KEBILIDI eladocagene exuparvovec-tneq 560000000000 {GC}/mL Suspension, 1 vial — NDC 52856-0601-01 package photo

KEBILIDI eladocagene exuparvovec-tneq 560000000000 {GC}/mL Suspension, 1 vial

by PTC Therapeutics, Inc. · 1 VIAL, GLASS in 1 CARTON (52856-601-01) / .5 mL in 1 VIAL, GLASS (52856-601-11)
NDC 52856-0601-01
🏷️ FDA NDC (as labeled) 52856-601-01 billing pads the product segment with a zero
Rx only Brand On market Non-controlled
🗂️ Data synced Jul 24, 2026 · sources: openFDA · FDA label (DailyMed) · FDA Orange & Purple Book · First Databank · CMS NADAC, ASP, Medicare & Medicaid · RxNorm
📋 All sources & update times →

🆔 Identity & classification

FDA NDC (as labeled) 52856-601-01
Product NDC 52856-601
11-digit billing NDC 52856060101
NCPDP billing unit ML — per mL (volume)
RxCUI 2698771, 2698778
UNII S51J6N56M7
UPC 0352856601017
Application # BLA125722
SPL Set ID 9d6a6401-c6b5-4f29-af11-67707d249482
DEA schedule Non-controlled
Marketing category BLA
Marketing status On market
FDA listing status Listed (active directory)
Marketing start 2024-11-13
Route INTRACEREBRAL
Dosage form SUSPENSION
Substance ELADOCAGENE EXUPARVOVEC
GPI-14 30902545101820
GCN Seq No 086743
GCN 56505
HICL code 050001
Ingredient (HICL) Eladocagene Exuparvovec-Tneq
HIC1 code Z
Therapeutic class — broad (HIC1) Body As A Whole
HIC2 code Z0
Therapeutic class — intermediate (HIC2) Biologicals (Continued 1)
HIC3 code Z0F
Therapeutic class — specific (HIC3) Gene Therapy Agents - Enzyme Deficiency
AHFS code 26:12.00.00
AHFS class Gene Therapy
FDB label name KEBILIDI VIAL
FDB brand name Kebilidi
Legend status F — Federal legend — prescription drug or device
Biologic (Purple Book) 351(a)
Why two NDCs? The FDA registers this code as 52856-601-01 — a 5-3-2 layout, and that's what's printed on the package and shown on DailyMed. For insurance claims, every NDC is standardized to a uniform 11-digit 5-4-2 format by adding a zero to the product segment → 52856-0601-01. Same drug, same package — only the format differs.
Where does this data come from?
Identifiers from the FDA openFDA NDC Directory and Structured Product Labeling; RxCUI from RxNorm (NLM); GPI from Medi-Span; GCN / HIC / AHFS / legend from First Databank.

🏷️ RxNorm drug class

This medicine belongs to the Enzymes class.

Drug family (ATC) Enzymes
Where does this data come from?
Therapeutic classes from RxNorm RxClass (U.S. National Library of Medicine) — Established Pharmacologic Class (FDA), ATC drug family (WHO) and mechanism of action, matched by this product’s RxCUI.

🏭 Manufacturer & labeler

LabelerPTC Therapeutics, Inc.
FDA applicationBLA125722 (BLA)
Labeler code52856
First marketedNov 2024
Product typeHuman Prescription Drug
Portfolio6 products on file
The labeler markets the product; the application holder owns the FDA approval. They’re often the same company but can differ (e.g. a repackager or an authorized generic). A mailing address / phone appears here when the manufacturer includes it in the product’s FDA label (not all do).
Where does this data come from?
Labeler, application holder and registered establishment from the FDA openFDA NDC Directory and Drugs@FDA; address/contact from the product’s FDA label.

