On November 22 nd , the Food and Drug Administration (FDA) approved the world’s first gene therapy for hemophilia B; Hemgenix (etranacogene dezaparvovec). Hemgenix will have a list price of $3. 5 million per use. This sets a new record with regard to the most expensive single-use gene therapy in the U. S. In spite of the very high price tag, the budgetary impact of the product for most payers will be comparatively small.

The product was first developed by the particular Dutch biotechnology company uniQure. In 2020, CSL Behring paid $450 million to license the therapy. CSL Behring will be marketing the drug.

Hemophilia B is caused by missing or low levels of a protein called Factor IX, which is needed to clot blood. Depending on the amount of Factor IX present, people with hemophilia B can experience spontaneous or excessive bleeding that can lead in order to severe health complications. To prevent these complications from occurring, the current standard associated with care includes treatments consisting of replacement of Factor IX. These must be taken regularly.

Hemgenix’s sponsor, CSL Behring, is no stranger to the hemophilia W disease space, as it’s been a specialist in plasma and blood-related products for a long time. The company sells two approved Factor IX therapies for hemophilia B.

Hemgenix is designed to replace the gene that is responsible for Element IX and is defective in people with hemophilia B. Results from the pivotal Phase 3 trial that led to Hemgenix’s approval showed that will seven in order to 18 months after infusion, the average adjusted annualized bleeding rate was reduced by 54% compared to baseline. The trial included 54 patients with hemophilia M, 51 associated with whom (or 94%) were able to discontinue make use of prophylaxis and remained free of those routine treatments 18 months associated with follow-up.

The cost-effectiveness watchdog, the Institute for Clinical and Economic Review (ICER), determined that it would become “fair” regarding Hemgenix in order to be priced at or near $2. 9 million.

Additionally , ICER noted that the gene therapy such as Hemgenix becomes more cost effective the more durable it is. The particular company marketing the product, CSL Behring, contends that by reducing costs associated with bleeding and prophylactic infusions, the particular one-time treatment will save money over time. This is because the present costs of treating people with moderate to severe hemophilia N can be quite significant. For serious hemophilia T patients, intended for example, over the period of a lifetime the cost can be as much as $20 mil per person.

However, at present, it’s not known how durable the particular treatment is going to be. It’s speculation whether the therapy will be “cost saving” because CSL Behring claims.

And, given the particular churn that will exists – the rate at which enrollees leave insurers – many payers in the U. S. may not have a long-term view. That is, they may not really be that interested inside cost savings that will occur to get patients years down the road. At the same time, payers will take notice associated with the high upfront expenses per patient.

However, the relatively little sub-population that can be treated along with Hemgenix should keep costs in check pertaining to most payers. As the rare disease, hemophilia Udemærket afflicts approximately 6, 000 people in the U. S. About 15% of patients require Factor IX therapy. And only a small portion of these people will be eligible meant for Hemgenix. In other words, the impact on most payers’ budgets – their financial exposure – is going to be relatively limited.

Estimates associated with global sales of Hemgenix project $1. 2 billion cumulatively through 2026. So , despite its eye-popping price of $3. 5 million, this is not a gene therapy that will necessarily break the bank. Moreover, it is likely that value-based pricing arrangements will be implemented which mitigate the particular impact from the high upfront costs per dose. For example , payers will likely negotiate “ milestone-based reimbursement , ” with installment payments based on (durable) outcomes achieved.

Looking to the future, certain other cell and gene therapies within the pipeline have considerably larger (potentially) eligible populations of patients, including the disease area ischemic stroke. In addition, currently authorized cell plus gene products in several cancers are now indicated for earlier lines associated with treatment, which usually implies substantial expansion of populations suitable for treatment. It’s these types of treatments that (will) hit payer radar screens.

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