Evicenter
P&T meetings

RP-A501 (AAV9.LAMP2B)

Danon disease

Regulatory submission
BLA planned for late 2028
Last updated
300 sources

Product information and disease descriptionPlace of product in therapyDisease descriptionBurden of Danon disease

Economic burden and healthcare resource utilization

16 evidence topics · 21 sources

Cost of illness or healthcare resource utilization study specific to Danon disease

No evidence found.

Danon disease: heart failure outcomes and cardiac device implantation in 116 registry participants
MeasureMale, No.Male, %Female, No.Female, %Total, No.Total, %
Participants“52”“44.8”“64”“55.2”“116”
Heart failure outcome“21”“40.4”“14”“21.9”“35”“30.2”
Heart transplantation“17”“32.7”“13”“20.3”“30”“25.9”
Left ventricular assist device“2”“3.8”“2”“3.1”“4”“3.4”
Cardiovascular implantable electronic device“19”“36.5”“26”“40.6”“45”“38.8”
Pacemaker“2”“3.8”“0”“0.0”“2”“1.7”
Automatic implantable cardioverter defibrillator“17”“32.7”“26”“40.6”“43”“37.1”

“Outcome measures include heart transplantation, left ventricular assist device, and death.” (opens the source at this quote in a new tab)

“Median follow‐up time was 5.82 years (2.67–11.76 years) for the entire cohort, and 5.82 years (2.67–14.96 years) and 5.86 years (2.93–8.51 years) for female and male patients, respectively.” (opens the source at this quote in a new tab)

“Within patients who received a CIED, 2 male patients (3.8%) were implanted with pacemakers alone; the remaining patients were implanted with an automatic implantable cardioverter defibrillator, of which 5.2% (n=2) were for secondary prevention.” (opens the source at this quote in a new tab)

Danon disease: age at heart failure outcome by sex

“The incidence of the HF outcome was higher in male (40%, n=21) compared with female patients (22%, n=14) (P=0.031) (Table 1).” (opens the source at this quote in a new tab)

“Additionally, the actuarial median age of HF outcome was younger in male compared with female patients with median time to HF outcome from birth being 25 years old in male and 63 years old in female patients (P<0.001).” (opens the source at this quote in a new tab)

“While age of HF outcome in male patients (19 years [16–23 years]) was numerically lower compared with female patients (26 years [14–39 years]) who did experience HF outcome, this did not reach statistical significance (P=0.162).” (opens the source at this quote in a new tab)

Danon disease: age at first heart failure hospitalization and use of transplantation or assist devices in a European registry

“RESULTS: The cohort comprised 30 male and 27 female patients.” (opens the source at this quote in a new tab)

“The age of first heart failure hospitalization was lower in men (18±6 versus 28±17 years; P<0.003).” (opens the source at this quote in a new tab)

“Heart failure was the leading cause of death (10 of 17; 59%), and LV systolic dysfunction predicted an adverse outcome.” (opens the source at this quote in a new tab)

“Eight men and 8 women (28%) underwent heart transplantation or received an LV assist device.” (opens the source at this quote in a new tab)

Danon disease: average ages at first symptom, heart transplantation, and death

“Men had a high morbidity and were unlikely to reach the age of 25 years without a cardiac transplantation.” (opens the source at this quote in a new tab)

“Combining our data with that of 63 other Danon disease case reports in the literature, the average ages of first symptom, cardiac transplantation, and death were 12.1, 17.9, and 19.0 years in men and 27.9, 33.7, and 34.6 years in women, respectively.” (opens the source at this quote in a new tab)

Danon disease: composite of death, heart transplantation, or ventricular assist device in published cases

“The composite outcome of death, heart transplant or ventricular assist devices occurred equally in both sexes (32% of females and 37% of males, p = 0.60) but later in females (median age 38 years) than in males (median age 21 years, p < 0.001).” (opens the source at this quote in a new tab)

Danon disease: age and ventricular function at heart transplantation in 38 recipients

“The median age at transplant for the cohort was 20.2 years (15.8-27.9 years), with no difference in age between sexes.” (opens the source at this quote in a new tab)

“Median pretransplant left-ventricular ejection fraction for the entire cohort was 30% (range 11%-84%).” (opens the source at this quote in a new tab)

