Summary

No SKY-0515-specific economic model, sales forecast, cost-effectiveness analysis, budget impact analysis, or health technology assessment was identified. A market research announcement estimates the seven-major-market HD market at approximately USD 320 million in 2025, with a projected 14% compound annual growth rate from 2026 to 2036, and lists SKY-0515 among pipeline candidates without product-level figures.

Three published disease-level models address disease-modifying therapy for Huntington's disease. A U.S. Markov model (early, middle, late, and death states; 30 annual cycles; 3% annual discount rate; modified societal perspective; willingness-to-pay threshold of $500,000 per QALY) estimated incremental costs of $2.11 million and 0.93 QALYs for tominersen (incremental cost-effectiveness ratio $2.28 million per QALY) and incremental costs of $1.49 million and 5.23 QALYs for AMT-130 ($285,703 per QALY) versus standard of care. Drug costs were assumed from spinal muscular atrophy therapies (tominersen $318,750 per year, based on nusinersen; AMT-130 $1,806,250 once, based on the Zolgensma list price), and treatment-arm utilities were set equal to standard of care. The ICERs were most sensitive to drug costs and early- and middle-stage utility values. In probabilistic analysis, AMT-130 had an 80% probability of being cost-effective at $350,000 per QALY, and tominersen had a 0% probability throughout the acceptability curve; adding a value of hope to utilities lowered the ICERs to $1.52 million per QALY (tominersen) and $236,540 per QALY (AMT-130). The authors stated that the model relied heavily on an assumption of sustained benefit beyond the available data. A U.S. decision-modeling framework estimated lifetime costs of $508,200 (prefunctional decline), $1.15 million (Shoulson and Fahn stage 1), and $1.07 million (stage 2), and found that hypothetical disease-modifying treatments reduced costs only in the prefunctional decline population. A U.K. progression and societal cost model using Enroll-HD data identified indirect costs as potential drivers of societal cost.

The Institute for Clinical and Economic Review announced in August 2026 an assessment of AMT-130 in adults with early symptomatic Huntington's disease, with a planned lifetime cost-effectiveness model based on Total Functional Capacity health states and a separate five-year health care system budget impact analysis. The model analysis plan is scheduled for November 16, 2026, and public deliberation for March 2027. SKY-0515 is not included as an intervention in the draft scope.

Market research forecast: HD market in the seven major markets

“The 7MM Huntington's disease market reached approximately USD 320 million in 2025.” (opens the source at this quote in a new tab)
“The market is expected to expand at a 14% CAGR between 2026 and 2036.” (opens the source at this quote in a new tab)
“Pipeline candidates including SKY-0515 and votoplam (PTC518) are advancing the shift toward huntingtin-lowering and RNA-targeted treatment strategies.” (opens the source at this quote in a new tab)

U.S. cost-effectiveness analysis of tominersen and AMT-130 in early-stage HD

“A Markov model with four health states (early, middle, late, and death) was developed to estimate lifetime costs and benefits from a modified societal perspective, with death as an absorbing state.” (opens the source at this quote in a new tab)
“As per the incremental cost-effectiveness ratio (ICER) value assessment framework, a willingness-to-pay threshold (WTP) of $500,000/QALY gained was considered given that HD is a rare disease.” (opens the source at this quote in a new tab)
“Compared with SoC, Tominersen increased costs by $2.11 million and QALYs by 0.93, resulting in an ICER of $2.28 million per QALY gained, whereas AMT-130 increased costs by $1.49 million and QALYs by 5.23, resulting in an ICER of $285,703 per QALY gained in the base case.” (opens the source at this quote in a new tab)
“Probabilistic analysis showed AMT-130 had 80% probability of being cost-effective at $350,000/QALY gained.” (opens the source at this quote in a new tab)
“Further evidence on long-term effectiveness and durability is needed to inform policy and reimbursement decisions.” (opens the source at this quote in a new tab)

U.S. cost-effectiveness analysis of tominersen and AMT-130: model structure, perspective, and time horizon

“A modified societal perspective was adopted to capture direct healthcare costs and non-healthcare costs such as caregiver burden.” (opens the source at this quote in a new tab)
“The cycle length was 1 year with an annual discount rate of 3% used for all future costs and utilities.” (opens the source at this quote in a new tab)
“a total of 30 annual cycles were considered sufficient to capture lifetime costs and health outcomes associated with the disease.” (opens the source at this quote in a new tab)
“All patients entered the model in the early stage, and at the end of each cycle, they could transition into a different health stage, stay in the early stage, or experience death” (opens the source at this quote in a new tab)
“For Tominersen, we postulated that even though the drug would result in slowing the disease progression, it would not result in disease reversal. Therefore, we made an assumption on the basis of expert opinion that the probability of disease progression from the early stage to the middle stage would reduce from 0.16 to 0.1 for the base-case model” (opens the source at this quote in a new tab)
“Utilities for Tominersen and AMT-130 were assumed equal to SoC because trial-based utility data are not yet available.” (opens the source at this quote in a new tab)