🩺 Clinical

Label name KEBILIDI VIAL Ingredient Eladocagene Exuparvovec-Tneq
Where does this data come from?
Plain-language summary from MedlinePlus (U.S. National Library of Medicine); supplement & herbal interactions and nutrient depletion data from the Natural Medicines database; our full guide is HelloPharmacist editorial content.

💊 What it looks like

Color White
One label can cover several strengths, so colors may be combined — always confirm a loose pill against the dispensed prescription label or a pharmacist.
Where does this data come from?
Physical description (imprint, shape, color, scoring, coating) from this product’s FDA Structured Product Labeling (SPL), mirrored from DailyMed / openFDA.

🧪 Inactive Ingredients / Excipients

Inactive ingredients, also called excipients, are components of the drug product other than the active ingredient. They may include fillers, dyes, coatings, preservatives, flavors, or other formulation ingredients.

💡 Tap an ingredient (hover on desktop) to see what it is and why it’s used.

  • UNII 4J9FJ0HL51
    Potassium phosphate, monobasic is a salt compound used in medicines as a pH buffer and electrolyte source. It helps stabilize the acidity level of liquid and powdered formulations.
  • UNII LQA7B6G8JG
    A synthetic polymer made by combining water-soluble compounds. It acts as a surfactant and solubilizer to help mix oil and water-based ingredients, improving the medicine's texture and how active ingredients dissolve.
  • UNII 660YQ98I10
    Potassium chloride is a mineral salt used in medicines as a buffer and to help maintain the proper pH balance and stability of the formulation during storage and use.
  • UNII 451W47IQ8X
    Sodium chloride is common table salt. It's used in medicines as a buffer to maintain proper pH, as a filler to add bulk, or to adjust the osmotic balance in liquid formulations.
  • UNII 22ADO53M6F
    A mineral salt derived from phosphoric acid, used as a buffer to maintain the pH balance of the medication and help stabilize the active ingredients.
  • UNII 059QF0KO0R
    Water is a liquid solvent that dissolves and mixes ingredients together in liquid medicines, syrups, and injections. It helps distribute the active drug evenly throughout the product.

6 inactive ingredients listed in the exact product block matched to this NDC.

Where does this data come from?
Data sourced from official FDA Structured Product Labeling (SPL) via DailyMedingredient classCode="IACT" elements from the exact product block matched by this NDC. Label-section narrative from DailyMed / the openFDA label index is shown separately when available.

Inactive ingredient FAQ

Are inactive ingredients the same for every manufacturer?
No. Inactive ingredients can differ by manufacturer, dosage form, strength, and package / product version.
Why might an inactive ingredient be missing?
Some SPLs do not provide a complete structured inactive-ingredient list, and older or unusual labels may only include the information in narrative text.
Can inactive ingredients matter?
Yes. They can matter for allergies, intolerances, dyes, gluten / lactose concerns, preservatives, and formulation differences — but confirm with a pharmacist or the manufacturer when it’s clinically important.

💲 Pricing

A drug doesn't have one price. Each row is a different public payment system, and none is what you'd pay at the counter — that depends on your insurance. The ⓘ on each row explains what it measures.

Price systemPer mLPer package
Retail pharmacies payNADAC · weekly Not in the retail survey — common for institutional, discontinued, or low-volume packs.
Medicaid paysCMS SDUD · 12 mo No recent Medicaid claims on file for this NDC — rare and low-volume NDCs are suppressed in the public data.
Medicare drug plans payPart D · quarterly No Part D plan price is available for this NDC in our data.
ℹ️
No price is published for this exact package yet. CMS surveys NADAC per package size, so a different pack of the same drug often has one.
Where does this data come from?
NADAC (National Average Drug Acquisition Cost) is the CMS weekly pharmacy-acquisition-cost survey — what pharmacies pay. ASP (Average Sales Price) is the CMS Medicare Part B drug-payment file, published quarterly. Medicaid pays is computed by us from CMS State Drug Utilization Data (total reimbursed ÷ units, trailing 12 months) — gross of rebates and inclusive of dispensing fees, so it reflects what Medicaid paid, not an acquisition cost. Medicare drug plans pay is the median negotiated point-of-sale unit cost across plans listing this NDC in the CMS quarterly Prescription Drug Plan pricing files, before rebates. The VA pays is the federal contract price (FSS, and the statutory Big 4 ceiling where listed) from the VA National Acquisition Center pharmaceutical price file. All are free public government data; each measures a different payer, so the figures are not directly comparable.

