HomeNDC LookupIngredientsLunsotogene Parvec-Cwha › 61755-0062-99
OTARMENI lunsotogene parvec-cwha 30000000000000 {GC}/mL Injection, Solution, 1 vial — NDC 61755-0062-99 package photo

OTARMENI lunsotogene parvec-cwha 30000000000000 {GC}/mL Injection, Solution, 1 vial

by Regeneron Pharmaceuticals, Inc. · 1 CARTON in 1 BAG (61755-062-99) / 1 VIAL, SINGLE-DOSE in 1 CARTON (61755-062-01) / 1 mL in 1 VIAL, SINGLE-DOSE (61755-062-00)
NDC 61755-0062-99
🏷️ FDA NDC (as labeled) 61755-062-99 billing pads the product segment with a zero
Rx only Brand On market Non-controlled
🗂️ Data synced Aug 20, 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) 61755-062-99
Product NDC 61755-062
11-digit billing NDC 61755006299
RxCUI 2741622, 2741629
UNII P6ZNU67HPU
Application # BLA125874
SPL Set ID a7a5e936-ab4a-4609-ac10-96efedc89035
DEA schedule Non-controlled
Marketing category BLA
Marketing status On market
FDA listing status Listed (active directory)
Marketing start 2026-04-23
Route INTRACOCHLEAR
Dosage form INJECTION, SOLUTION
Substance LUNSOTOGENE PARVEC
GCN Seq No 088880
GCN 59063
HICL code 051281
Ingredient (HICL) Lunsotogene Parvec-Cwha
HIC1 code Q
Therapeutic class — broad (HIC1) Ear/Eye/Nose/Rectum/Topical/Vagina/Other
HIC2 code Q1
Therapeutic class — intermediate (HIC2) Drugs Acting On The Ear
HIC3 code Q1B
Therapeutic class — specific (HIC3) Otic Cell/Gene Therapy Agents
AHFS code 26:12.00.00
AHFS class Gene Therapy
FDB label name OTARMENI VIAL
FDB brand name Otarmeni
Legend status F — Federal legend — prescription drug or device
Why two NDCs? The FDA registers this code as 61755-062-99 — 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 → 61755-0062-99. 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.

🏭 Manufacturer & labeler

LabelerRegeneron Pharmaceuticals, Inc.
FDA applicationBLA125874 (BLA)
Labeler code61755
First marketedApr 2026
Product typeHuman Prescription Drug
Portfolio27 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 OTARMENI VIAL Ingredient Lunsotogene Parvec-Cwha
📖 What it is MedlinePlus · NLM

Lunsotogene is used to treat certain types of inherited hearing loss. Lunsotogene is in a class of medications called gene therapy. It works by helping to produce a protein to restore function of a nerve that is necessary for hearing.

Read the full MedlinePlus article ↗
📗 Our plain-language guide HelloPharmacist
  • Otarmeni delivers a working copy of the OTOF gene directly into your inner ear so your hearing cells can finally make the protein they were missing. In the clinical trial, many pat...
  • What exactly does this treatment do — is it a cure?
  • Yes — Otarmeni is given through a surgical procedure under general anesthesia, so you'll be asleep for it. A specially trained surgeon makes a small opening to access your inner ea...
  • Is this a surgery? What will the procedure be like?
📖 Read our full Lunsotogene Parvec-Cwha guide →
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.

🧪 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 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 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 70WT22SF4B
    A mineral salt that acts as a buffer to maintain the pH balance of the medication. It helps stabilize the drug's potency and prevents unwanted chemical changes during storage.
  • UNII 593YOG76RN
    A salt made from sodium and phosphate that helps regulate acidity and maintain proper pH balance in the medicine. It also acts as a buffer to keep the product stable.
  • UNII C151H8M554
    A natural sugar derived from sugar cane or sugar beets. It's used as a sweetener, filler, and binder to improve taste, add bulk, and help hold tablet or capsule ingredients together.
  • 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
Otarmeni 30000000000000 {GC}/mLthis 61755-0062-99 Regeneron 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

