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Delandistrogene moxeparvovec (delandistrogene moxeparvovec-rokl; Elevidys®)
08.02.13

Policy

EXPERIMENTAL/INVESTIGATIONAL

The use of delandistrogene moxeparvovec (delandistrogene moxeparvovec-rokl; Elevidys​®) is considered experimental/investigational and, therefore, not covered for all indications including the treatment of Duchenne muscular dystrophy​ (DMD) because the safety and/or effectiveness of this service cannot be established by review of the available published peer-reviewed literature.

Guidelines

BENEFIT APPLICATION

Subject to the terms and conditions of the applicable benefit contract, delandistrogene moxeparvovec-rokl (Elevidys; Sarepta Therapeutics)​ is not eligible for payment under the medical benefits of the Company's products because it is considered experimental/investigational and, therefore, not covered.

Services that are experimental/investigational are a benefit contract exclusion for all products of the Company. Therefore, they are not eligible for reimbursement consideration.


U.S. Food and Drug Administration (FDA)


In June 2023, delandistrogene moxeparvovec-rokl (Elevidys; Sarepta Therapeutics) was approved by the FDA for treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene. This indication was approved under accelerated approval based on expression of delandistrogene moxeparvovec-rokl microdystrophin in skeletal muscle observed in patients treated with delandistrogene moxeparvovec-rokl. Continued approval for this indication may be contingent on verification and description of clinical benefit in a confirmatory trial(s).


As per the FDA guidance document for developing drugs for the treatment of dystrophinopathies, the FDA has no defined set of required or recommended clinical outcome measures to be used in clinical studies. The guidance states that manufacturers should propose and, if necessary, develop endpoints that can validly and reliably assess individuals with a wide spectrum of symptoms and disease stages. Further, it states, “The sponsor should include an assessment of multiple efficacy endpoints, when feasible, to characterize the breadth of effects on dystrophin-related pathologies, including skeletal, respiratory, and cardiac muscle function, even if the primary endpoint is only 1 of these measures.” 


Health Outcome Measures That May Be Relevant to Muscular Dystrophinopathies
Outcome MeasureDescriptionScaleClinically Meaningful Difference/Comment
Griffiths scale of mental developmentComprehensive, child-friendly developmental measure for continuous use from birth to 6 years (72 months).Consists of 2 sets of scales, 1 for each age group: 0 to 2 years and 2 to 8 years.Although used in DMD, this is a nonspecific measure and its appropriateness to measure clinical efficacy for DMD has not been established.
Bayley scales of infant and toddler development (Third edition)Designed to assess developmental functioning from 1 month to 42 months of age. Covers 5 domains: cognitive, language, motor, adaptive, and social-emotional development.Composite scores are derived for cognitive, language, and motor development and scaled to a metric, with a mean of 100, standard deviation of 15, and range of 40 to 160.Although used in DMD, this is a nonspecific measure and its appropriateness to measure clinical efficacy for DMD has not been established.
North Star Ambulatory Assessment (NSAA) or an age-appropriate modified NSAAMeasures functional motor abilities. Appropriate for ambulatory children ages ≥3 years of age with DMD.17-item scale that grades each activity from 0 (unable to achieve independently) to 2 (normal- no obvious modification of activity). Scores can range from 0 to 34. Higher scores indicate improvement. Also includes recording timed items such as the 10-meter timed walk/run test and time to rise from the floor (Gower’s test). These times are not included in the global scoreHealthy boys obtain score of 34 by 4 years of age.
Boys with DMD achieve peak score of 26 around age 6 years.
6-minute walk test (6MWT) or shorter versions such as the 2-minute walk testMeasures strength and endurance; can be appropriate for individuals as young as 5 to 6 years of age. Performance may increase with time in very young individuals, whereas performance tends to worsen with time in older individuals. Floor effect of losing ambulation in older individuals with more advanced disease and analyses of change in 6MWT can be strongly influenced by the inclusion or exclusion of individuals who lose ambulation during the trial; such individuals contribute zero values.Assesses distance walked in 6 minutes.Estimates of minimum clinically important difference for DMD individuals of a change of 30 meters have been reported.
Interpretation of 6MWT results is limited by the variability in testing procedures and individual motivation.
Myometric assessmentsAppropriate to measure increase or preservation of muscle strength, and it can be used to provide reliable measurements in children ages 5 years and older