U.S. cost-effectiveness analysis: standard of care inputs by disease stage

Input	Base case	Range
Transition probability, early to middle	“0.16”	“0.1–0.2”
Transition probability, middle to late	“0.32”	“0.3–0.4”
Mortality rate, early to death	“0.01”	“0–0.05”
Mortality rate, middle to death	“0.03”	“0–0.05”
Mortality rate, late to death	“0.1”	“0–0.15”
Early stage, inpatient cost	“$1067”	“$1044–1091”
Early stage, outpatient cost	“$3962”	“$3878–4047”
Early stage, pharmacy cost	“$11,124”	“$10,705–11,544”
Early stage, caregiver cost	“$6120”	“$6117–6124”
Middle stage, inpatient cost	“$4492”	“$4456–4528”
Middle stage, outpatient cost	“$10,356”	“$10,293–10,420”
Middle stage, pharmacy cost	“$13,001”	“$12,633–13,370”
Middle stage, caregiver cost	“$38,380”	“$33,756–43,004”
Late stage, inpatient cost	“$19,188”	“$18,933–19,444”
Late stage, outpatient cost	“$17,942”	“$17,621–18,263”
Late stage, pharmacy cost	“$19,283”	“$18,909–19,657”
Late stage, caregiver cost	“$112,287”	“$82,119–142,454”
Utility, early stage	“0.73”	“0.7–0.9”
Utility, middle stage	“0.63”	“0.5–0.7”
Utility, late stage	“0.57”	“0.4–0.6”

U.S. cost-effectiveness analysis: drug price and administration cost assumptions

“Since Tominersen and AMT-130 are currently in clinical trials, we lacked established cost data and therefore relied on cost estimates from treatments for spinal muscular atrophy (SMA).” (opens the source at this quote in a new tab)
“After accounting for the discount, the estimated cost of a single 60 mg injection of Tominersen was $106,250.” (opens the source at this quote in a new tab)
“Based on this schedule, the estimated annual cost of Tominersen was $318,750.” (opens the source at this quote in a new tab)
“Considering these similarities, the cost for AMT-130 was assumed to be the same as the list price for Zolgensma” (opens the source at this quote in a new tab)
“The regular payer discount of 15% was then applied to the list price, for a one-time dose of AMT-130, which was estimated to be $1,806,250.” (opens the source at this quote in a new tab)

Treatment arm input	Base case	Range
Tominersen, early to middle transition probability	“0.10”	“0–0.16”
AMT-130, early to middle transition probability	“0”	“0–0.16”
Tominersen, adverse events rate	“0.4”	“0.3–0.5”
AMT-130, adverse events rate	“0.2”	“0.1–0.3”
Tominersen, annual drug cost: 3 intrathecal injections of 60 mg (low dose)	“$318,750”	“$207,188–375,000”
Tominersen, adverse event	“$24,443”	“$21,998–26,887”
AMT-130, one-time drug cost (low dose)	“$1,806,250”	“$1,381,250–2,125,000”
AMT-130, adverse event	“$22,215”	“$19,994–24,437”

Treatment arm administration cost	Base case
Tominersen	“$3627”
AMT-130	“$23,516”

U.S. cost-effectiveness analysis: base-case results

Intervention	Total cost	Total QALYs	Incremental cost	Incremental QALYs	ICER ($/QALY gained)
Standard of care	“$685,662”	“6.11”	“–”	“–”	“–”
Tominersen	“$2,795,956”	“7.03”	“$2,110,294”	“0.93”	“$2,280,427”
AMT-130	“$2,180,147”	“11.34”	“$1,494,484”	“5.23”	“$285,703”