🏛️
2026
On the market since
Apr 2026
📍
2026
Currently FDA-listed
listed with the FDA
🧬
·
Biosimilars
see Purple Book
🧬Biologic — competition comes from biosimilars

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

📦 Packaging — all sizes for this product

Package NDCDescription Marketing startStatus
61755-0062-99 You're viewing this 1 CARTON in 1 BAG (61755-062-99) / 1 VIAL, SINGLE-DOSE in 1 CARTON (61755-062-01) / 1 mL in 1 VIAL, SINGLE-DOSE (61755-062-00) 2026-04-23 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 — Not published for this NDC Applies only to products approved under an NDA/ANDA; many listings are out of scope.
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 61755-062-99, 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: 61755-0062-99, written without dashes as 61755006299. 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 61755-0062-99, the first segment (61755) is the labeler code FDA assigned to Regeneron Pharmaceuticals, Inc.; the middle segment (0062) identifies this specific product — its ingredient, strength, and dosage form; and the last segment (99) 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 Regeneron Pharmaceuticals, Inc.. Listing status can change — the directory data on this page refreshes weekly.
Who lists this product with the FDA?
Regeneron Pharmaceuticals, 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 ~1 min read

1 INDICATIONS AND USAGE OTARMENI is indicated for the treatment of pediatric and adult patients with severe-to-profound and profound sensorineural hearing loss (any frequency >90 dB HL) associated with molecularly confirmed biallelic variants in the OTOF gene, preserved outer hair cell function, and no prior cochlear implant in the same ear. This indication is approved under accelerated approval based on the improvement of hearing sensitivity assessed by average pure tone audiometry (PTA) at Week 24 [see Clinical Studies (14) ] .

Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory clinical trial. Limitations of Use OTARMENI is not recommended in patients in whom preoperative imaging demonstrates that access to the inner ear is not feasible including those with abnormal mastoid pneumatization or clinically significant anatomic variations of the middle ear and inner ear. OTARMENI is an adeno-associated virus vector-based gene therapy indicated for the treatment of pediatric and adult patients with severe-to-profound and profound sensorineural hearing loss (any frequency >90 dB HL) associated with molecularly confirmed biallelic variants in the OTOF gene, preserved outer hair cell function, and no prior cochlear implant in the same ear.

( 1 ) This indication is approved under accelerated approval based on the improvement of hearing sensitivity assessed by average pure tone audiometry (PTA) at Week 24. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory clinical trial. ( 1 , 14 ) Limitations of Use OTARMENI is not recommended in patients in whom preoperative imaging demonstrates that access to the inner ear is not feasible including those with abnormal mastoid pneumatization or clinically significant anatomic variations of the middle ear and inner ear.

( 1 )

⏱️ Dosage and Administration ~3 min read

2 DOSAGE AND ADMINISTRATION For intracochlear infusion only. Recommended dose of OTARMENI for each ear is 7.2 × 10 12 vector genomes (vg) in a total volume of 0.24 mL, administered by a single-dose intracochlear infusion. ( 2.2 )

2.1Critical Dosing and Administration Information Confirm patient has biallelic, likely pathogenic or pathogenic OTOF variants. OTARMENI should be administered by a surgeon experienced in intracochlear surgery and trained in the administration procedure. Administer bilateral OTARMENI, if applicable, in a single surgical session.

For prophylaxis against inflammatory and immunological responses, administer systemic oral corticosteroids equivalent in strength to prednisone at 1 mg/kg/day the day of the OTARMENI infusion, maintain the same dose for two weeks, and taper over the following two weeks. The total corticosteroid dose should not exceed 60 mg/day. For prophylaxis against post-operative infection, administer antibiotics before incision.

Administer age-appropriate vaccination prior to treatment with OTARMENI. Administer vaccines at least 1 month before the first corticosteroid dose and at least 1 month after the last dose. Vaccinate patients against micro-organisms that cause meningitis before surgery as meningitis is a known risk of inner ear surgery.

