspected visually for particulate matter, and discoloration prior to administration. KEBILIDI is clear to slightly opaque. The color of KEBILIDI should be a colorless to faint white suspension Do not use if particulates, or discoloration are visible in the suspension. Preparing KEBILIDI in Syringe Gather supplies listed in Table 2 for preparation: Table 2: Supplies for KEBILIDI Preparation Abbreviations: PC=Polycarbonate; PP=Polypropylene Component Material of Construction 1mL lubricated sterile Luer-lock syringe with elastomer plunger Or 5mL lubricated sterile Luer-lock syringe with elastomer plunger Silicone, PC; Silicone, PP Silicone, PP 18 or 19 G sterile needle with 5µm filter Stainless steel, PC hub; Stainless steel, PP hub Sterile Luer-lock syringe cap - Plastic bag for delivery into surgical unit - Secondary container for delivery into surgical unit - Prepare KEBILIDI using sterile techniques under aseptic conditions, proper engineering controls (e.g., biological safety cabinets or isolator) as per the institutional policies. Open the syringe and label it as the product-filled syringe. Attach the filter needle to the syringe. Draw the full volume of the vial of KEBILIDI into the syringe. Invert the vial and syringe and partially withdraw or angle the needle as necessary to maximize recovery of product. Draw air into the syringe so that the needle is emptied of product. Carefully remove the needle from syringe containing KEBILIDI. Purge the air from the syringe until there is no air bubble and then cap with a syringe cap. Place the syringe in a plastic bag and seal the bag. Place the plastic bag in an appropriate secondary container for delivery to the surgical suite at room temperature. The filled syringe prepared under aseptic conditions for delivery to the surgical site should be used immediately. Notes: Do not refreeze thawed product. Dispose any remaining KEBILIDI or disposable material in compliance with institutional policy 2.4 Administration Gather supplies for administration: - KEBILIDI [see How Supplied/Storage and Handling ( 16 )] - SmartFlow Neuro Cannula - Syringe pump, capable of an infusion rate of 0.003 mL/min and compatible with 1 mL or 5 mL syringe sizes - Stereotactic system Identification of the Target Points Within the Putamen Using standard neurosurgical stereotactic procedure, brain imaging for stereotactic planning and intraoperative navigation should be done prior to the procedure (see Figure 1 ). Figure 1: Four Target Points within the Putamen for Infusion Sites After stereotactic registration is complete, mark the entry point on the skull. Surgical access through the skull bone and dura should be performed. Figure 2: Infusion Delivery System Intraputaminal Administration of KEBILIDI Administer KEBILIDI by bilateral intraputaminal infusion using a single infusion cannula in one surgical session at two sites (anterior and posterior) per putamen. The infusion cannula is placed and infusion performed separately for each target point (see Figure 2 ). Tightly connect the syringe containing the prepared KEBILIDI to Luer Lock connector at the proximal end of the infusion cannula. Load syringe onto the infusion pump and secure appropriately. Set infusion pump occlusion limit to the highest level to prevent pump from alarming or disrupting the infusion.

Dosage forms and strengths

3 DOSAGE FORMS AND STRENGTHS KEBILIDI is a sterile suspension for intraputaminal infusion. Each single-dose vial contains 2.8×10 11 vg/0.5 mL (nominal concentration of 5.6×10 11 vg/mL) of KEBILIDI and each 2 mL vial contains an extractable volume of 0.5 mL. Following product thaw, the suspension for infusion is a clear to slightly opaque, colorless to faint white liquid, free of visible particulates [see How Supplied/Storage and Handling (16) ] . KEBILIDI is a suspension for intraputaminal administration with a nominal concentration of 5.6×10 11 vg/mL. ( 3 ) KEBILIDI is supplied in a single-dose vial that contains 2.8×10 11 vg of eladocagene exuparvovec-tneq in an extractable volume of 0.5 mL of suspension. Each mL of suspension contains 5.6×10 11 vg of eladocagene exuparvovec-tneq. ( 3 , 16 )

Contraindications

4 CONTRAINDICATIONS KEBILIDI is contraindicated in patients who have not achieved skull maturity assessed by neuroimaging. Skull maturity is needed for stereotactic neurosurgical administration of KEBILIDI. Patients who have not achieved skull maturity assessed by neuroimaging. ( 4 )

