With a 2021 Food and Drug Administration (FDA) submission now on the cards, following impressive data from its pivotal trial HOPE-B, uniQure’s etranacogene dezaprvovec (EtranaDez) is now set to become a first-in-class gene therapy agent with a label in hemophilia B. This will allow uniQure to tap into a market worth $1.3bn per year and is expected to reach peak sales of $649m, according to GlobalData, a leading data and analytics company.

Sakis Paliouras, PhD, Oncology Analyst at GlobalData, comments: “The data presented at ASH20 are impressive – with the drug having achieved a high enough increase in factor IX levels at 26 weeks to avoid bleeding events and a 96% decrease in the need for factor IX replacement therapies. EtranaDez, essentially, offered normal function to most patients. Plus, the drug’s designation as a breakthrough therapy by the FDA should allow it a fast review process, leading to approval potentially as early as Q4 2021.”

GlobalData estimates that 2020 will see 12,000 prevalent cases of hemophilia B in the 8MM. Of these, over 70% are of moderate-to-severe-intensity symptoms and thus eligible for treatment with EtranaDez.

Hemophilia B is a highly competitive market with a severe lack of innovative marketed agents. Most of the competition results from factor IX replacement therapies, with a similar mechanism of action.

Agents of a novel mechanism of action, particularly when of curative intent, do not compete on the same level with less innovative therapies. Therefore, it is likely that upon successful negotiation for a fair price, EtranaDez will capture a large share of the hemophilia B market.

Earlier in June, the marketing rights for EtranaDez were acquired by CSL Behring, in a deal totaling $2bn – but only if EtranaDez is approved and marketed.

Paliouras adds: “Prophylactic recombinant factor IX agents cost between $400,000 and $900,000 per year in the US. Over a lifetime of use, the cost of symptom-relieving therapy can amass to several million dollars. Thus, uniQure can, and is expected to, set a high price of over $1m per year for EtranaDez. This is similar to other marketed gene therapies, justified by its curative intent. However, the high price may be offset by outcome-based pricing deals in cost-conscious countries.”

*8MM: The US, France, Spain, Germany, Italy, the UK, Japan and China