Waskyra (etuvetidigene autotemcel) is a new gene therapy approved to treat Wiskott-Aldrich syndrome (WAS), a rare inherited condition that affects the immune system and blood cells.People with WAS often have frequent infections, easy bruising or bleeding, skin problems like eczema, and a higher chance of developing autoimmune diseases or certain cancers.This condition is caused by a change in the WAS gene, which affects how blood and immune cells work.
Waskyra is the first gene therapy approved by the FDA for this condition.
It uses your own blood stem cells, which are collected and changed in a lab to fix the faulty gene.
These corrected cells are then given back to you through an IV. The goal is to help your body make healthy immune and blood cells again. Before Waskyra, the only long-term treatment was a stem cell transplant from a matched donor. But not everyone has a suitable donor, and the procedure works best when done early in life. This medicine offers a new option for people who don’t have a matched donor, especially young children who need early treatment.
Why Was It Approved? Waskyra was approved after studies showed it helped reduce serious symptoms of Wiskott-Aldrich syndrome (WAS). The treatment was tested in 27 people with severe WAS through two studies and an expanded access program. In the year after treatment, moderate to severe bleeding dropped by 60%, compared to the year before. Four years later, most people still had no serious bleeding. Serious infections also went down by 93% in the six to 18 months after treatment. The most common side effects included rashes, infections, a fever with low white blood cells, vomiting, and liver problems. One person died about 4 and a half months after treatment due to serious brain-related issues. Because WAS is a rare and serious condition with limited treatment options, the FDA used a flexible review process to speed up approval. Waskyra also received special designations, including Orphan Drug and Regenerative Medicine Advanced Therapy status, to help bring it to people faster.
What Do I Need to Know?Waskyra is given as a one-time IV infusion using your own blood stem cells that have been changed in a lab to fix the gene problem. Before getting the treatment, you’ll need to go through a process to collect your stem cells. Then, you’ll receive a type of chemotherapy to prepare your body for the infusion. This treatment is only for people who haven’t had a recent stem cell transplant or gene therapy and who don’t have a matched donor. After the infusion, you’ll need close follow-up to check for side effects. These may include low blood counts, infections, liver problems, and a rare risk of developing cancer later in life. You may also test falsely positive for HIV on some lab tests because of how the gene therapy is made. Waskyra is not recommended if you are pregnant or breastfeeding, due to risks from the chemotherapy used before the infusion. You also should not donate blood, organs, or tissues at any time after receiving this therapy. Talk with your health care provider to understand what to expect before and after treatment.