Warnings and precautions

Procedural complications: Monitor patients for procedural complications for neurosurgery, including events of respiratory and cardiac arrest after administration of KEBILIDI. ( 5.1 ) Dyskinesia: Monitor patients for dyskinesia after treatment with KEBILIDI. The use of dopamine antagonists can be used to control dyskinesia symptoms. ( 5.2 ) 5.1 Procedural Complications Procedural complications have been reported after neurosurgery required for KEBILIDI administration. These events included respiratory and cardiac arrest which occurred within 24 hours of the neurosurgical procedure and during post-surgical care [see Adverse Reactions (6) ] . KEBILIDI administration has the potential risk for additional procedure related adverse events including cerebrospinal fluid (CSF) leak, intracranial bleeding, neuroinflammation, acute infarction, and infection. Monitor patients for procedure related adverse events with KEBILIDI administration, including continuous cardiorespiratory monitoring during hospitalization. 5.2 Dyskinesia Dyskinesia was reported after administration of KEBILIDI. All events were reported within 3 months of administration and 2 events required hospitalization [see Adverse Reactions (6) ] . Monitor patients for signs and symptoms of dyskinesia after KEBILIDI treatment which may include involuntary movements of face, arm, leg, or entire body. These may present as fidgeting, writhing, wriggling, head bobbing or body swaying. The use of dopamine antagonists may be considered to control dyskinesia symptoms.

Side effects

Most common adverse reactions (incidence ≥15%) were dyskinesia, pyrexia, hypotension, anemia, salivary hypersecretion, hypokalemia, hypophosphatemia, insomnia, hypomagnesemia, and procedural complications. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact PTC Therapeutics, Inc at toll-free phone 1 866 562 4620 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch . 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. The safety data described in this section reflects exposure to KEBILIDI in 13 pediatric patients with genetically confirmed AADC deficiency who received a single dose of 1.8×10 11 vg. The median duration of follow-up was 72 weeks (range 23 to 109 weeks) [see Clinical Studies ( 14 )] . The most common adverse reactions (incidence ≥15%) are summarized in Table 3 . Table 3: Adverse Reactions in ≥15% of Patients in Study 1 * Procedural complications included respiratory and cardiac arrest. Adverse Reaction Patients Treated with KEBILIDI N=13 (%) Dyskinesia 10 (77%) Pyrexia 5 (38%) Hypotension 4 (31%) Anemia 4 (31%) Salivary hypersecretion 3 (23%) Hypokalemia 3 (23%) Hypophosphatemia 3 (23%) Insomnia 3 (23%) Hypomagnesemia 2 (15%) Procedural complications * 2 (15%) Other clinically significant adverse reaction includes worsening in duration and frequency of oculogyric crises during hospitalization following administration of KEBILIDI reported in one patient.

Use in specific populations

8.1 Pregnancy Risk Summary There are no clinical data from the use of KEBILIDI in pregnant women. Animal reproductive and developmental toxicity studies have not been conducted with KEBILIDI. In the US general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively. 8.2 Lactation Risk Summary There is no data on the presence of KEBILIDI in human milk, the effects on the breastfed infant, or the effects on milk production. 8.3 Females and Males of Reproductive Potential Pregnancy Testing Pregnancy status of females with reproductive potential should be verified. Sexually active females of reproductive potential should have a negative pregnancy test before administering KEBILIDI. Contraception There are insufficient exposure data to provide a recommendation concerning duration of contraception following treatment with KEBILIDI. Infertility There is no data on the effects of KEBILIDI on fertility. 8.4 Pediatric Use The safety and effectiveness of KEBILIDI have been established in pediatric patients. The use of KEBILIDI was evaluated in a single-arm, open-label study that enrolled 13 pediatric patients aged 16 months to 10 years who had achieved skull maturity [see Adverse Reactions ( 6 ) and Clinical Studies ( 14 )] . The safety and effectiveness of KEBILIDI have not been studied in pediatric patients younger than 16 months. 8.5 Geriatric Use KEBILIDI has not been studied in patients 65 years of age and older.

Pregnancy

Pediatric use

8.4 Pediatric Use The safety and effectiveness of KEBILIDI have been established in pediatric patients. The use of KEBILIDI was evaluated in a single-arm, open-label study that enrolled 13 pediatric patients aged 16 months to 10 years who had achieved skull maturity [see Adverse Reactions ( 6 ) and Clinical Studies ( 14 )] . The safety and effectiveness of KEBILIDI have not been studied in pediatric patients younger than 16 months.