🔁 Therapeutic equivalents

ProductLabelerPackNADAC/unitTEStatusPrice vs. this
Kebilidi 560000000000 {GC}/mLthis 52856-0601-01 PTC 1 vial FDA listed
About this product: this is a biologic. Biologics don't have small-molecule generics — competition comes from FDA-licensed biosimilars (shown above), not generics.
Where does this data come from?
Equivalents are other NDCs of the same ingredient, form and route from the openFDA NDC Directory, ranked least-expensive-first by NADAC. Therapeutic-equivalence (AB) ratings come from the FDA Orange Book; biologics use the FDA Purple Book for biosimilar & interchangeable status.

Availability & biosimilar status

🏛️
2024
First FDA approval
Nov 2024
📍
2026
Currently FDA-listed
2 years listed
🛡️
2036
Latest patent/protection listed
not a guaranteed launch date
🧬Biologic — competition comes from biosimilars

Biologics have no small-molecule generics; biosimilar competition is tracked in the FDA Purple Book.

🛡️ Latest patent/protection date listed: FDA patent/protection data lists protections through Nov 2036. This may affect when biosimilars become widely available, but it is not a guaranteed launch date.
📅 FDA approved Nov 13, 2024 ⏳ ~10.2 yr to latest listed protection

Why the date isn’t exact: Biosimilar timing can change because patents may be challenged, settled, licensed, added or removed, and litigation can move the real date earlier or later.

FDA Purple Book — biosimilars & interchangeables
🔒 No FDA-licensed biosimilars or interchangeable biosimilars are listed yet for this biologic. It currently has no biosimilar competition in the FDA Purple Book.
Source: FDA Purple Book (purplebooksearch.fda.gov), matched on the reference product’s active ingredient.
Patents & exclusivity — FDA Purple Book
Exclusivity RefProduct
2024 2026 2028 2030 2032 2034 2036
Today
LOE
Biologic patent Exclusivity
🏛️Reference-product exclusivity
A flat 12 years of FDA market protection from first licensure. No biosimilar can be licensed before it ends — regardless of patents.
🧪Listed biologic patents
Patents the reference maker lists covering the molecule, formulation, or manufacturing. A biosimilar generally can’t launch until these resolve.
🔁Interchangeability
An interchangeable biosimilar may be substituted at the pharmacy (state laws vary). The first one can earn its own exclusivity period.
🛈 What do these terms mean?
Biologic patent
A patent the reference product’s maker has publicly listed. A biosimilar generally can’t launch until these expire — unless they’re invalidated or resolved in a settlement.
Reference-product exclusivity
A flat 12 years of FDA market protection from the biologic’s first licensure (the BPCIA). No biosimilar can be licensed before it ends, regardless of patents.
Interchangeable exclusivity
The first interchangeable biosimilar can earn a period as the only interchangeable version (pharmacists can substitute it without the prescriber).
Earliest biosimilar (LOE)
The latest of all the dates above — the soonest a biosimilar can realistically reach the market. Litigation and settlements can move it earlier.

Biologics have no small-molecule “generics” — competition comes from FDA-licensed biosimilars, tracked in the FDA Purple Book.