U.S. cost-effectiveness analysis: scenario and sensitivity analyses

“After considering the additional value of hope to the base-case utility value, the total QALYs for Tominersen increased from 7.03 to 7.49, and for AMT-130, it increased from 11.34 to 12.43.” (opens the source at this quote in a new tab)
“As a result, the ICER value decreased significantly from $2.28 million per QALY gained to $1.52 million per QALY gained for Tominersen, and for AMT-130, it changed from $285,703 per QALY gained to $236,540 per QALY gained.” (opens the source at this quote in a new tab)
“One-way sensitivity analyses indicated that the ICERs were most sensitive to the cost of the drugs, particularly AMT-130 and Tominersen, as well as health-state utility values for early and middle-stage HD.” (opens the source at this quote in a new tab)
“The cost-effectiveness acceptability curve revealed that the probability of AMT-130 being considered cost-effective at a WTP threshold of $250,000 per QALY gained was 30%” (opens the source at this quote in a new tab)
“The probability of Tominersen being considered cost-effective was 0% throughout the curve” (opens the source at this quote in a new tab)
“Findings from the PSA also indicated that the probability of both of the treatments being considered cost-effective was 0% at the conventional WTP threshold of $100,000–150,000 per QALY gained.” (opens the source at this quote in a new tab)

U.S. cost-effectiveness analysis: stated limitations

“The model relied on many assumptions and simplifications owing to limited clinical trial evidence and the lack of established cost data for Tominersen and AMT-130.” (opens the source at this quote in a new tab)
“The model relied heavily on the assumption of sustained benefit for both treatments beyond the data.” (opens the source at this quote in a new tab)
“The statistical generalizability or the comparative efficacy of AMT-130 and Tominersen with the SoC cannot be assessed.” (opens the source at this quote in a new tab)
“Additionally, it should be noted that the Tominersen trial recently updated its protocol to continue only the 100-mg dose” (opens the source at this quote in a new tab)

U.S. decision-modeling framework for future disease-modifying treatments

“The lifetime costs of HD were $508,200 ($310,300 to $803,700) for the PFD population, $1.15 million ($684,500 to $1.89 million) for SF1 individuals, and $1.07 million ($571,700 to $2.26 million) for SF2 individuals.” (opens the source at this quote in a new tab)
“Although hypothetical DMTs led to cost savings in the PFD population by delaying the cost burdens of functional decline, they increased costs in SF1 and SF2 populations by prolonging time spent in expensive progressive HD states.” (opens the source at this quote in a new tab)
“Recent research estimates that annual costs associated with HD in the United States ranges from $6,600 to $30,300 per year depending on the stage of progression.” (opens the source at this quote in a new tab)
“Furthermore, the DMTs currently under development vary in mechanism of action (small molecule, ASO, gene therapy), route of administration (oral, intrathecal, intraparenchymal), and once vs recurrent therapy; these contingencies make it difficult at this time to assign a DMT price in our exploratory analysis.” (opens the source at this quote in a new tab)

United Kingdom progression model with societal cost estimation

“Data from the Enroll-HD observational study were used to fit a continuous-time hidden Markov disease progression model, which identified five distinct states.” (opens the source at this quote in a new tab)
“Finally, the results of the UK cost modelling show that indirect costs of HD such as state benefits and lost gross domestic product contribution could be the driving factors for the societal cost, over and above health and social care costs.” (opens the source at this quote in a new tab)

Health technology assessment: ICER assessment of AMT-130 announced

“The Institute for Clinical and Economic Review (ICER) announced today that it will assess the comparative clinical effectiveness and value of ifezuntrigene inilparvovec (uniQure N.V.) for Huntington’s disease.” (opens the source at this quote in a new tab)
“The assessment will be publicly discussed during a meeting of the Midwest Comparative Effectiveness Public Advisory Council (CEPAC) in March 2027, where the independent evidence review panel will deliberate and vote on evidence presented in ICER’s report.” (opens the source at this quote in a new tab)

Health technology assessment: ICER draft scope for population, comparator, and planned models

“The population of focus for the review is adults with early symptomatic Huntington’s disease.” (opens the source at this quote in a new tab)
“As a complement to the evidence review, we will develop an economic model to assess the lifetime cost-effectiveness of AMT-130 relative to usual symptomatic care.” (opens the source at this quote in a new tab)
“The base case analysis will take a health care system perspective (i.e., focus on direct medical care costs only).” (opens the source at this quote in a new tab)
“The model will consist of health states capturing distinct levels of clinical progression, based on Total Functional Capacity (TFC) scores.” (opens the source at this quote in a new tab)
“In separate analyses, we will explore the potential health care system budgetary impact of treatment over a five-year time horizon, utilizing published or otherwise publicly available information on the potential population eligible for treatment and results from the economic model for treatment costs and cost offsets.” (opens the source at this quote in a new tab)
“A detailed economic model analysis plan with proposed methodology, model structure, model parameters, model inputs, and model assumptions will be published on November 16, 2026.” (opens the source at this quote in a new tab)

SKY-0515-specific sales forecast, economic models, and health technology assessments

No evidence found.

