Biomarin limits Roctavian development to US, Germany, Italy
Cost cuts aimed at making therapy profitable by end of 2025
In an effort to cut costs and boost profitability, Biomarin Pharmaceutical is limiting commercial development of hemophilia A gene therapy Roctavian (valoctocogene roxaparvovec-rvox) to three countries: the U.S., Germany, and Italy.
The company has struggled to turn a profit from the gene therapy, which was approved in the European Union in 2022 and in the U.S. in 2023. In the second quarter, three patients in the U.S. and two in Italy received the gene therapy, generating just over $7 million in revenue.
The company said it will focus on marketing Roctavian in these three countries where the therapy is approved and reimbursement policies are in place, but won’t invest in expanding it to other markets. Biomarin is also making other efforts to reduce costs, including a pause on manufacturing and clinical trial enrollment.
The goal is to cut annual expenses related to Roctavian to $60 million, which the company expects would allow the gene therapy to become profitable by the end of next year.
“We continue to believe that ROCTAVIAN is an important option for people with severe hemophilia A, offering the potential for years of bleed control after a single, one-time treatment,” Alexander Hardy, president and CEO of Biomarin, said in a company press release. “By rightsizing our resourcing, we are creating a path for ROCTAVIAN to contribute to our profitability while still providing full support to patients.”
One-time therapy designed to lower bleed risk
Roctavian is a gene therapy that’s designed to provide patients with a functional version of the F8 gene. Mutations in this gene are the cause of the excessive and prolonged bleeding episodes that characterize hemophilia A. The one-time therapy is expected to reduce the risk of bleeds and lower or eliminate the need for conventional preventive replacement therapy.
Biomarin previously noted that “reimbursement and market access challenges” for the therapy, which has a list price of nearly $3 million in the U.S., has affected the “ability of interested patients” to receive the therapy.
The company is working on various efforts to overcome those challenges in the three countries it has decided to target. In the U.S., that includes working on single-patient reimbursement agreements with insurers, and aiming to make sure more hemophilia treatment centers are ready to administer Roctavian.
Biomarin is also advancing discussions with sub-insurers in Germany to allow coverage for patients interested in Roctavian, and is making efforts in Italy to promote patient access and interest.
Future expansion into other markets will depend on the progress made in these three countries.
Biomarin will also readjust other aspects of Roctavian’s development, including reducing investments in development and manufacturing. Since enough of the gene therapy has been produced to meet expected demand, Roctavian’s manufacturing facility will remain idle until new batches are needed.
On the research side, Biomarin won’t be enrolling any new participants in clinical trials. It will, however, continue to follow those who have already been treated and gather long-term safety and efficacy data on the therapy.
“We are deeply grateful to the hemophilia community and to the patients and healthcare providers who participated in our clinical trials, and we will continue to provide support, as well as to meet our regulatory commitments for ongoing monitoring,” Hardy said.
Despite these real-world difficulties, trial data continue to demonstrate the clinical benefits of Roctavian for hemophilia A patients.
In the most recent update from the Phase 3 GENEr8-1 trial (NCT03370913) that supported Roctavian’s regulatory approvals, more than half of the participants were completely free of bleeds requiring on-demand treatment four years after receiving the gene therapy. Most patients (82%) remained off of routine preventive therapies, and life quality measures were also generally improved, Biomarin said.
Seven-year data from an earlier Phase 1/2 trial (NCT02576795) similarly demonstrated sustained declines in bleeding rates.
“We continue to be impressed by the durable and sustained bleed control demonstrated following treatment with Roctavian based on more than 500 patient years of observation in our pivotal program,” said Hank Fuchs, MD, president of worldwide research and development at Biomarin.
The effects of Roctavian are “a significant therapeutic achievement when considering the high burden of chronic treatment for people with severe hemophilia A,” Fuchs said.
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