Clinical meaningfulness of differences in muscle strength should be supported by the magnitude of the effect observed or by the demonstration of a drug effect on an appropriate functional measure.
Specific clinical respiratory outcomesNocturnal desaturation, aspiration pneumonia, and progression to mechanically assisted ventilationVaried outcome measure (dichotomous or continuous)Clinical meaningfulness of differences should be supported by the magnitude of the effect observed or by the demonstration of a drug effect on an appropriate functional measure.
Biomarker (such as dystrophin or microdystrophin)Deficiency of functional dystrophin appears to be the proximate cause of the symptomatic and functional consequences of dystrophinopathies.
Microdystrophin produced by cells transduced by delandistrogene moxeparvovec-rokl is a novel, engineered protein consisting of selected domains of the normal, full-length dystrophin protein.
Microdystrophin levels are measured in muscle fibers by immunohistochemical analysis to detect the presence or absence of microdystrophin regardless of the actual quantity of microdystrophin present while Western blot analysis quantifies the amount of microdystrophin in the muscle tissue sample and expressed as a percent of control (i.e., as a percent of levels of normal, wild-type dystrophin in muscle tissues of healthy individuals without DMD)Microdystrophin expression can only be viewed as supportive of the proof of principle. It is currently uncertain how predictive of sustained functional improvement the detected microdystrophin level could be, and what levels may be required for a meaningful clinical improvement. Further, no epidemiologic or pathophysiologic evidence is available regarding the function of microdystrophin. The protein differs in important ways from both the endogenous shortened forms of dystrophin in patients with Becker muscular dystrophy, and the internally truncated dystrophins expressed through exon-skipping drugs. Therefore, clinical effects of microdystrophin is still not fully known.
 6MWT: 6-minute walk test; DMD: Duchenne muscular dystrophy; NSAA: North Star Ambulatory Assessment.



Description

Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is an X-linked recessive disorder that occurs in approximately one in every 3500 to 5000 males. Although it primarily affects males, a small number of females are also affected, but are usually asymptomatic. Even when symptomatic, most females typically only present with a mild form of the disease. According to epidemiologic data from the United States, the first signs or symptoms of DMD are noted at a mean age of 2.5 years (range, 0.2 to 1 years). Although histologic and laboratory evidence of myopathy may be present at birth, the clinical onset of skeletal muscle weakness usually does not become evident until early childhood. The average age at diagnosis is approximately 5 years. Symptoms include motor difficulties such as difficulty running, jumping, and walking up the stairs, along with an unusual waddling gait. Some improvement in symptoms may be seen from 3 to 6 years of age, although gradual deterioration resumes, and most individuals lose ambulation by age 12 and require noninvasive ventilation by the late teenage years. Individuals progress from needing noninvasive ventilation only during night sleeping, followed by noninvasive ventilation during day and night sleeping, and then noninvasive ventilation during day and night over the course of 5 to 10 years. Median life expectancy more recently has increased into the fourth decade, primarily through improved respiratory management and cardiac care.

DMD occurs as a result of variant(s) in the gene responsible for producing dystrophin, a cohesive protein that is essential for maintaining muscle support and strength. DMD is the longest known human gene, and several variants can cause DMD. Most deletion variants disrupt the translational reading frame in the dystrophin messenger RNA, resulting in an unstable, nonfunctional dystrophin molecule. As a result, there is progressive muscle degeneration leading to loss of independent ambulation, as well as other complications, including respiratory and cardiac complications. Genetic testing is required to determine the specific DMD gene variant(s) for a definitive diagnosis, even when the absence of dystrophin protein expression has been confirmed by muscle biopsy. There are over 4700 variants in the Leiden DMD mutation database; the most common variants are concentrated between exons 45 and 53.

Delandistrogene moxeparvovec-rokl (Elevidys; Sarepta Therapeutics) 

In June 2023, delandistrogene moxeparvovec-rokl (Elevidys; Sarepta Therapeutics) was approved by the U.S. Food and Drug Administration (FDA) for treatment of ambulatory pediatric individuals aged 4 through 5 years with DMD with a confirmed mutation in the DMD gene. This indication was approved under accelerated approval based on expression of delandistrogene moxeparvovec-rokl microdystrophin in skeletal muscle observed in individuals treated with delandistrogene moxeparvovec-rokl. Continued approval for this indication may be contingent on verification and description of clinical benefit in a confirmatory trial(s).


