FDA approves world’s most expensive drug, a hemophilia B treatment expected to cost $3.5M
CSL has announced that etranacogene dezaparvovec-drlb, the company’s new gene therapy for adult patients with hemophilia B, has been approved by the U.S. Food and Drug Administration (FDA).
The medication, to be sold and marketed under the name Hemgenix, will be the world’s most expensive medication with a price of $3.5 million per dose. The world’s priciest medication was previously onasemnogene abeparvovec-xioi, a gene therapy for spinal muscular atrophy sold as Zolgensma, according to a report from CBS News.
In its statement highlighting the new approval, CSL wrote this is the first single-use gene therapy for hemophilia B. Etranacogene dezaparvovec-drlb is approved for adult patients who currently use factor IX prophylaxis therapy, have a history of life-threatening hemorrhages or have “repeated, serious spontaneous bleeding episodes.”
According to research the FDA reviewed when making its decision, the medication can reduce a patient’s risk of annual bleeds and help them discontinue factor IX prophylaxis therapy. Read up on the HOPE-B clinical trial here.
“As part of our promise to patients, CSL is committed to delivering innovative and groundbreaking solutions to address unmet medical needs, and we are proud to introduce the next wave of breakthrough medicines for people living with hemophilia B,” Paul Perreault, CSL’s CEO and managing director, said in a statement. “We recognize and thank all trial participants, scientists and investigators—without whom this important achievement would not have been possible—and look forward to seeing the positive impact of Hemgenix on the hemophilia B community.”
“Today’s historic approval builds on our promise to put patients first in all that we do to discover, develop and deliver biotherapeutics and vaccines that meet their needs,” added Bill Mezzanotte, head of research and development and chief medical officer of CSL. “With Hemgenix, we now offer a comprehensive portfolio of innovative medicines for hemophilia B, giving people living with the condition more choice in treatments and better and more durable control over their disease.”
The lengthy development program for Hemgenix was led by uniQure. Matt Kapusta, uniQure’s CEO, described the approval as a “historic achievement.”
“We have always believed that gene therapy had the potential to provide transformative benefits to people living with hemophilia B and are excited that the hemophilia community will have a new, safe and effective treatment option available to them,” he added.
Michael has more than 18 years of experience as a professional writer and editor. He has written at length about cardiology, radiology, artificial intelligence and other key healthcare topics.