Gene Therapy For Beta-Thalassemia: CRISPR Therapeutics/Vertex's Casgevy Secures FDA Nod Ahead of Schedule

Tuesday, the FDA approved CRISPR Therapeutics AG’s CRSP/Vertex Pharmaceutical Incorporated’s VRTX Casgevy (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited cell therapy, for transfusion-dependent beta-thalassemia (TDT) in patients 12 years and older.

The approval comes two months before the PDUFA date of March 30, 2024.

Vertex is engaging with experienced hospitals to establish a network of independently operated treatment centers (ATCs) throughout the U.S. to offer Casgevy to patients. 

Also Read: CRISPR Therapeutics’ Gene-Edited Oncology Pipeline To Focus On Expanded Use In Autoimmune Diseases.

Casgevy is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with sickle cell disease (SCD) or TDT, in which a patient’s hematopoietic stem and progenitor cells are edited at the erythroid-specific enhancer region of the BCL11A gene through a precise double-strand break. 

This edit produces high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells.

Citing Oppenheimer analyst, Reuters noted a “slow and steady launch” for the therapy and estimated combined peak sales of about $400 million.

“We think Casgevy’s profile will do fine for patients naive to therapy,” Reuters highlighted, citing the analyst.

In December, the FDA approved Casgevy and Bluebird Bio Inc’s BLUE Lyfgenia as the first cell-based gene therapies for sickle cell disease in patients 12 years and older. 

In November, the U.K. Medicines and Healthcare Products Regulatory Agency granted conditional marketing authorization for Casgevy for sickle cell disease and transfusion-dependent beta-thalassemia.

William Blair analyst is pleasantly surprised with earlier than expected FDA approval for Casgevy for TDT. 

William Bliar writes that Casgevy is a competitive product with blockbuster potential, especially when backed by Vertex’s exceptional commercialization proficiency in rare diseases. 

Vertex’s effective reimbursement agreement with Synergie Medication Collective and the prompt establishment of ATCs post-approval in December demonstrates the company’s adeptness in payer negotiations and high-level execution.

Price Action: VRTX shares closed at $437.49, and CRSP shares closed at $63.43 on Tuesday.