PEER-REVIEWED EVIDENCE

DMD is an inherited disorder that results in progressive muscle weakness and loss of muscle mass, primarily affecting males. DMD results from non-sense or frame-shifting variant(s) in the DMD gene, which is responsible for producing dystrophin, a cohesive protein essential for maintaining muscle support and strength. Delandistrogene moxeparvovec-rokl is an adeno-associated virus vector-based gene therapy that encodes a novel, engineered microdystrophin protein. This novel microdystrophin protein is a shortened version (138- vs 427-kDa size of dystrophin expressed in normal muscle cells) that contains selected domains of dystrophin expressed in normal muscle cells.


Summary of the Clinical Development Program for Delandistrogene Moxeparvovec-rokl
StudyNCT NoPhaseStudy PopulationStatusStudy DatesDesignSample SizeFollow-Up
Study 101NCT033751641Ambulatory boys with DMD, age 4 to 7 yearsCompleted and unpublished2018-2023Open-label, single-arm412 weeks
Study 102NCT037691162Ambulatory boys with DMD, age 4 to 7 yearsOngoing2018-2026Part 1: DBRCT; placebo-controlled (48 weeks)
Part 2: Cross-over, blinding maintained (48 weeks)
Part 3: Open-label follow-up (5 years)
4148 weeks
Study 103 (Endeavor)NCT046266741Cohort 1: Ambulatory boys ages 4 to 7 years old
Cohort 2: Ambulatory boys age 8 to 17 years old
Cohort 3: Nonambulatory boys, no age restriction
Cohort 4: Ambulatory boys ages 3 to 4 years old
Ongoing2020-2028Open-label, single-arm3912 weeks
Study 301 (Embark)NCT050962213Ambulatory boys with DMD, age 4 to 7 yearsOngoing2021-2024Part 1: DBRCT; placebo-controlled
Part 2: Cross-over, blinding maintained
1262 years
Study 303 (Envision)NCT058814083Cohort 1: Nonambulatory
Cohort 2: Ambulatory and ≥8 to <18 years of age at the time of screening.
Ongoing2023-2027Part 1: DBRCT; placebo-controlled
Part 2: Cross-over, blinding maintained
148128 weeks
DMD: Duchenne muscular dystrophy; DBRCT: double-blind randomized controlled study; NCT: national clinical trial.



For individuals with a confirmed diagnosis of DMD who are ambulatory and who receive delandistrogene moxeparvovec-rokl, the current published evidence includes one randomized controlled trial (RCT) and one prospective cohort study. Relevant outcomes are disease-specific survival, change in disease status, functional outcomes, health status measures, quality of life, and treatment-related mortality and morbidity. In the single pivotal RCT, 41 study participants were randomly assigned 1:1 to receive either delandistrogene moxeparvovec-rokl (n=20) or placebo (n=21). Overall, there was no statistically significant difference in the primary endpoint of change in the North Star Ambulatory Assessment (NSAA) total score from baseline to week 48 between the treated group and the placebo group (1.7 vs 0.9 points, respectively; P=0.37). However, the least squares (LS) mean change in NSAA total score from baseline to week 48 among the subgroup of study participants aged 4 to 5 years was numerically greater for the treated (n=8) versus the placebo (n=8) group (4.3 vs 1.9 points, respectively). 


Study 103 included a cohort of 20 participants aged 4 through 7 years who received delandistrogene moxeparvovec-rokl. Muscle biopsies were obtained at baseline prior to infusion of gene therapy and at week 12 in all study participants. The mean delandistrogene moxeparvovec-rokl microdystrophin expression levels (change from baseline) at week 12 following infusion was 95.7% in study 102 and 51.7% in study 103. Multiple limitations were noted. First, the exploratory subgroup analysis on which the approval was based was not prespecified for hypothesis testing, and no prespecified multiplicity adjustment strategy was employed. Such post hoc subgroup analysis following an overall nonsignificant test in the overall population can only be considered as hypothesis-generating. Second, while data from open-label studies are interpretable under certain conditions—such as when the disease being studied is homogeneous, the treatment has a large effect, and the clinical endpoint can be objectively assessed—none of these conditions are applicable for DMD. Lastly, biomarker data reported in studies 102 and 103 only provide information about expression of the transgene product in cells transduced by delandistrogene moxeparvovec-rokl rather than insight into a pharmacologic effect on a known biomarker in the pathway of the disease. 