Budget impact analysis: eligible population and intervention mix (ISPOR task force)

“The eligible population is open in the sense that individuals enter or leave depending on whether they meet the criteria for inclusion (e.g., by developing the indication, meeting the restrictions, no longer having symptoms, and dying).” (opens the source at this quote in a new tab)
“A BIA compares scenarios defined by sets of, rather than specific individual, interventions. The starting scenario should be the current intervention mix for the eligible population. The current mix may include no intervention as well as interventions that might be replaced by the new one.” (opens the source at this quote in a new tab)

Potential budget impact: uptake convention in the ICER reference case

“Assume an equal proportion of patients (20%) are treated each year over five years, arriving at cumulative 100% at five years.” (opens the source at this quote in a new tab)

Budget impact analysis: perspective, time horizon, and discounting (ISPOR task force)

“Given the purpose of a BIA, the recommended perspective is that of the budget holder.” (opens the source at this quote in a new tab)
“A time horizon of 1 to 5 years is common, with the results presented for each budget period after the new intervention is covered.” (opens the source at this quote in a new tab)
“Thus, the BIA should present the financial streams at each budget period as undiscounted costs.” (opens the source at this quote in a new tab)

Budget impact model: perspective and time horizon (AMCP Format, version 5.0)

“The perspective of the HCDM organization is recommended. The time horizon of the model should be of relevance to the HCDM, typically one to five years.” (opens the source at this quote in a new tab)

United States all-state closed claims: annual prevalence of diagnosed Huntington's disease per 100,000, 2020 to 2024

“Of the 59 million individuals in the study population, 38.4 million (65.1%) met the continuous enrollment requirement of 12 months during the study period.” (opens the source at this quote in a new tab)

Year	Prevalence rate per 100,000 (95% CI)
2020	“6.7 (6.3-7.1)”
2021	“7.4 (7.0-7.8)”
2022	“7.9 (7.5-8.3)”
2023	“8.4 (8.0-8.8)”
2024	“9.4 (8.9-9.8)”

British Columbia: multisource prevalence of Huntington's disease

“Prevalence of HD was estimated at 13.7 per 100,000 (95% confidence interval [CI]: 12.6-14.8 per 100,000) in the general population, and 17.2 per 100,000 (95% CI: 15.8-18.6 per 100,000) in the Caucasian population.” (opens the source at this quote in a new tab)

United States commercial claims: share of diagnosed people aged 20 years or younger

“Among those diagnosed, 1.18% (n = 44) were identified as 20 years old or less, indicating juvenile onset.” (opens the source at this quote in a new tab)

North American Enroll-HD participants with manifest Huntington's disease: Total Functional Capacity stage

“Most participants were in TFC stages I–III, with the highest proportion (41.3%) of participants in stage II (Table 1).” (opens the source at this quote in a new tab)

Stage (Total Functional Capacity score)	Participants
Stage I (11 to 13)	“n = 845”
Stage II (7 to 10)	“n = 1068”
Stage III (3 to 6)	“n = 540”
Stage IV (1 to 2)	“n = 116”
Stage V (0)	“n = 15”

Pooled incidence of Huntington's disease

“Pooled incidence was 0.48 cases per 100,000 person-years (95% CI, 0.33-0.63).” (opens the source at this quote in a new tab)

United Kingdom primary care: median survival from first recorded diagnosis

“The KM survival curve (Figure 1) shows a median survival time of 12.4 years (95% CI 10.6–15.2).” (opens the source at this quote in a new tab)

Colorado all-payer claims: share of people with Huntington's disease seen by a neurologist in a calendar year

“Approximately half (49.9%) received neurologist care, with 33.8% seeing a MDS during the given calendar year.” (opens the source at this quote in a new tab)

Launch prices of oral drugs for rare neurological diseases: Skyclarys and Daybue

“Reata announced that Skyclarys would have a wholesale acquisition price of $370,000 annually.” (opens the source at this quote in a new tab)

“The list price of Daybue will be $21.10 per milliliter, or an estimated average net realized cost to payors of about $375,000 annually” (opens the source at this quote in a new tab)

SKY-0515: safety and efficacy assessments named in the phase 2/3 study record

“Safety and efficacy will be evaluated through blood tests, imaging (MRI), and clinical assessments, including the Unified Huntington's Disease Rating Scale (UHDRS).” (opens the source at this quote in a new tab)

Budget impact model: reporting of results (AMCP Format, version 5.0)

“When reporting the economic impact of the intervention, it is recommended to present the findings as both the” (opens the source at this quote in a new tab)
“PMPM cost difference and the overall budget impact on the health system. Results may also include cost per-treated member per-month (PTMPM).” (opens the source at this quote in a new tab)