Heart transplantation in the United States, all diagnoses: estimated 2025 billed charges by component of care
Component of careEstimated billed charges per heart transplant, United States dollars
30 days pretransplant“67,000”
Procurement“214,500”
Hospital transplant admission“1,220,400”
Physician during transplant admission“105,200”
180 days posttransplant discharge“277,400”
Outpatient immunosuppressants and other drugs“34,200”
Total“1,918,700”

“Consistent with our 2020 report, billed charges for pretransplant, follow-up, outpatient (OP) immunosuppressants, and other drugs used cover the period from 30 days pretransplant to 180 days posttransplant discharge for follow-up and outpatient immunosuppressant and other drugs.” (opens the source at this quote in a new tab)

“Billed charges are unlikely to equal the actual amount paid for the transplant services due to the presence of negotiated reimbursement arrangements such as case rates, prescribed fee schedules, discounts, or other agreements in place.” (opens the source at this quote in a new tab)

“The transplant billed charge estimates also do not reflect differences in charges due to patient age.” (opens the source at this quote in a new tab)

“Charges may continue after the first year and may include continued testing and evaluation, medical services for transplant rejection, and outpatient immunosuppressants.” (opens the source at this quote in a new tab)

Heart transplantation in the United States, all diagnoses: estimated 2025 volume, utilization, and length of stay
MeasureValue for heart transplant
Total estimated number of transplants, 2025“4,109”
Estimated annual utilization per 1,000,000, under age 65“11.99”
Estimated costs per member per month, under age 65, United States dollars“1.92”
Estimated hospital length of stay, days“40.6”
Pediatric heart transplant recipients, all diagnoses: hospitalization costs, 2002 to 2016

“Methods We identified all pediatric heart transplant recipients in a unique linked PHIS/SRTR dataset (2002–2016).” (opens the source at this quote in a new tab)

“Hospital costs were estimated from charges using cost-to-charge ratios, inflated to 2016 dollars.” (opens the source at this quote in a new tab)

“Results A total of 2896 pediatric heart transplant recipients were included; Era-1:649 (22.4%), Era-2:1028 (35.5%), and Era-3:1219 (42.1%).” (opens the source at this quote in a new tab)

“However, between Era-2 and Era-3 there was a decline in total ($906,454 vs. $767,221; p<0.001), pretransplant ($435,554 vs. $353,364; p<0.001), and posttransplant ($586,133 vs. $508,719; p=0.002) hospitalization costs.” (opens the source at this quote in a new tab)

Pediatric heart transplant hospitalizations, all diagnoses: hospital charges, 1997 to 2006

“METHODS: A retrospective analysis of the Healthcare Cost and Utilization Project Kids' Inpatient Database was performed on admissions surrounding heart transplantation for the years 1997, 2000, 2003, and 2006.” (opens the source at this quote in a new tab)

“Mean hospital charges increased from $279,399 in 1997 to $451,738 in 2006 (p < 0.001).” (opens the source at this quote in a new tab)

Children bridged to heart transplantation with mechanical circulatory support, all diagnoses: hospitalization costs

“Methods All pediatric HT recipients (2002–2016) were identified from a unique, linked PHIS/SRTR dataset.” (opens the source at this quote in a new tab)

“Costs were calculated from hospital charges, inflated to 2016 Dollars and adjusted for patient-specific characteristics using generalized linear mixed-effects models.” (opens the source at this quote in a new tab)

“Results A total of 2873 pediatric HT recipients were included; no MCS: 2268 (78.9%), VAD: 470 (16.4%), and ECMO: 135 (4.7%).” (opens the source at this quote in a new tab)

“Both VAD and ECMO were associated with greater total hospitalization costs compared to no MCS ($755,345 and $808,771 vs. $457,086; p<0.001).” (opens the source at this quote in a new tab)

“Outpatients with VAD support at HT demonstrated significantly lower total costs compared to those who were inpatient with continuous flow devices ($552,222 vs. $663,071, p=0.003).” (opens the source at this quote in a new tab)

“RESULTS: There were 145 children who underwent VAD implantation at 19 centers in North America.” (opens the source at this quote in a new tab)

“The median duration of VAD support was 43 days (range, 1 to 465 days).” (opens the source at this quote in a new tab)

“The mean hospital cost was $624,798.” (opens the source at this quote in a new tab)

Adults receiving a left ventricular assist device in the United States, all diagnoses: hospitalization cost and national expenditure