Summary of Pivotal Trials
StudyStudy TypeCountrySitesDatesParticipantsInterventionFollow-Up
ActiveControl
Study 102 (NCT03769116)Part 1: DBRCT; placebo-controlled (48 weeks)
Part 2: Cross-over, blinding maintained (48 weeks)
US220018-2026

Inclusion

  • Male at birth ambulatory DMD patients aged 4 through 7 years
  • Confirmed frameshift mutation, or a premature stop codon mutation between exons 18 to 58 in the DMD gene
  • Indication of symptomatic muscular dystrophy by protocol-specified criteria

Exclusion

  • Impaired cardiovascular function on ECHO.
  • Exposure to another investigational drug or exon skipping medication within 6 months of screening.
  • Abnormal liver or renal function by protocol-specified criteria

Primary endpoint(s)

  • Change in the quantity of delandistrogene moxeparvovec-rokl microdystrophin protein from baseline to week 12 (Part 1), as measured by Western blot
  • Change in NSAA total score from baseline to week 48 (Part 1)
  • Single intravenous infusion of delandistrogene moxeparvovec-rokl via a peripheral limb (n=20)

Dosinga

  • 8 received intended dose (1.33 X 1014 vg/kg)
  • 6 received two-thirds of intended dose (8.94 X 1013 vg/kg)
  • 6 received half of the intended dose (6.29 X 1013 vg/kg)
Placebo (N = 21)48 weeks
Study 103 (NCT04626674)Open-label, single-armUS52018-2028

Inclusion

  • Male at birth ambulatory DMD patients aged 4 through 7 years (Cohort 1)
  • Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing

Exclusion

  • Exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression within protocol-specified time limits.
  • Abnormality in protocol-specified diagnostic evaluations or laboratory tests

Primary endpoint

  • Change from baseline in quantity of delandistrogene moxeparvovec microdystrophin protein expression at week 12
  • Single intravenous infusion of delandistrogene moxeparvovec-rokl via a peripheral limb at the intended dose (1.33 × 1014 vg/kg)
  • N=20
None12 weeks
DBRCT: double-blind randomized controlled trial; DMD: Duchenne muscular dystrophy; ECHO: echocardiogram; FDA: US Food and Drug Administration; NCT: national clinical trial identification number; NSAA: North Star Ambulatory Assessment.
a It is unclear if the variation in dosing was prespecified or not. According to the FDA documents, the manufacturer retrospectively determined that in the delandistrogene moxeparvovec-rokl group, 12/20 participants received less than the intended dose. This discrepancy was identified following a change in the analytical method for dose determination.
Summary of Clinical Outcomes in Pivotal Trial
Study 102Least square mean changes (±SE) in NSAA total score from baseline to week 48

Delandistrogene moxeparvovec-roklPlaceboP value
All ages1.7 (0.6) (n=20)0.9 (0.6) (n=21)0.37
Age group 4 through 54.3 (±0.7) (n=8)1.9 (±0.7) (n=8)Exploratory analysis
Age group 6 through 7−0.2 (±0.7) (n=12)0.5 (±0.7) (n=13)Exploratory analysis​



MICRODYSTROPHIN


Delandistrogene moxeparvovec-rokl microdystrophin is a novel, engineered protein that contains selected domains of the normal, wild-type dystrophin expressed in healthy muscle cells. No epidemiologic or pathophysiologic evidence is available regarding the function of delandistrogene moxeparvovec-rokl microdystrophin. The protein differs in important ways from both the endogenous shortened forms of dystrophin in individuals with Becker muscular dystrophy and the internally truncated dystrophins expressed through exon-skipping drugs. Thus, the clinical benefit of treating DMD with delandistrogene moxeparvovec-rokl, including improved motor function and pulmonary function, has not been demonstrated. A confirmatory, prospective, and adequately powered trial is necessary to assess the net health outcome of delandistrogene moxeparvovec-rokl in individuals with DMD.


EMBARK (PART I of Confirmatory Trial)


In October 2023, Sarepta Therapeutics, manufacturer of gene therapy SRP-9001 (Elevidys) for the treatment of ambulatory patients with DMD, announced data from the Phase 3 EMBARK study (NCT05096221). Full results from EMBARK are expected to be shared at a future medical meeting and publication will be pursued in a medical journal.


On October 30, 2023, Sarepta Therapeutics announced the 1-year results of part 1 of the EMBARK Phase 3 clinical trial. This multinational, randomized, controlled, and double-blind study aims to assess the safety and efficacy of delandistrogene moxeparvovec in boys aged 4 to 7 with DMD.