FDA exclusivity
CodeWhat it grantsExpires
RefProductReference-product exclusivity (12-year, BPCIA) — no biosimilar can be licensed before this dateNov 13, 2036
Common questions
Is there a biosimilar for KEBILIDI VIAL?
No FDA-licensed biosimilar is currently listed for this biologic in the FDA Purple Book.
Why do different websites show different biosimilar dates?
Biosimilar availability isn’t based on one single date. Some sources use the reference-product exclusivity, some use the last listed patent, and patent litigation, settlements, and licenses can all change the real-world launch date. This page shows the underlying Purple Book dates so you can see why estimates differ.
Can a biosimilar launch before the last patent expires?
Sometimes. A biosimilar maker may settle with the reference manufacturer or receive a license to launch earlier. In other cases, the last listed protection delays competition.
What does “current Purple Book estimate” mean?
It means we’re using the latest patent and exclusivity dates currently listed in the FDA Purple Book. It is not a guaranteed launch date.
What does “FDA listed” mean?
It means the product appears in the FDA’s official directory. That’s a good sign a product exists for the U.S. market, but on its own it does not confirm a pharmacy can get it today. Where we have recent retail pricing data, we label it “Availability likely” instead.
What does a patent or protection date mean here?
It’s the latest date currently listed in the FDA Purple Book for a patent or exclusivity on the reference biologic. It can affect when a biosimilar becomes widely available — but it is not a guaranteed launch date. Settlements and licenses can move the real date earlier or later.
Built from the FDA Purple Book Patent List (patents the reference-product sponsor has publicly listed under the BPCIA) plus reference-product exclusivity. Biosimilars cannot launch until these clear; patent litigation and settlements can shift the real date. Biologics have no small-molecule generics — competition comes from FDA-licensed biosimilars, not the Orange Book.
Where does this data come from?
Patents and exclusivity from the FDA Purple Book (biologics), refreshed from public FDA data. Biosimilar launch timing is an estimate, not a guarantee.

🔬 Reported adverse events (FAERS)

Read carefully: FAERS reports are voluntary and unverified. Counts are not incidence, do not establish causation, are subject to reporting bias, and cannot be used to compare one drug to another. Shown for signal context only. Reports for KEBILIDI (this brand).

Top reported reactions

Anxiety1
Dyskinesia1
Inflammatory Marker Increased1
Procedural Headache1
Pyrexia1
Subdural Haematoma1

Reporter sex

3 reports
Male · 100%

Serious outcomes

Hospitalization1
Reports over time (by year) — tap or hover for the count & year
2025 2026 2 1
Most recent year is provisional (FAERS lags ~3 months).
Where does this data come from?
Adverse-event reports from the FDA Adverse Event Reporting System (FAERS) via openFDA. FAERS reports are voluntary and unverified — counts are not incidence and don’t establish causation.

📦 Packaging — all sizes for this product

Package NDCDescription Marketing startStatus
52856-0601-01 You're viewing this 1 VIAL, GLASS in 1 CARTON (52856-601-01) / .5 mL in 1 VIAL, GLASS (52856-601-11) 2024-11-13 Active

🧭 About this NDC listing & data coverage

Finished prescription product
What data is (and isn’t) available for this NDC — tap to expand
NDC identity (package / product / labeler codes) ✓ Available
Labeler ✓ Available
Product & package description ✓ Available
Marketing category & status ✓ Available
Active ingredient / dosage form / route ✓ Available
FDA label (SPL via DailyMed) ✓ Available
Package photos ✓ Available
Inactive ingredients (structured) ✓ Available
NADAC pharmacy acquisition price (CMS) — Not published for this NDC CMS publishes NADAC only for NDCs reported in its retail-pharmacy survey.
Orange Book / therapeutic-equivalence data ✓ Available
HCPCS J-code billing crosswalk — Not published for this NDC Most self-administered / retail products have no J-code — that is normal.
Medicaid utilization (CMS SDUD) — Not published for this NDC CMS reports utilization only for NDCs with Medicaid claims above its privacy threshold.
“Not published” reflects what the public FDA / CMS / NLM sources provide for this exact package code — it is a property of the data feeds, not a judgment about the product.

