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HAS Assessment Outcomes | 2024 | France | Healthcare | Pharma | News | iPharmaCenter






Fabry disease




Parkinson's disease








January 08, 2024

ELFABRIO (pegunigalsidase alfa) - Unfavorable Reimbursement Opinion for Fabry Disease

The reimbursement outlook for ELFABRIO in the context of "long-term enzyme replacement therapy in adult patients with Fabry disease (alpha-galactosidase deficiency) whose diagnosis has been confirmed" is not favorable, based on several considerations:

  • Firstly, the results from the BALANCE study, which demonstrated the non-inferiority of ELFABRIO compared to agalsidase beta at the 24-month final analysis. However, at the 12-month interim analysis in patients previously treated with agalsidase beta, the non-inferiority was not established. Moreover, the study lacked a placebo arm, leading to a questionable assessment of inferiority limits and limiting the interpretability of the results.

  • Secondly, the uncertainty surrounding the effectiveness of ELFABRIO raises concerns about a potential missed opportunity for patients, especially when no additional benefits, such as improved tolerance or enhanced convenience of use, are evident compared to other available treatments.

  • Thirdly, the non-comparative BRIDGE study, conducted on a smaller number of patients and with a shorter duration (12 months) than the BALANCE study, does not dispel doubts regarding the effectiveness of ELFABRIO (pegunigalsidase alfa).

  • Additionally, indirect comparisons face methodological limitations, rendering them unreliable for drawing conclusive insights.

In light of these considerations, it is concluded that ELFABRIO (pegunigalsidase alfa) does not find a place in the therapeutic strategy for Fabry disease.

The actual benefit of ELFABRIO 2 mg/ml (pegunigalsidase alfa), a concentrate for infusion, is deemed insufficient in the Marketing Authorization (MA) indication to warrant coverage by national solidarity, especially considering the available alternatives.


January 08, 2024

HAS agrees to reimburse Aquipta for migraine

SMR: Important; ASMR: V

Reimbursement support for AQUIPTA (atogepant) is conditionally granted to adults grappling with severe migraines, experiencing a minimum of eight migraine days per month. This eligibility hinges on previous unsuccessful attempts with at least two prophylactic treatments and the absence of cardiovascular disease.

The endorsement draws from the ELEVATE study's efficacy data, emphasizing its relevance for patients who have failed 2 to 4 prophylactic treatments. However, concerns loom over the uncertainty surrounding cardiac tolerance, even as patients with severe cardiovascular diseases were systematically excluded from the study protocols.

Aquipta's tangible benefits materialize in the realm of preventive treatment for severe migraines, showcasing its superiority over placebo in both episodic and chronic migraine cases. Significant improvements in quality of life, particularly among individuals who failed 2 to 4 prophylactic treatments, are evident.

The drug's short-term safety profile is robust, but apprehensions persist regarding its long-term safety, especially concerning cardiovascular risks. The absence of substantial comparative data against an active comparator, coupled with the exclusion of severe cardiovascular cases from the studies, underscores the complexity of evaluating its overall efficacy. Despite the evident medical need for an effective solution in this population suffering from severe migraines, the Commission concludes that Aquipta does not substantially enhance the actual benefit for these specific criteria.


January 25, 2024

Opdualag received favorable reimbursement for melanoma

Opdualag (nivolumab/relatlimab) received favorable reimbursement for melanoma. A favorable verdict for reimbursement applies solely to the first-line treatment of advanced melanoma (unresectable or metastatic) in individuals aged 12 years and older, exhibiting PD-L1 expression in tumor cells less than 1%, with an ECOG score of 0 or 1, and lacking active brain metastasis.

The actual benefit of Opdualag (nivolumab/relatlimab) 240 mg/80 mg, solution concentrate for infusion, is significant exclusively in the first-line management of advanced melanoma in adults and adolescents aged 12 years and above. This determination aligns with criteria including PD-L1 expression in tumor cells below 1%, with an ECOG score of 0 or 1, and the absence of active brain metastasis.

Key considerations include:

  • Demonstrated superiority of the nivolumab – relatlimab combination over nivolumab alone concerning progression-free survival in a randomized, double-blind phase 3 trial

  • Progression-free survival outcomes indicating variability in treatment effect based on PD-L1 level, favoring patients with PD-L1 expression < 1%

  • Similar care standards observed between adults and adolescents, with efficacy data from the RELATIVITY 047 study deemed extrapolatable to adolescents aged 12 and over

  • Despite:

  • Lack of evidence demonstrating an increase in overall survival.

  • A safety profile notably characterized by elevated grade 3-4 adverse events (22% in the nivolumab + relatlimab group versus 12.0% in the nivolumab group) and serious adverse events (16.1% and 8.4%), attributed to the treatment

  • Absence of conclusive results from exploratory analyses of secondary endpoints, including quality of life

  • Absence of comparative data relative to the nivolumab - ipilimumab combination, hindering precise positioning of nivolumab/relatlimab treatment in relation to nivolumab - ipilimumab combination

In light of these factors, the Commission concludes that Opdualag (nivolumab/relatlimab) 240 mg/80 mg, solution concentrate for infusion, provides only a marginal improvement in actual benefit (ASMR IV) compared to nivolumab in the first-line treatment of advanced melanoma in adults and adolescents aged 12 years and older with PD-L1 expression in tumor cells less than 1%, an ECOG score of 0 or 1, and absence of active brain metastasis.


February 02, 2024

Scyova achieved ASMR V for patiens with Parkinson's disease

Scyova (foslevodopa/foscarbidopa) achieved positive appraisal for reimbursement in the "management of advanced Parkinson's disease with motor fluctuations and severe hyperkinesia or dyskinesia and responsiveness to levodopa when existing combinations of antiparkinsonian medications have proven ineffective."

Key considerations considered by HAS were:

  • Demonstrated superiority over oral levodopa/carbidopa after a 12-week treatment period regarding:

    • Normalized average daily duration in the "on" state without bothersome dyskinesia (primary endpoint): 2.72 hours compared to 0.97 hours, resulting in a mean difference of 1.75 hours 

    • The standardized average duration in the "off" state (primary secondary endpoint): -2.75 hours versus -0.96 hours, indicating an average difference of -1.79 hours

  • Lack of comparison with other available second-line treatments for Parkinson's disease, notably continuous subcutaneous infusion of apomorphine, despite feasibility

  • Absence of demonstrated impact on quality of life due to insufficient robust data

  • The safety profile of foslevodopa/foscarbidopa is characterized by specific adverse cutaneous events

In light of these factors, the Commission concludes that Scyova (foslevodopa/foscarbidopa) 240 mg/mL + 12 mg/mL, infusion solution, does not offer a substantial improvement in actual benefit (ASMR V) in the therapeutic approach to treating advanced-stage Parkinson's disease with motor fluctuations and severe hyperkinesia or dyskinesia responsive to levodopa, in cases where existing combinations of antiparkinsonian drugs have yielded unsatisfactory outcomes.


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