The results show that the study failed to achieve its primary objective (also known as endpoint), which was to demonstrate a significant difference in the change of the North Star Ambulatory Assessment (NSAA) total score following Elevidys treatment. The NSAA is a recognized way of evaluating motor function (the ability to move) in people living with DMD (see the Benefits section of this policy document). Although some improvement in motor function was observed in children treated with Elevidys compared to the placebo group, these differences were not statistically significant. Trial participants treated with Elevidys improved by 2.6 points on the primary endpoint/main measure of NSAA, compared to 1.9 points among those given placebo. Despite the lack of this statistical significance and failure to meet the primary endpoint, Sarepta plans to ask the FDA to expand its clearance of the therapy and has shared the results with the agency.


The study appears to meet two of its secondary endpoints: the time required to rise from the floor and the 10-meter walk test. These secondary measures showed statistically significant improvements in children who received Elevidys over the course of 1 year.


Additionally, there were no new safety concerns raised during this part of the study.


The study involved 125 boys with DMD who can walk unassisted, and who were randomly assigned to either the treatment group or the placebo group. Participants will continue to be closely monitored for the next 5 years for both safety and clinical outcomes.


Part 2 of the EMBARK Phase 3 study is currently ongoing and should finish in late 2024, when it will provide further insights into the safety and efficacy of Elevidys in boys with DMD.


SUMMARY


For individuals with a confirmed diagnosis of DMD who are ambulatory and who receive delandistrogene moxeparvovec-rokl, the evidence includes one RCT and one prospective cohort study. Relevant outcomes are disease-specific survival, change in disease status, functional outcomes, health status measures, quality of life, and treatment-related mortality and morbidity. In the single pivotal RCT, 41 study participants were randomly assigned 1:1 to receive either delandistrogene moxeparvovec-rokl (n=20) or placebo (n=21). Overall, there was no statistically significant difference in the primary endpoint of change in the NSAA total score from baseline to week 48 between the treated group and the placebo group (1.7 vs 0.9 points, respectively; P=0.37). However, the least squares (LS) mean change in NSAA total score from baseline to week 48 among the subgroup of study participants aged 4 to 5 years was numerically greater for the treated (n=8) versus the placebo (n=8) group (4.3 vs 1.9 points, respectively). Study 103 included a cohort of 20 participants aged 4 through 7 years who received delandistrogene moxeparvovec-rokl. Muscle biopsies were obtained at baseline prior to infusion of gene therapy and at week 12 in all study participants. The mean delandistrogene moxeparvovec-rokl microdystrophin expression levels (change from baseline) at week 12 following infusion was 95.7% in study 102 and 51.7% in study 103. Multiple limitations were noted. First, the exploratory subgroup analysis on which the approval was based was not prespecified for hypothesis testing, and no prespecified multiplicity adjustment strategy was employed. Such post hoc subgroup analysis following an overall nonsignificant test in the overall population can only be considered as hypothesis-generating. Second, while data from open-label studies are interpretable under certain conditions, such as when the disease being studied is homogeneous, the treatment has a large effect, and the clinical endpoint can be objectively assessed, none of these conditions are applicable for DMD. Lastly, biomarker data reported in studies 102 and 103 only provides information about expression of the transgene product in cells transduced by delandistrogene moxeparvovec-rokl rather than insight into a pharmacologic effect on a known biomarker in the pathway of the disease. Delandistrogene moxeparvovec-rokl microdystrophin is a novel, engineered protein that contains selected domains of the normal, wild-type dystrophin expressed in healthy muscle cells. No epidemiologic or pathophysiologic evidence is available regarding the function of delandistrogene moxeparvovec-rokl microdystrophin. The protein differs in important ways from both the endogenous shortened forms of dystrophin in individuals with Becker muscular dystrophy, and the internally truncated dystrophins expressed through exon-skipping drugs. Thus, the clinical benefit of treating DMD with delandistrogene moxeparvovec-rokl, including improved motor function and pulmonary function, has not been demonstrated. 


A successful confirmatory, prospective and adequately powered trial is still necessary to assess the net health outcome of delandistrogene moxeparvovec-rokl in individuals with DMD, because in EMBARK, participants treated with Elevidys​ (delandistrogene moxeparvovec-rokl) showed an increase on the NSAA, a measure of motor function, compared to placebo-treated patients at 52 weeks, although the primary endpoint was not met. EMBARK was meant to confirm that approval by showing increased levels of a muscle-protecting protein translate to more definitive health benefits.