Questions about this listing

Why is there no price listed?
The pricing shown on our NDC pages comes from CMS NADAC, a voluntary survey of retail community pharmacy invoices. CMS does not publish a NADAC for every NDC — packages outside the retail survey (institutional and hospital products, bulk packages, discontinued items, and many OTC items) may never receive one. A missing price reflects the survey's scope, not this product's actual cost, and does not mean the product is free or unavailable.
Is the NDC printed on the package the same as the 11-digit billing NDC?
Yes, they identify this exact package in different formats. The FDA registers it as 52856-601-01, which is what is printed on the packaging and shown on DailyMed. Insurance claims use a fixed 11-digit 5-4-2 format, so the short segment is padded with a leading zero: 52856-0601-01, written without dashes as 52856060101. The Identity section at the top of this page lists every form of this code.
What do the three segments of this NDC mean?
In 52856-0601-01, the first segment (52856) is the labeler code FDA assigned to PTC Therapeutics, Inc.; the middle segment (0601) identifies this specific product — its ingredient, strength, and dosage form; and the last segment (01) identifies this exact package size and type. Together they name one specific package of one specific product.
Is this package still being marketed?
Yes, per the latest FDA NDC Directory data on this page: this package is listed as actively marketed, with no marketing end date reported by PTC Therapeutics, Inc.. Listing status can change — the directory data on this page refreshes weekly.
Who lists this product with the FDA?
PTC Therapeutics, Inc. is the labeler of record for this NDC — the company under whose FDA-assigned code the package is listed. The labeler may be the manufacturer itself or a distributor marketing the product under its own code.
Do I need a prescription for this product?
This NDC is listed with FDA as a prescription product, so it is dispensed under a prescriber's order. Your pharmacist can tell you whether any over-the-counter forms of the same medication exist.
This page identifies an FDA-listed package (the NDC) and reports public regulatory and pricing data about the listing. It is reference information, not a medical recommendation — talk to your pharmacist or prescriber about your own medication.
Where does this data come from?
Listing facts (marketing category, packager status, marketing dates) from the FDA openFDA NDC Directory; label availability from DailyMed; pricing coverage from CMS NADAC; equivalence scope from the FDA Orange Book.

📄 Full prescribing information FDA SPL

The complete FDA label for this product — the official prescribing information, verbatim, section by section. Jump with a chip, search within the label, or expand everything.
🎯 Indications and Usage 153 words

1 INDICATIONS AND USAGE KEBILIDI (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 ~3 min read

2 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 )

2.1Important 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.

2.2Recommended 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

2.3Preparation 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 syri…

💊 Dosage Forms and Strengths 129 words

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 39 words

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 Cautions 207 words

5 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 )

5.1Procedural 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.

5.2Dyskinesia 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.

🤒 Adverse Reactions ~1 min read

6 ADVERSE REACTIONS 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 .

6.1Clinical 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 ~1 min read

8 USE IN SPECIFIC POPULATIONS

8.1Pregnancy 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.

8.2Lactation 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.

8.3Females 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.

8.4Pediatric 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.

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

🤰 Pregnancy 57 words

8.1Pregnancy 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.

🧒 Pediatric Use 72 words

8.4Pediatric 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 16 words

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

🧬 Clinical Pharmacology ~3 min read

12 CLINICAL PHARMACOLOGY

12.1Mechanism 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.

12.2Pharmacodynamics Homovanillic Acid in Cerebrospinal Fluid In Study 1, homovanillic acid (HVA), a downstream metabolite of dopamine, in cerebrospinal fluid (CSF) was measured at baseline, Week 8, and Week 48 using a high-performance liquid chromatography with tandem mass spectrometry (HPLC-MS/MS). In all patients of Study 1, an increase in CSF HVA levels from baseline was observed at Week 8 and Week 48 ( Table 4 ). Table 4: HVA Levels in CSF (Study 1) Note: Lower limit of quantification (LLOQ) was 2 nmol/L, and values reported as <LLOQ were imputed as 0.5 * LLOQ.

