CRISPR therapy Casgevy earns second FDA nod, this time for beta-thalassemia
16 Jan 2024
Gene TherapyDrug Approval
The FDA greenlit a second indication for Vertex Pharmaceuticals and CRISPR Therapeutics’ gene therapy on Tuesday, more than two months earlier than the deadline for the agency’s decision. Transfusion-dependent beta-thalassemia (TDT) was added to the label of Casgevy (exagamglogene autotemcel), the first therapy approved in the US to use CRISPR/Cas9 gene-editing technology to treat the blood disorder.
Casgevy was initially approved in the US last month for sickle cell disease (SCD), and has also received clearances in the UK, Saudi Arabia, and Bahrain for both SCD and TDT. EU regulators are expected to decide whether to approve Casgevy for SCD and TDT next month. Now with these landmark decisions under its belt, investors are keeping an eye on the gene therapy’s rollout, as well as a conditioning agent in early development from CRISPR that could significantly increase the treatable patient population.
Slow and steady wins the race?
Analysts have anticipated a slow launch and little revenue over the next six months, given the uptake hurdles that gene therapies have historically faced, such as reluctant payer coverage and months-long treatment cycles – but they are ultimately optimistic of Casgevy’s treatment paradigm-changing potential.
Treatment with Casgevy requires several hospital stays to conduct blood transfusions, apheresis, chemotherapy, and a final infusion with the CRISPR-edited cells.
According to an analyst note from Jefferies, the Street remains mostly cautious on Casgevy’s launch given the time required to get patients on the treatment, and cited bluebird bio’s haemophilia gene therapy Zynteglo as an example of a similar treatment that was very slow to launch.
Vertex has nine authorised treatment centres (ATCs) in the US to administer Casgevy, which carries a price tag of $2.2 million, and intends to open a total of 50 by 2025, with plans for 25 in the EU. In the US, Vertex has also secured an outcomes-based contract with Synergie Medication Collective, which covers about 100 million people, to provide access to Casgevy.
Patient populations and demand
Across all the countries the gene therapy is approved in, Vertex estimates an addressable patient population of about 30,000 in SCD, and roughly 5000 in TDT.
However, an analyst from UBS pointed to a preclinical programme from CRISPR that could increase the treatable population by up to four-fold. The biotech is developing a targeted conditioning programme – an anti-CD117 antibody-drug conjugate (ADC) – that could replace the need for chemotherapy prior to infusion.
However, Alexis Leonard – a haematologist and assistant faculty member at St. Jude Children's Research Hospital – told FirstWord that she thinks only a "tiny fraction" of SCD patients will receive a gene therapy. For more, see KOL Views Q&A: Leading haematologist says patients, payers will decide winner of Casgevy, Lyfgenia in SCD.
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