 

 



References

Birnkrant DJ, Bushby K, Bann CM, et al. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management. Lancet Neurol. 2018;17(3):251-267. 


Birnkrant DJ, Bushby K, Bann CM, et al. Diagnosis and management of Duchenne muscular dystrophy, part 2: respiratory, cardiac, bone health, and orthopaedic management. Lancet Neurol. 2018;17(4):347-361. PMID 29395990.


Bushby K, Finkel R, Birnkrant DJ, et al. Diagnosis and management of Duchenne muscular dystrophy, part 2: implementation of multidisciplinary care. Lancet Neurol. 2010;9(2):177-189. 


Centers for Disease Control and Prevention. Muscular Dystrophy: MD STARnet Data and Statistics. Available at: https://www.cdc.gov/ncbddd/musculardystrophy/data.html. Accessed July 7, 2023.


Duchenne Muscular Dystrophy and Related Dystrophinopathies: Developing Drugs for Treatment Guidance for Industry. Published February 2018. Available at: https://www.fda.gov/media/92233/download. Accessed July 7, 2023.


Falzarano MS, Scotton C, Passarelli C, et al. Duchenne Muscular Dystrophy: From Diagnosis to Therapy. Molecules. 2015;20(10):18168-84. 


Feingold B, Mahle WT, Auerbach S, et al. Management of Cardiac Involvement Associated With Neuromuscular Diseases: A Scientific Statement From the American Heart Association. Circulation. 2017;136(13): e200-e231.


Food and Drug Administration. SRP-9001 (delandistrogene moxeparvovec) for the treatment of Duchenne muscular dystrophy (DMD). Sponsor Briefing Document. Cellular, Tissue, and Gene Therapies Advisory Committee. Meeting date 12 May 2023. Available at: https://www.fda.gov/media/168022/download. Accessed July 12, 2023.


Food and Drug Administration. Summary Basis for Regulatory Action: Elevidys. June 21, 2023. Available at: https://www.fda.gov/media/169746/download. Accessed July 5, 2023.


Gloss D, Moxley RT, Ashwal S, et al. Practice guideline update summary: Corticosteroid treatment of Duchenne muscular dystrophy: Report of the Guideline Development Subcommittee of the American Academy of Neurology. Neurology. 2016;86(5):465-472. 


Henricson E, Abresch R, Han JJ, et al. The 6-Minute Walk Test and Person-Reported Outcomes in Boys with Duchenne Muscular Dystrophy and Typically Developing Controls: Longitudinal Comparisons and Clinically-Meaningful Changes Over One Year. PLoS Curr. 2013 Jul 8:5:ecurrents.md.9e17658b007eb79fcc6723089f79e06.


McDonald CM, Henricson EK, Abresch RT, et al. The 6-minute walk test and other endpoints in Duchenne muscular dystrophy: longitudinal natural history observations over 48 weeks from a multicenter study. Muscle Nerve. 2013;48(3):343-356.


Prescribing Label: Elevidys (delandistrogene moxeparvovec-rokl) suspension, for intravenous infusion. Available at: https://www.fda.gov/media/169679/download. Accessed on July 5, 2023.


Sarepta Therapeutics announces topline results from EMBARK, a global pivotal study of ELEVIDYS gene therapy for Duchenne muscular dystrophy. News release. Sarepta Therapeutics. October 30, 2023. Accessed October 31, 2023. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-topline-results-embark-global-0.


Sarepta Therapeutics announces FDA approval of Elevidys, the first gene therapy to treat Duchenne muscular dystrophy. News release. June 22, 2023. Accessed October 31, 2023. https://www.businesswire.com/news/home/20230622454844/en/.​


Zambon AA, Ayyar Gupta V, Ridout D, et al. Peak functional ability and age at loss of ambulation in Duchenne muscular dystrophy. Dev Med Child Neurol. 2022;64(8):979-988. 


Coding

CPT Procedure Code Number(s)
N/A

ICD - 10 Procedure Code Number(s)
N/A

ICD - 10 Diagnosis Code Number(s)
N/A

HCPCS Level II Code Number(s)
J1413 Injection, delandistrogene moxeparvovec-rokl, per therapeutic dose

Revenue Code Number(s)
N/A


Coding and Billing Requirements


Policy History

1/10/2024
1/10/2024
08.02.13
Medical Policy Bulletin
Commercial
No