The U.S. Food and Drug Administration (FDA) granted an Accelerated Approval on Sept. 16, 2022, for Skysona® (elivaldogene autotemcel). It is approved to delay neurologic decline for males between the ages of four years old and 17 years old who have mild symptoms or who are still symptomatic for the rare condition, cerebral adrenoleukodystrophy (CALD). A progressive genetic disorder, CALD allows fatty-acid buildup that causes increasingly severe damage to myelin coverings in the brain and spinal cord. Patients lose mental and physical capabilities. To treat it, hematopoietic stem cells are harvested from the individual patient at qualified treatment centers – mainly children’s hospitals. The cells are enhanced with additional CD34+ cells altered to contain a protein that keeps fatty acids from accumulating and then infused after the patient undergoes extensive conditioning. At least 5.0 × 106 CD34+ cells/kg are needed for treatment to be effective. The wholesale acquisition cost (WAC) is $3 million for each one-time dose. It includes a boxed warning that using it may be associated with hematological cancers. Prescribing information for Skysona may be found here.

At a Glance

• Brand (Generic) Name: Skysona® (elivaldogene autotemcel). Previously, it also has been called eli-cel or Lenti-D
• Manufacturer: bluebird bio, Inc.
• Date Approved: Sept. 16, 2022
• Indication: to slow the progression of neurologic dysfunction in boys four years to 17 years of age who have early, active cases of CALD
• Dosage Forms Available: individualized suspension containing 5.0 × 106 CD34+ cells/kg or more for IV infusion
• Launch Date: before the end of 2022
• Estimated Cost: WAC for a one-time infusion of Skysona is $3 million.
• Typically diagnosed in patients between the ages of four years old and 10 years old, CALD is an inherited condition linked to X chromosomes.
• Annually, CALD is believed to affect an estimated 40 new babies, almost all boys, in the U.S. Around ten patients are expected to be candidates for treatment with Skysona, each year.
• Although boys affected by CALD generally develop normally until about two and one-half or three years of age, deposits of long-chain fatty acids in their central nervous systems (CNS) damage the myelin covering on nerve cells.
• Symptoms such as seizures and difficulties with learning and walking, gradually get worse. If not treated, approximately 50% of patients do not survive for five years after diagnosis.
• By replacing the malfunctioning gene, Skysona inhibits or potentially prevents further myelin damage. Full effectiveness may not be reached for as long as a year and one-half, however.
• In clinical trials, disability did not advance significantly for 92% of patients treated with Skysona after two years, as compared with 57% of those who did not receive treatment with it.
• Roughly one-tenth of patients who have CALD have a matched donor, usually a brother or sister, who is an appropriate match for a stem cell transplant.
• Other current treatment for CALD focuses on relieving symptoms with medications, physical therapy and special schooling.
• As outlined in a boxed warning and patient Medication Guides, a few patients given Skysona developed blood cancers that could be fatal. Complete blood counts and possible signs of cancer should be checked every six months after the administration of Skysona.
• Skysona does not affect adrenal deficiency associated with CALD.
• Because the approval is accelerated, further positive clinical trial results are needed before the FDA will approve Skysona, fully. Additionally, already-treated patients will be monitored for 15 years after their infusions.
• Skysona has Orphan Drug status and is a Breakthrough Therapy for CALD.
• Embarc Benefit Protection Strategy: Evernorth is in the process of reviewing the clinical efficacy and safety information in addition to the financial impact of bluebird bio’s newly approved gene therapy – Skysona– and further evaluation is needed. As such, we have not yet made a determination if Skysona will be added into Embarc Benefit Protection. The Embarc team will provide confirmation as soon as possible.