2.2Recommended Dosage For intracochlear infusion only. The recommended dose of OTARMENI for each ear is 7.2 × 10 12 vector genomes (vg) in a total volume of 0.24 mL, administered by a single-dose intracochlear infusion.

2.3Preparation Prepare syringe containing OTARMENI using sterile technique under aseptic conditions in a Class II biological safety cabinet (BSC). Administer OTARMENI within 4 hours of puncturing the vial. Below is the list of items required for administration syringe preparation.

Confirm component packaging and inspect each item prior to use and do not use if any damage is identified. One single-dose vial of OTARMENI in a sealed barrier bag (package 1 of 2) [see How Supplied/Storage and Handling (16) ] Administration Kit for use with OTARMENI (package 2 of 2) [see How Supplied/Storage and Handling (16) ] Not supplied but required: One isopropyl alcohol pad One sterile sealable plastic bag Secondary container for delivery to surgical suite Label and a pen Preparation of OTARMENI for Infusion For each treated ear, thaw one single-dose vial of OTARMENI at room temperature.

The contents of the vial will thaw in approximately 30 minutes at room temperature. DO NOT thaw or warm the vial any other way. Gently invert the vial 5 times (DO NOT shake or vortex the vial).

Inspect the OTARMENI vial to ensure that it is thawed, clear to slightly opalescent, and that the vial has no signs of damage. If there is any evidence of cloudiness, particulates, or color, do not use the vial. If not used immediately, store the unpunctured thawed vial in the refrigerator for up to 24 hours or at room temperature for up to 8 hours.

Choose the smallest compatible syringe size, either 1 mL or 3 mL included in the kit, according to the syringe pump parameters. Discard the unused syringe. Do not open the catheter sterile packaging until transferred to the surgical suite.

Prepare the septum of the OTARMENI vial using an isopropyl alcohol pad to wipe the top. Wait for the septum to dry. Secure the 21G, 1.5-inch needle onto the 1 mL or 3 mL syringe.

When filling the syringe for administration, push the needle until it is completely inserted into the vial and the tip touches the bottom edge of the upright vial. Administer OTARMENI within 4 hours of puncturing the vial. Withdraw all OTARMENI vial contents into the syringe.

Tilt the vial to ensure complete withdrawal. Confirm that at least 0.6 mL is available in the syringe. To prevent the introduction of air, keep the needle bevel submerged in the liquid at the bottom edge of the vial by orienting the bevel towards the vial wall (Figure 1).

Figure 1: Holding the syringe with the needle pointing up, check the syringe…

💊 Dosage Forms and Strengths 100 words

3 DOSAGE FORMS AND STRENGTHS OTARMENI is a sterile suspension for intracochlear infusion. Each single-dose vial contains a nominal titer of 3.0 × 10 13 vg/mL of OTARMENI and has an extractable volume of 0.63 mL. Following product thaw, the suspension for infusion is a clear to slightly opalescent, colorless liquid, free of visible particulates [see How Supplied/Storage and Handling (16) ] .

OTARMENI is a sterile suspension for intracochlear infusion. ( 3 ) Each single-dose vial contains a nominal titer of 3.0 × 10 13 vg/mL of OTARMENI and has an extractable volume of 0.63 mL. ( 3 )

Contraindications 7 words

4 CONTRAINDICATIONS None. None. ( 4 )

⚠️ Warnings and Cautions 72 words

5 WARNINGS AND PRECAUTIONS Procedure-Related Risks: Monitor patients for procedure-related adverse reactions with OTARMENI administration. ( 5.1 )

5.1Procedure-Related Risks Procedure-related adverse reactions may occur with OTARMENI administration including vertigo, tinnitus, cerebral spinal fluid leak, ipsilateral facial paresis, ipsilateral change in taste, meningitis, wound infection, mastoiditis, numbness around the ear, blood or fluid collection at surgical site, and labyrinthitis. Monitor patients for procedure-related adverse reactions with OTARMENI administration and manage accordingly.

