Questions Remain Regarding Access for Newly Approved Sickle Cell Treatments

December 14

The recent approvals of the first two gene therapies for sickle cell disease represent potential major breakthroughs for patients who have faced significant burdens associated with the condition. However, there are still questions about how and whether payers will cover the high-cost treatments and how many patients will be open to taking the new treatments.

The FDA on Dec. 8 approved Casgevy (exagamglogene autotemcel) from CRISPR Therapeutics and Vertex Pharmaceuticals Inc. and Lyfgenia (lovotibeglogene autotemcel) from bluebird, Inc. for patients who are 12 or older and have recurrent vaso-occlusive events (VOEs), the term for severe pain and organ damage. The agency noted about 100,000 people in the U.S. have sickle cell disease and that VOEs “can lead to life-threatening disabilities and/or early death.”

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Tim Casey

Tim has been a reporter and editor for newspapers, websites and magazines for more than 20 years, including 10 years covering health care business topics. He has a deep knowledge of the managed care industry and pharmacy benefit management. He also has experience covering medical conferences and clinical and legislative health care issues. In 2014, the Society for Advancing Business Editing and Writing selected Tim as one of 15 journalists to participate in a national symposium on the Affordable Care Act. Tim has a B.A. in Psychology from the University of Notre Dame and an M.B.A. from Georgetown University.