Otarmeni

Lunsotogene parvec-cwha · Injection, Solution · Intracochlear

Prescription (Rx)

Uses

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

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 ) 2.1 Critical 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. 2.2 Recommended 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. 2.3 Preparation 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 for bubbles. If there are bubbles, gently tap the syringe with your finger until the bubbles rise to the top. Remove the needle and prime the air bubbles out of the syringe by depressing the plunger rod until the air is fully expelled and the meniscus is visible at the syringe opening. Cap and label the syringe with the following: Lot number of OTARMENI Expiration date of OTARMENI Time of preparation to ensure that product is administered within 4 hours after puncturing the vial Place the capped syringe into the sterile plastic bag and seal the bag. Place the bag into an appropriate secondary container for delivery to the surgical suite. Figure 1 Chemical Structure 2.4 Administration Administer OTARMENI in the surgical suite under general anesthesia with controlled aseptic conditions by a surgeon experienced in intracochlear surgery and trained in the OTARMENI administration procedure. Administer OTARMENI within 4 hours of puncturing the vial. The following items are required for administration: Syringe containing OTARMENI Catheter supplied with Administration Kit for use with OTARMENI (package 2 of 2) [see How Supplied/Storage and Handling (16) ] Not supplied but required: Syringe pump, capable of delivering a volume of 0.24 mL at an infusion rate of 0.9 mL/hr and is compatible with the provided 1 mL or 3 mL syringe sizes Electrode insertion forceps Fine straight pick Mayo stand Note: Only administer OTARMENI using the provided Administration Kit for use with OTARMENI . Confirm the availability of OTARMENI (1 vial and 1 Administration Kit per treated ear) prior to administration of anesthesia. Inspect each item prior to use and do not use if any damage is identified. Do not use if the sterile bag was damaged during transport or if the syringe was transported without the use of a sterile bag. Follow the steps below for intracochlear infusion: Perform a standard mastoidectomy and an opening of the facial recess (also called a posterior tympanotomy).

Dosage forms and strengths

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

None. None. ( 4 )

Warnings and precautions

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

Side effects

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 . 6.1 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 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

8.1 Pregnancy 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. 8.2 Lactation 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. 8.3 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 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. 8.4 Pediatric 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) ] . 8.5 Geriatric Use OTARMENI has not been studied in patients 65 years of age and older.

Pregnancy

Pediatric use

8.4 Pediatric 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

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

Description

(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.

Mechanism of action

12.1 Mechanism 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

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

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.

Patient 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) ].

Label text from the FDA structured product label by Regeneron Pharmaceuticals, Inc. (revised Apr 27, 2026). Long sections are shortened; the complete label is on DailyMed.

Active ingredients

Lunsotogene Parvec in 1 product

Otarmeni NDC products (1)

NDC	Strength & form	Labeler	Type
61755-062	Lunsotogene Parvec 30000000000000 {GC}/mL Injection, Solution	Regeneron Pharmaceuticals, Inc.	BLA

Frequently asked questions

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Otarmeni is listed by 1 labeler in the FDA NDC directory, including Regeneron Pharmaceuticals, Inc..