🤒 Adverse Reactions ~1 min read

6 ADVERSE REACTIONS The most common adverse reactions (incidence ≥5%) include otitis media, vomiting, nausea, dizziness, procedural pain, gait disturbance, and nystagmus. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Regeneron at 1-866-500-GENE 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 reflect exposure to OTARMENI in one study (Study DB-OTO-001) which treated 24 patients with OTOF gene associated profound sensorineural hearing loss. A total of 10 patients received unilateral dosing, and a total of 14 patients received bilateral dosing of OTARMENI at a nominal dose of 7.2 × 10 12 vg per ear.

The median duration of follow-up was 45 weeks (range 9 to 115 weeks) [see Clinical Studies (14) ] . Table 1 lists the most common adverse reactions that occurred in ≥ 5% patients in Study DB-OTO-001. Table 1: Adverse Reactions Occurring in ≥5% of Patients in Study DB-OTO-001 (N=24) Adverse Reaction Overall n (%) Otitis Media 9 (38) Vomiting 8 (33) Nausea 7 (29) Dizziness 5 (21) Procedural Pain 4 (17) Gait Disturbance 2 (8) Nystagmus 2 (8) Other clinically significant adverse reactions each occurring in 1 patient included transient balance disorder (4%), abnormal otoacoustic emissions (4%), and wound dehiscence (4%).

👥 Use in Specific Populations ~1 min read

8 USE IN SPECIFIC POPULATIONS

8.1Pregnancy Risk Summary There are no data from the use of OTARMENI in pregnant women. Animal reproductive and developmental toxicity studies have not been conducted with OTARMENI. 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 are no data on the presence of OTARMENI 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 OTARMENI. Contraception There are insufficient exposure data to provide a recommendation concerning duration of contraception following treatment with OTARMENI.

Infertility There are no data on the effects of OTARMENI on fertility.

8.4Pediatric Use The safety and effectiveness of OTARMENI have been established in pediatric patients with OTOF gene associated profound sensorineural hearing loss. The use of OTARMENI was supported by evidence from one clinical study that treated 24 pediatric patients aged 10 months to 16 years [see Adverse Reactions (6) and Clinical Studies (14) ] .

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

🤰 Pregnancy 56 words

8.1Pregnancy Risk Summary There are no data from the use of OTARMENI in pregnant women. Animal reproductive and developmental toxicity studies have not been conducted with OTARMENI. 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 56 words

8.4Pediatric Use The safety and effectiveness of OTARMENI have been established in pediatric patients with OTOF gene associated profound sensorineural hearing loss. The use of OTARMENI was supported by evidence from one clinical study that treated 24 pediatric patients aged 10 months to 16 years [see Adverse Reactions (6) and Clinical Studies (14) ] .

🧓 Geriatric Use 16 words

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

🧬 Clinical Pharmacology ~3 min read

12 CLINICAL PHARMACOLOGY

12.1Mechanism of Action Intracochlear infusion of OTARMENI results in expression of the OTOF gene primarily in inner hair cells (IHCs), which facilitates production of functional otoferlin protein and restores synaptic transmission to the auditory nerve as shown in animal studies in mice.

12.2Pharmacodynamics There are no pharmacodynamics data for OTARMENI.

12.3Pharmacokinetics Biodistribution (within the body) and Vector Shedding (excretion/secretion) OTARMENI vector DNA levels in various matrices and nonclinical tissues were determined using a qualified quantitative polymerase chain reaction (qPCR) assay. Expression of human OTOF mRNA was determined in selected nonclinical tissues. Nonclinical data Systemic distribution of vector DNA was evaluated in cynomolgus monkeys at weeks 7 and 27 after a single bilateral intracochlear infusion of dose levels up to 4.4 × 10 12 vg/ear (7.3 × 10 13 vg/mL), and in juvenile mice at days 30 and 90 and in adult mice at days 30 and 60 after a single unilateral inner ear infusion of dose levels up to 1.3 × 10 11 vg/ear (6.5 × 10 13 vg/mL).

