Encelto (revakinagene taroretcel-lwey) Implant
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Payer drug policy governing coverage and utilization management for the Encelto implant for treatment of idiopathic Macular Telangiectasia Type 2 across Univera Healthcare lines of business; affects prescribing ophthalmologists and network prior-authorization reviewers.
No material clinical or coverage changes in this revision.
Coverage Criteria
inv-01: Commercial/Essential/Child Health Plus - Not Medically Necessary
Commercial/Essential/Child Health Plus
Refer to Corporate Medical Policy #11.01.03 Experimental or Investigational Services
inv-02: Medicare Advantage/Medicaid/HARP - Coverage Criteria — Covered when ALL of the following are met
Medicare Advantage/Medicaid/HARP - Covered when ALL of the following are met
Approval duration 6 months
inv-03: General Gene/Cellular Therapy Coverage Constraints — Covered when ALL of the following are met (policy-level constraints)
Covered when ALL of the following are met (policy-level constraints):
Most gene/cellular therapies evaluated as single-administration interventions
Prior exposure generally makes patient ineligible
Safety, efficacy, and durability of repeat or sequential administration not established; risks include immune activation, insertional mutagenesis, and vector immunity
Coverage exclusions: Encelto will not be authorized for patients with Macular Telangiectasia (MacTel) Type 1. The product is not authorized for any non‑FDA approved indication. In addition, coverage is limited to one implant per eye per lifetime; authorizations are issued for up to 6 months to allow the procedure to occur.
Retreatment, repeat administration, and sequential use of additional or different gene/cellular therapies are discouraged and treated as experimental. The policy states that most gene and cellular therapies are designed and studied as a one‑time administration, and repeat dosing, reinfusion, or sequential therapy has not been established as safe or effective. Prior exposure to any gene or cellular therapy (autologous, allogeneic, in vivo gene therapy, or ex vivo gene‑modified cell product) generally makes a patient ineligible for further gene/cellular therapy unless there is documented evidence supporting safety and anticipated benefit. For Encelto specifically, requests that propose retreatment or administration after prior gene/cellular therapy exposure risk denial.
Rationale for Commercial/Essential/Child Health Plus stance: based on review of the peer‑reviewed literature, the evidence for Encelto is judged inconclusive with respect to meaningful clinical benefit. The policy lists insufficient evidence that the therapy produces consistent positive health outcomes, durable improvement over time, or benefit in routine clinical practice; therefore, use of Encelto is considered not medically necessary under these commercial lines and will be denied per Corporate Medical Policy #11.01.03.
Retreatment with the same gene or cellular therapy product is explicitly classified as experimental and investigational and therefore not medically necessary. The policy explains that clinical trials evaluated these products as single‑administration interventions, and the safety, efficacy, and durability of a second administration have not been established; repeat dosing may increase risks such as immune activation, insertional mutagenesis, or vector immunity.
Initial Therapy Criteria
inv-25: Initial therapy criteria — Initial authorization for Medicare Advantage/Medicaid/HARP
Initial authorization for Medicare Advantage/Medicaid/HARP
Limited to one implant per eye per lifetime; approval for 6 months
inv-26: Initial Therapy — initial administration guidance and requirements
Initial administration guidance and requirements
Most gene and cellular therapies are designed and studied as one-time treatments
Continuation / Retreatment Criteria
inv-27: Retreatment / Continuation Therapy — continuation/retreatment guidance
Continuation/retreatment guidance
Safety, efficacy, and durability of a second administration have not been established
Lack of evidence for anticipated clinical benefit, safety of sequential administration, or justification for selecting a second gene/cellular intervention
Prior gene/cellular therapy exposure may lead to denial unless documented evidence supports safety and anticipated benefit
Coding
| J3403 | HCPCS : J3403 |
Provider Actions and Documentation
Prior authorization — required documentation
Prior authorization is required. For Medicare Advantage/Medicaid/HARP requests, the provider must document the patient is ≥18 years, that an ophthalmologist prescribes and will administer the implant, the diagnosis of idiopathic MacTel Type 2, which eye(s) will be treated, fluorescein angiography findings, EZ break area by SD-OCT within 0.16–2.00 mm², BCVA (ETDRS or Snellen) with documentation within the last 6 months, absence of intraretinal or subretinal neovascularization, and that the request is for an FDA‑approved indication. Authorization is limited to a single implant per eye per lifetime and, when approved, will be issued for up to 6 months to allow the procedure to occur.
- Document patient age ≥18
- Document ophthalmologist as prescriber/administrator
- Document diagnosis: idiopathic MacTel Type 2
- Specify treated eye(s)
- Provide fluorescein angiography showing leakage typical of MacTel
- Provide at least one listed clinical sign (hyperpigmentation >500 µm from foveal center; retinal opacification; crystalline deposits; right-angle vessels; or lamellar cavities)
- Provide EZ (IS/OS) break area by SD-OCT between 0.16 and 2.00 mm²
- Provide BCVA (54‑letter ETDRS or ≥20/80 Snellen) with documentation within 6 months
- Document absence of intra- or subretinal neovascularization
- Confirm FDA‑approved indication and single implant per eye per lifetime
Prior authorization — gene/cell therapy one-time use
Prior authorization reviewers should treat Encelto and similar gene/cellular therapies as therapies designed for one-time administration; requests for retreatment or additional gene/cellular therapy after prior exposure should be evaluated as experimental and investigational.
- Most gene/cellular therapies are studied as single-administration treatments
- Repeat dosing, reinfusion, or sequential therapy has not been established as safe or effective
- Retreatment or additional therapy after prior exposure is considered experimental/investigational
Step therapy — contract/LCD/NCD may apply
This policy does not specify drug-specific step therapy sequences; step therapy may still be imposed per contract language or applicable LCD/NCD guidance.
