Skysona’s Indication Narrowed
On Aug. 8, 2025, the US Food and Drug Administration (FDA) restricted Bluebird Bio’s Skysona® (elivaldogene autotemcel) following reports of blood cancers. Skysona is a one-time gene therapy for cerebral adrenoleukodystrophy (CALD), a rare genetic disorder, approved for boys aged four years to 17 years with early, active CALD.The therapy’s boxed warning for myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) was updated after 10 of 67 clinical trial participants (15%) developed blood cancers, up from 4% at initial approval, including one death. Cancer onset ranged from 14 months to 10 years post-treatment, with some cases occurring before therapeutic benefit. The indication is now restricted to patients who do not have a human leukocyte antigen (HLA)-matched donor for allogeneic hematopoietic stem cell transplant. The restriction also mandates quarterly blood counts and annual clonal assessments for life. Here is the updated prescribing information.
KETARx Approved for Surgical Pain Amid Ongoing Ketamine Shortage and Targets Broader Indications
On Aug. 8, 2025, the FDA approved KETARx™ (racemic ketamine – PharmaTher Holdings) intravenous (IV) injection for the treatment of surgical pain in adults. Initially approved as an anesthetic in the 1970’s, ketamine has also been utilized in the treatment of mood disorders, chronic pain and certain neurological conditions due to its rapid-acting and dissociative properties. KETARx is designed to provide a consistent supply of ketamine, addressing a long-standing shortage listed by the FDA since 2018. This shortage led to most ketamine being supplied through compounding pharmacies. PharmaTher has ongoing studies and plans to seek approval across multiple therapeutic areas, including depression, Parkinson’s disease, amyotrophic lateral sclerosis (ALS) and complex regional pain syndrome (CRPS). The global ketamine market is projected to reach $3.42 billion by 2034. Cost and launch details are not yet known.
Generics to Venofer Available
The FDA approved Viatris’ iron sucrose injection, a generic to IV Venofer® (iron sucrose – American Regent) on Aug. 11, 2025, for the treatment of iron deficiency anemia (IDA) in patients two years of age and older with chronic kidney disease (CKD). Although there were no patents or regulatory exclusivities remaining, this is the first generic available and holds 180 days of exclusivity. Amphastar’s generic was also FDA approved with launch expected later in 2025. Both Amphastar and Viatris’ iron sucrose injection will be available in 50mg/2.5mL, 100mg/5mL and 200mg/10mL single-dose vials. Per IQVIA, US sales for Venfor were $515 million ending June 30, 2025. Viatris is planning to launch immediately.
MedWatch Update
FreeStyle Libre Transition
Effective Sept. 30, 2025, Abbott announced they will discontinue the FreeStyle Libre 2 and FreeStyle Libre 3 sensors and transition patients to the updated FreeStyle Libre 2 Plus and FreeStyle Libre 3 Plus sensors. The FreeStyle Libre system is an FDA-approved continuous glucose monitoring (CGM) device indicated for the management of diabetes. It provides real-time glucose readings and is designed to replace fingerstick testing for treatment decisions. Patients currently using FreeStyle Libre 2 or Libre 3 sensors will require new prescriptions for the Plus versions to continue therapy. For more information, see the FreeStyle Provider Transition Page.