Questions Remain Regarding Access for Newly Approved Sickle Cell Treatments

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.”