Geriatric use

8.5 Geriatric Use KEBILIDI has not been studied in patients 65 years of age and older.

Description

(eladocagene exuparvovec-tneq) is a gene therapy product that expresses the human aromatic L-amino acid decarboxylase enzyme (hAADC). It is a recombinant adeno-associated virus serotype 2 (rAAV2) based vector containing the complementary DNA of the human DDC gene under the control of the cytomegalovirus immediate-early promoter. Eladocagene exuparvovec-tneq is produced in human embryonic kidney cells by recombinant DNA technology. KEBILIDI is a sterile suspension administered by bilateral intraputaminal infusion in one surgical session at two sites (anterior and posterior) per putamen. Each single-dose 2 mL vial contains 2.8×10 11 vg in an extractable volume of 0.5 mL of suspension. Each mL of suspension contains 5.6×10 11 vg. Patients will receive a total dose of 1.8×10 11 vg delivered as four 0.08 mL (0.45×10 11 vg) infusions (two per putamen). KEBILIDI is provided in a single-dose 2 mL vial containing a clear to slightly opaque, colorless to faint white liquid, free of visible particulates following thaw from its frozen state. The excipients include potassium chloride (3 mM), sodium chloride (337 mM), potassium dihydrogen phosphate (2 mM), disodium hydrogen phosphate (8 mM), and poloxamer 188 (0.001%).

Mechanism of action

12.1 Mechanism of Action KEBILIDI is a recombinant adeno-associated virus serotype 2 (rAAV2) based gene therapy designed to deliver a copy of the DDC gene which encodes the AADC enzyme. Intraputaminal infusion of KEBILIDI results in AADC enzyme expression and subsequent production of dopamine in the putamen.

How supplied

How Supplied KEBILIDI is supplied in a single-dose 2 mL vial containing sterile, clear to slightly opaque, colorless to faint white liquid free of visible particulates, following thaw from its frozen state. Each KEBILIDI (eladocagene exuparvovec-tneq) vial contains 2.8×10 11 vg of eladocagene exuparvovec-tneq in an extractable volume of 0.5 mL of suspension. Each mL of suspension contains a nominal concentration of 5.6×10 11 vg of eladocagene exuparvovec-tneq. Package (carton): NDC Number 52856-601-01 Container (vial): NDC Number 52856-601-11 Storage and Handling Store and transport frozen at ≤-65°C (-85°F). Keep the vial in the supplied carton. Thaw KEBILIDI prior to administration. If not used immediately, store at room temperature (up to 25°C [77°F]) and use within 6 hours of starting product thaw [see Dosage and Administration ( 2.3 )] . Do not refreeze vial once thawed.

Patient information

Discuss the following with patients and caregivers: Administration: Inform patients/caregivers that KEBILIDI administration involves an infusion into the brain that is administered during the neurosurgical procedure [see Administration 2.4 )] . Procedural Complications: Inform patients/caregivers about the complications of the neurosurgical procedure required for administration of KEBILIDI, including respiratory and cardiac arrest, cerebrospinal fluid (CSF) leak, intracranial bleeding, neuroinflammation, acute infarction, and infection [see Warnings and Precautions ( 5.1 )] . Dyskinesia: Inform patients/caregivers that they may experience dyskinesia within 3 months after treatment with KEBILIDI. Symptoms of dyskinesia may include involuntary movements of face, arm, leg, or entire body which may present as fidgeting, writhing, wriggling, head bobbing or body swaying. Advise patients and caregivers to contact their healthcare provider if these symptoms occur [see Warnings and Precautions ( 5.2 )] . Vector Shedding: Inform patients/caregivers that temporary vector shedding of KEBILIDI may occur for 3 weeks after administration. Advise patients/caregivers on proper hand hygiene and appropriate handling of waste materials generated from dressings and/or any secretions (e.g., blood, nasal secretions, urine, stool, and CSF). Recommended procedures include storage of waste material in sealed bags prior to disposal and wearing gloves for dressings changes and waste disposal. Patients should not donate blood, organs, tissues, or cells for transplantation [see Pharmacokinetics ( 12.3 )] . Manufactured by: PTC Therapeutics, Inc. Warren, NJ 07059 USA US License No. 2168

Label text from the FDA structured product label by PTC Therapeutics, Inc. (revised Jun 11, 2026). Long sections are shortened; the complete label is on DailyMed.

Active ingredients

Eladocagene Exuparvovec in 1 product

Kebilidi NDC products (1)

NDC	Strength & form	Labeler	Type
52856-601	Eladocagene Exuparvovec 560000000000 {GC}/mL Suspension	PTC Therapeutics, Inc.	BLA

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Kebilidi is listed by 1 labeler in the FDA NDC directory, including PTC Therapeutics, Inc..