“RESULTS: There were 2,038 LVAD implantations from 2005 to 2011.” (opens the source at this quote in a new tab)

“Average length of stay decreased from 44 days in the pulsatile-flow era (2005 to 2007) to 36 days in the continuous-flow era (2008 to 2011).” (opens the source at this quote in a new tab)

“Cost of hospitalization increased from $194,380 in 2005 to $234,808 in 2011 but remained constant from 2008 to 2011.” (opens the source at this quote in a new tab)

“METHODS: Using the National Inpatient Sample from 2009 through 2014, the study identified index hospitalizations for OHT and LVAD.” (opens the source at this quote in a new tab)

“The annual expenditure increased from ∼$288 to $451 million for OHT and from ∼$400 to $800 million for LVAD during the study period.” (opens the source at this quote in a new tab)

“Overall, Medicare and Medicaid contributed to more than 50% of the costs associated with these hospitalizations.” (opens the source at this quote in a new tab)

Pediatric heart failure-related hospitalizations in the United States, all diagnoses: volume, length of stay, and charges

“CONCLUSIONS: Heart failure-related hospitalizations occur in 11,000-14,000 children annually in the United States, with an overall mortality of 7%.” (opens the source at this quote in a new tab)

“Hospital length of stay increased from 1997 (mean 13.8 days, 95% CI 12.5-15.2) to 2006 (mean 19.4 days, 95% CI 18.2 to 20.6).” (opens the source at this quote in a new tab)

“Inflation-adjusted charges are used as a proxy for cost.” (opens the source at this quote in a new tab)

“There were a total of 33,189 HFRH captured from 2000 to 2009.” (opens the source at this quote in a new tab)

“Median charges per HFRH rose from $35,079 in 2000 to $72,087 in 2009 (p<0.0001).” (opens the source at this quote in a new tab)

“The greatest median charges were incurred in patients on extracorporeal membrane oxygenation ($442,134 vs $53,998) or ventricular assist devices ($462,647 vs $55,151).” (opens the source at this quote in a new tab)

Adult heart failure hospitalizations in the United States, all diagnoses: cost per hospitalization and annual medical cost

“For hospitalizations with primary HF, the estimated mean cost was $11,552 in 2014, totaling an estimated $11 billion.” (opens the source at this quote in a new tab)

“Cost per HHF ranged from $7,094 to $9,769 (median) and $10,737 to $17,830 (mean).” (opens the source at this quote in a new tab)

“Charge per HHF ranged from $22,162 to $40,121 (median), and $50,569 to $50,952 (mean).” (opens the source at this quote in a new tab)

“The annual median total medical costs for heart failure care were estimated at $24,383 per patient, with heart failure-specific hospitalizations driving costs (median $15,879 per patient).” (opens the source at this quote in a new tab)

Implantable cardioverter-defibrillator placement admissions in United States adults with and without congenital heart disease: hospital charges

“METHODS: We analyzed data from the 2002-2014 United States National Inpatient Sample (NIS).” (opens the source at this quote in a new tab)

“Compared to those without CHD, ACHD patients had higher adjusted total hospital charges ($147,002 ± 5516 vs $132,455 ± 2182; p < 0.001), length of stay (6.2 ± 0.5 vs 5.2 ± 0.1 days; p < 0.001), lower readmission score (5.5 ± 0.5 vs 9.7 ± 0.1; p = 0.04) and a higher complication rate (13.4% vs 8.3%; p < 0.001).” (opens the source at this quote in a new tab)

Adults with obstructive hypertrophic cardiomyopathy in United States commercial and Medicare supplemental claims: annual resource use and cost

“METHODS: We used the US IBM MarketScan Commercial and Medicare Supplemental database to identify patients with oHCM (January 2009-March 2019).” (opens the source at this quote in a new tab)

“These differences were driven mainly by the clinical and economic burden of symptomatic oHCM, which was associated with significant increases in 1-year hospitalization rates (38.0 vs. 6.9%), hospital days (3.7 ± 9.9 vs. 0.4 ± 3.0), and total healthcare costs ($43,586 ± 103,756 vs. $6,768 ± 27,618; all p<.001).” (opens the source at this quote in a new tab)

“CONCLUSION: In a real-world population, oHCM was associated with substantial increases in HRU and incremental costs of ∼$20,000/year when compared with matched controls-a difference that increased to ∼$35,000/year among symptomatic patients.” (opens the source at this quote in a new tab)