The European Commission has granted conditional marketing approval to BioMarin Pharmaceutical's Roctavian (valoctocogene roxaparvovec), marking the first gene therapy approval for hemophilia. This one-time treatment is intended for adults with severe hemophilia A who do not have a history of Factor VIII inhibitors. The decision sets the stage for BioMarin to establish a viable business in Europe, following an initial rejection in the US due to durability concerns.
Roctavian utilizes an adeno-associated virus (AAV) vector to deliver a functional copy of the Factor VIII gene into the patient's cells. This approach aims to provide a potentially curative therapy for hemophilia A, freeing patients from the need for regular Factor VIII replacement therapy or antibody therapies like Roche's Hemlibra (emicizumab).
Pricing and Market Access
BioMarin anticipates a 'net' price of around €1.5 million for Roctavian in Europe, after potential discounts and outcomes-based agreements. The company plans to launch first in Germany, followed by France. The final list price will depend on negotiations with reimbursement authorities. This pricing strategy follows the precedent set by bluebird bio's Zynteglo for beta thalassemia, which faced market access challenges due to pricing concerns, ultimately leading to bluebird's withdrawal from the European market.
BioMarin is prepared to engage in outcomes-based agreements, offering rebates if patients require a return to preventive therapies for bleeding episodes. Clinical trial data have demonstrated Factor VIII production for up to five years following Roctavian administration. However, the long-term efficacy and durability of the treatment remain under investigation.
US Regulatory Pathway
BioMarin plans to resubmit its application for Roctavian's approval in the US before the end of next month. The company anticipates a decision around late March 2023, pending a six-month review by the FDA. The US pricing for Roctavian is projected to be between $2 and $3 million after discounts, reflecting the high cost of current hemophilia treatments.
The FDA's decision may be influenced by the availability of three-year results from BioMarin's phase 3 trial, expected in the coming months. These data will provide further insights into the long-term benefits and risks of Roctavian, potentially solidifying its position as a transformative treatment for severe hemophilia A.