Abbreviations: CSF=cerebrospinal fluid; HVA=homovanillic acid; N=number of subjects; Max=maximum; Min=minimum Timepoint Observed Values (nmol/L) Change from Baseline (nmol/L) Percent Change from Baseline(%) Baseline N 13 - - Median (Min, Max) 3.34 (1.00, 93.73) - - Week 8 N 12 12 12 Median (Min, Max) 35.09 (15.09, 150.48) 26.62 (12.49, 56.75) 534.7 (57.4, 2810.0) Week 48 N 9 9 9 Median (Min, Max) 29.16 (14.21, 125.84) 24.7 (13.21, 58.02) 773.1 (33.9, 3991.0) 18 F-DOPA Uptake in the Putamen 18 F-DOPA is a positron-emitting fluorine-labeled substrate of the AADC enzyme.

Following administration of 18 F-DOPA, its uptake into the putamen assessed by positron emission tomography (PET) imaging reflects AADC enzyme activity of dopaminergic neurons in the putamen. In Study 1, 18 F DOPA uptake in the putamen was assessed at baseline and followed up at Week 8 in 12 out 13 patients and at Week 48 in 10 out 13 patients indicating increased AADC 18 F DOPA uptake in all assessed patients. The median (range) percent increase from baseline was 259% (65% to 620%) at Week 8 and 271% (25% to 760%) at Week 48.

12.3Pharmacokinetics Biodistribution (within the body) and Vector Shedding (excretion/secretion) KEBILIDI vector DNA levels in various tissues and secretions were determined using a validated quantitative polymerase chain reaction (qPCR) assay. Nonclinical data Biodistribution of eladocagene exuparvovec-tneq was evaluated in rats at Days 7, 30, 90, and 180 after single-dose intraputaminal infusion at dose levels up to 7.5×10 9 vg/animal (21-fold higher than the recommended human dose based on relative brain weight).

At Day 7, vector DNA was observed in the putamen, cerebellum, cerebrum, and spinal cord. Vector DNA levels declined from Day 7 to Day 90, with DNA levels primarily sustained in the putamen at Day 180. Clinical data Following administration of KEBILIDI at a total dose for each patient of 1.8×10 11 vg in Study 1, biodistribution and viral shedding in CSF, blood, and urine were evaluated in 13 patients.

CSF was collected at Weeks 8 and 48, and blood and urine were collected from Day 3 up to Week 48. Five (38%) patients showed detectable vector DNA levels in blood at Day 3 ranging from 4.0×103 to 6.5×10 3 copies/mL, which became below the limit of detection (<3.1×10 3 copies/mL) by Week 3. No vector was detected in CSF or urine.

12.6Immunogenicity The observed incidence of anti-AAV2 antibodies is highly dependent on the sensitivity and specificity of the assay. Differences in assay methods preclude meaningful comparisons of the incidence of anti-AAV2 antibodies in the studies described below with the incidence of anti-AAV2 antibodies in other studies. There is no clinical experience with KEBILIDI in patients with pre-existing anti-AAV2 neutralizing antibody at titers >1:1200.

In Study 1, anti-AAV2 total binding antibodies and anti-AAV2 neutralizing antibodies were assessed from Day 3 up to Week 48 following administration of KEBILIDI. In all patients (N=13), titers of total binding antibody and neutralizing antibody increased from Week 3 a…

🧬 Mechanism of Action 47 words

12.1Mechanism 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 / Storage and Handling 140 words

16 HOW SUPPLIED/STORAGE AND HANDLING 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.

📋 Description 186 words

11 DESCRIPTION KEBILIDI (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%).

💬 Information for Patients ~1 min read

17 PATIENT COUNSELING 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.

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Source: FDA Structured Product Labeling, mirrored from DailyMed / openFDA. Prefer the government’s original formatting? View this label on DailyMed ↗
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