Spark has experts envisioning an amortized payment plan for voretigene in RPE65-mediated IRDs

27 Feb 2017

Spark Therapeutics’ (NASDAQ:ONCE) voretigene neparvovec will most likely see an amortized payment plan from payers if approved regardless of its label, experts said.

Analysts have stated that Spark’s therapy has shown promising four-year durability from the Phase I study, which could support a one-time upfront cost and wide uptake, with global sales of USD 573 million expected in 2023.

Yet, experts this news service interviewed noted, regardless of efficacy, the payment plan has to be affordable and feasible for the patient. A one-time upfront payment plan is unlikely because voretigene has easily identifiable endpoints, which makes it simpler to test and reimburse for sustained efficacy over time, they explained.

The BLA's proposed indication aligns with the FDA-granted request to amend the drug's orphan designation from LCA specifically to the treatment of inherited retinal disease (IRD) due to biallelic RPE65 mutations, a company spokesperson noted. The company is on track to complete a rolling BLA submission in early 2017.

This news service reported on 15 February that experts are optimistic on approval in LCA but hesitant about getting an FDA okay for the broader label given the uncertainty around how much data there is outside of LCA. Nonetheless, as reported earlier today, coverage is expected for whatever label voretigene gets approved for, and payers are unlikely to pay a price tag of much more than USD 1.5m.

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