EMA Speeds Approval Process for Hemophilia B Gene Therapy

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

Share this article:

Share article via email
hemophilia gene therapy | Hemophilia News Today | CHOP honors researcher | DNA illustration

The European Medicines Agency (EMA) has approved an accelerated assessment request for etranacogene dezaparvovec, an experimental gene therapy for hemophilia B.

The decision means that, once an application is submitted seeking approval for marketing authorization of etranacogene dezaparvovec, it will be reviewed more quickly than normal — which could allow patients in Europe to access the therapy sooner, according to a press release.

The EMA grants accelerated assessment to products that are expected to be of major interest for public health and that represent a therapeutic innovation.

“The acceptance of an accelerated regulatory review underscores the high unmet need for a long-term treatment option for haemophilia B patients,” said Karen Pinachyan, head of medical affairs Europe at CSL Behring. The biotechnology company secured global marketing rights to etranacogene dezaparvovec earlier this year from the therapy’s original developer, uniQure.

CSL Behring said it is planning to apply for the therapy’s approval in both Europe and the U.S. in the first half of next year.

Recommended Reading
hemophilia b gene therapy | Hemophilia News Today | 2nd patient group being dosed B-LIEVE trial | clinical trials illustration of graphs

HOPE-B Trial Update: Gene Therapy Candidate AMT-061 Meeting Goals

Hemophilia B is caused by mutations in the F9 gene, which provides instructions for making the clotting protein factor IX, known as FIX. Etranacogene dezaparvovec, previously called AMT-061, is designed to deliver a copy of FIX-Padua, a highly functional version of the F9 gene, to a patient’s cells.

The gene therapy is given via a single infusion into the bloodstream, and it delivers its genetic payload using a specially engineered virus called an adeno-associated virus (AAV). AAV is commonly used in gene therapies because it does not carry substantial safety risks and is easy to work with in a lab setting.

The ongoing Phase 3 HOPE-B clinical trial (NCT03569891), sponsored by uniQure, enrolled 54 men with moderate to severe hemophilia B who were treated with etranacogene dezaparvovec.

New data from the trial showed that, at 18 months after treatment, the men’s bleeding rates decreased significantly — from more than four bleeds per year to less than two, on average. Levels of clotting factor IX activity also increased substantially.

“These encouraging results illustrate the potential that gene therapy has to be a long-term treatment option for patients living with haemophilia B and we look forward to sharing more detailed data with the medical community in the near future,” said Lutz Bonacker, senior vice president and general manager Europe at CSL Behring.

The investigational therapy was generally well-tolerated in the HOPE-B study, and 80% of reported adverse events (side effects) were considered mild.

“Consistent stable Factor IX expression, observed at 6 months, 12 months and now the 18-month final analysis of the HOPE-B pivotal trial, is preparing the ground for future approvals and implementation in clinical practice,” said Wolfgang Miesbach, MD, PhD, a professor at the University Hospital of Frankfurt, in Germany, and an investigator for HOPE-B.

In addition to the new accelerated assessment, etranacogene dezaparvovec has been given Priority Medicine (PRIME) designation by the EMA. It also received breakthrough therapy designation by the U.S. Food and Drug Administration.

“It would be essential that a collaborative effort between all stakeholders is made to bring this innovation to patients,” Miesbach concluded.