The highest levels of vector DNA were detected in the cochleae. Human OTOF mRNA expression in the cochleae was sustained over the study durations (27 weeks in monkeys, 60-90 days in mice), with peak expression at 6 weeks in monkeys and 4 weeks in mice, then plateauing through the final time points. Vector DNA distribution to non-otic tissues was limited, with levels at least 10-fold lower than cochlear levels in monkeys and at least 3-fold lower in mice.

The highest levels of OTARMENI vector DNA were detected in the parotid lymph node, cervical dorsal root ganglion, cervical spinal cord, and brain, while sporadic or low levels were detected in other non-otic tissues. No measurable vector DNA was detected in ovaries, and only minimal levels were detected in testes (1 of 4 mouse samples near detection limit). Human OTOF mRNA expression in non-otic tissues was restricted to neural tissues at substantially lower levels than in the cochlea.

In monkeys, human OTOF mRNA was detected only in cervical and lumbar dorsal root ganglia of one monkey at Week 7 (no detection at Week 27). In mice, human OTOF mRNA was detected in brain, cervical dorsal root ganglia, and cervical spinal cord at levels 4.9- to 96.4-fold lower than cochlear expression. No human OTOF mRNA was detected in reproductive tissues or other non-neural tissues in either species.

Clinical data Systemic distribution of vector DNA in plasma and vector DNA shedding in urine, saliva, and stool were evaluated in 15 patients administered OTARMENI at the dose of 7.2 × 10 12 vg/ear in Study DB-OTO-001. Samples were collected at baseline, at Day 1, and Week 2, 4, 6, 12, 24, and 36 following OTARMENI administration. Vector DNA was detectable in plasma, urine, and saliva at Day 1 after OTARMENI administration and was undetectable by Week 2 (the next sampling timepoint).

In stool, vector DNA was detectable through Week 6 and became undetectable by Week 12. Peak concentration was observed at Day 1 after OTARMENI administration and declined substantially thereafter.

12.6Immunogenicity The observed incidence of anti-AAV1 neutralizing antibodies and anti-OTOF antibodies is highly dependent on the sensitivity and specificity of the assays. Differences in assay methods preclude meaningful comparisons of the incidence of antibodies in the study described below with the incidence of antibodies in other studies. In the DB-OTO-001 study, patients were not excluded based on pre-existing serum anti-AAV1 neutralizing antibodies.

Immunogenicity was assessed from baseline through Week 12. A total of 12/15 (80%) patients had anti-AAV1 neutralizing antibodies at baseline (titer <1,000). All patients developed anti-AAV1 neutralizing antibody responses through Week 12 with 10/15 (67%) having titer >10,000, 3/15 (20%) having titer 1,000 to 10,000, and 2/15 (13%) having titer <1,000 responses.

A total of 1/1…

🧬 Mechanism of Action 43 words

12.1Mechanism of Action Intracochlear infusion of OTARMENI results in expression of the OTOF gene primarily in inner hair cells (IHCs), which facilitates production of functional otoferlin protein and restores synaptic transmission to the auditory nerve as shown in animal studies in mice.

📦 How Supplied / Storage and Handling ~1 min read

16 HOW SUPPLIED/STORAGE AND HANDLING How Supplied OTARMENI is supplied as a single-dose vial enclosed in a sealed barrier bag. Following thaw from its frozen state, OTARMENI is a sterile, clear to slightly opalescent colorless liquid, free of visible particulates. Each vial contains a nominal titer of 3.0 × 10 13 vg/mL of OTARMENI and has an extractable volume of 0.63 mL.

The Administration Kit for use with OTARMENI is supplied separately as package 2 of 2 [ see Dosage and Administration (2.3 and 2.4) ]. Contents of Package 1 NDC Number OTARMENI 2 mL vial 61755-062-00 OTARMENI vial carton 61755-062-01 OTARMENI sealed barrier bag 61755-062-99 Contents of Package 2 NDC Number Administration Kit for use with OTARMENI One BD PrecisionGlide™ 21G, 1.5-inch sterile needle One BD 1 mL sterile syringe, Luer-Lok™ tip One BD PlastiPak™ 3 mL sterile syringe, Luer-Lok™ tip One BD Luer-Lok™ syringe tip cap One Vygon Premicath ® catheter 61755-062-11 Storage and Handling OTARMENI suspension (2 mL vial): Store and transport OTARMENI (package 1) frozen at -80°C (-112°F).