- Refer to contract/benefit and any applicable NCD/LCD for step therapy requirements
- Medicare Advantage may follow NCD/LCD; in absence of NCD/LCD the MAO may set criteria
Step therapy — retreatment and sequential therapy discouraged
The policy generally discourages retreatment or sequential use of other gene or cellular therapies; repeat dosing or switching between gene/cellular products is considered experimental and may be denied.
- Retreatment with the same product is considered experimental/investigational
- Sequential use of additional or different gene/cellular therapies lacks evidence of safety and benefit
Submit clinical documentation for initial and recertification requests
Clinical documentation must be submitted for each initial and recertification request unless otherwise specified; supporting documentation should include progress notes, prior treatments/treatment history, diagnostic testing, laboratory/genetic testing if relevant, imaging (including fluorescein angiography and SD‑OCT with EZ measurement), and objective measures such as BCVA.
- Progress notes documenting previous treatments and treatment history
- Diagnostic testing and laboratory/genetic test results (if applicable)
- Imaging: fluorescein angiography and SD‑OCT with EZ break area measurement
- BCVA documentation (ETDRS or Snellen) within last 6 months
Reimbursement eligibility — check subscriber contract
Eligibility for reimbursement and whether codes are payable depend on the member’s subscriber contract; codes listed in the policy may not be covered under all circumstances and should be reviewed against benefit language.
- Verify member contract/benefit to determine medical vs. pharmacy benefit applicability
- Check for applicable NCD/LCD that may affect coverage
Denial risk — Commercial/Essential/Child Health Plus
Requests for Encelto under Commercial, Essential, or Child Health Plus lines will be denied as not medically necessary per this policy.
- Encelto is considered not medically necessary for adults with idiopathic MacTel Type 2 in these lines of business
Denial risk — failure to meet MA/Medicaid/HARP clinical criteria
Requests that do not meet the Medicare Advantage/Medicaid/HARP clinical criteria (age ≥18, ophthalmologist prescriber/administrator, correct diagnosis, required imaging and EZ/BCVA thresholds, absence of neovascularization, FDA‑approved indication, and implant‑per‑eye limit) risk denial.
- Missing or out-of-range EZ break area (not 0.16–2.00 mm²) or BCVA outside required threshold
- Lack of fluorescein angiography showing leakage typical of MacTel or missing required clinical signs
- Evidence of intraretinal or subretinal neovascularization
Denial risk — retreatment and sequential gene/cellular therapy
Retreatment, repeat dosing, or sequential therapy with other gene or cellular products is considered experimental/investigational; such requests may be denied.
- Retreatment with the same product is experimental/investigational due to lack of safety/efficacy data
- Sequential use of different gene/cellular therapies is considered investigational without documented evidence
Denial risk — prior gene/cellular therapy exposure
Prior exposure to any autologous cellular therapy, allogeneic genetically modified cellular therapy, any in vivo gene therapy, or any ex vivo gene‑modified cell product generally renders a patient ineligible for additional gene or cellular therapy and may lead to denial unless documented evidence supports safety and anticipated benefit.
- Prior autologous cellular therapies (e.g., CAR‑T, TCR‑T, TIL) exclude eligibility
- Prior in vivo gene therapies (e.g., AAV, lentiviral) or ex vivo gene‑modified products exclude eligibility
- Requests without documented evidence of safety/benefit after prior exposure likely to be denied
Definitions
Step Therapy
| Step therapy requirement | Notes |
|---|---|
| No drug‑specific step therapy specified | Step therapy may be imposed per contract language or by applicable Medicare NCD/LCD; see contract and CMS guidance |
| Coverage dependent on contract/NCD/LCD | Policy guidelines state Utilization Management is contract dependent and step therapy may be imposed in addition to LCD/NCD requirements |
| Step therapy / sequencing | Notes |
|---|---|
| Retreatment or sequential gene/cellular therapies | Policy considers repeat dosing, retreatment with the same product, or treatment with an additional/different gene or cellular therapy experimental and investigational due to lack of established safety, efficacy, and durability; sequential therapy is discouraged and may be denied |
| No explicit approved step sequences provided | The policy does not specify any required or permitted stepwise drug sequences for Encelto; clinical trials evaluated therapies as one‑time interventions |
Quantity Limits
Site of Care
Administration site — medical benefit unless contract states otherwise
Administration must occur in a healthcare setting (office, infusion center, or hospital outpatient) and, because it is healthcare professional‑administered, is covered under the medical benefit unless the member's contract specifies otherwise.
- Allowed sites: office, infusion center, hospital outpatient
- Generally billed to the medical benefit unless contract excludes
Requested site of care may affect approval timing
If an infusion center (or other requested site) is specified, reviewers should note the requested site of care may impact approval timeframe and is subject to review.
- Requested site of care can affect approval timeframe
- Site requests are reviewed as part of authorization processing
Background
Macular Telangiectasia (MacTel) Type 2 is a bilateral, slowly progressive macular disorder that commonly leads to central visual decline over years to decades. Diagnosis relies on multimodal retinal imaging (for example spectral‑domain OCT and fluorescein angiography) showing typical telangiectatic leakage and structural changes. Encelto is an encapsulated, cell‑based gene/cellular therapy delivering rhCNTF intended to support photoreceptor survival; clinical trials reported reduction in ellipsoid zone (EZ) area loss (the IS/OS break used as a structural endpoint) but effects on functional measures such as retinal sensitivity and vision were inconsistent. Given the chronic, progressive nature of MacTel Type 2 and the one‑time administration model for gene/cellular therapies, policy criteria focus on specific imaging (EZ break area) and visual acuity thresholds to identify patients similar to those studied.
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