Keep the vial in the supplied carton. Thaw OTARMENI at room temperature prior to administration. If not used immediately, the unpunctured thawed vial can be stored in the refrigerator (2°C to 8°C [36°F to 46°F]) for up to 24 hours or at room temperature (up to 25°C [77°F]) for up to 8 hours [ see Dosage and Administration (2.3) ].

Do not refreeze vial once thawed. Administration Kit for use with OTARMENI: Store at room temperature in original carton separately from OTARMENI until time of use. Do not freeze.

Avoid excessive heat and direct sunlight.

📦 Storage and Handling 113 words

Storage and Handling OTARMENI suspension (2 mL vial): Store and transport OTARMENI (package 1) frozen at -80°C (-112°F). Keep the vial in the supplied carton. Thaw OTARMENI at room temperature prior to administration.

If not used immediately, the unpunctured thawed vial can be stored in the refrigerator (2°C to 8°C [36°F to 46°F]) for up to 24 hours or at room temperature (up to 25°C [77°F]) for up to 8 hours [ see Dosage and Administration (2.3) ]. Do not refreeze vial once thawed. Administration Kit for use with OTARMENI: Store at room temperature in original carton separately from OTARMENI until time of use.

Do not freeze. Avoid excessive heat and direct sunlight.

📋 Description 184 words

11 DESCRIPTION OTARMENI (lunsotogene parvec-cwha) is an adeno-associated virus vector-based gene therapy for intracochlear infusion. OTARMENI contains a dual adeno-associated virus serotype 1 vector (AAV1) that utilizes an engineered hair cell-specific promoter derived from regulatory elements of myosin 15 ( Myo15 ) to drive complementary DNA ( cDNA ) expression of human OTOF transcript variant 5 encoding isoform e of the otoferlin protein (OTOF). The vector is produced in human embryonic kidney cells by recombinant DNA technology.

OTARMENI is a sterile, aqueous suspension administered by intracochlear infusion. Each single-dose vial contains a nominal vector genome titer of 3.0 × 10 13 vg/mL in an extractable volume of 0.63 mL. Patients receive a total nominal dose of 7.2 × 10 12 vg per ear in a total infused volume of 0.24 mL per ear.

OTARMENI is provided in a single-dose 2 mL vial containing a clear to slightly opalescent, colorless suspension, free of visible particulates following thaw from its frozen state. The excipients include sodium phosphate (10 mM), sodium chloride (180 mM), sucrose (5% w/v), and poloxamer 188 (0.001% w/v) with a pH of 7.3.

💬 Information for Patients 202 words

17 PATIENT COUNSELING INFORMATION Inform patients or caregivers that: Prior to treatment with OTARMENI, it is recommended that patients receive the appropriate vaccinations for their age. Vaccines should be administered at least 1 month before the first corticosteroid dose and at least 1 month after the last dose [ see Dosage and Administration (2.1) ]. Before surgery, patients should consult their primary care physician and surgeon regarding vaccination status against microorganisms that cause meningitis.

Meningitis is a known risk of inner ear surgery [ see Dosage and Administration (2.1) ]. Procedural-related adverse reactions may occur following OTARMENI administration. These may include vertigo, tinnitus, cerebral spinal fluid leak, ipsilateral facial paresis, ipsilateral change in taste, meningitis, wound infection, mastoiditis, numbness around the ear, blood or fluid collection at surgical site, and labyrinthitis. [ see Warnings and Precautions (5.1) ].

Temporary vector shedding of OTARMENI occurs primarily through body waste. Practice proper hand hygiene, such as hand washing, when coming into direct contact with patient body waste. Place potentially contaminated materials that may have the patient's bodily fluids/waste in a sealable bag and dispose into regular trash.

These precautions should be followed for 2 weeks after OTARMENI infusion [ see Clinical Pharmacology (12.3) ].

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