FDA Issues Voucher for Rare Disease Gene Therapy
The FDA has issued a Priority Review Voucher for WASKYRA (etuvetidigene autotemcel), a gene therapy for a rare pediatric disease. The voucher system is designed to incentivize pharmaceutical companies to invest in developing treatments for uncommon conditions that might otherwise be overlooked.
- WASKYRA treats Wiskott-Aldrich syndrome (WAS), a rare genetic disease caused by a mutation in the WAS gene that almost exclusively affects males. The condition impairs the function of immune cells and platelets, leading to frequent infections, eczema, and an increased risk of bleeding and certain cancers. - The therapy is a one-time, personalized treatment. A patient's own blood stem cells are collected and, in a lab, a modified, harmless virus (a lentiviral vector) is used to deliver functional copies of the WAS gene into them. These genetically corrected cells are then infused back into the patient. - A Priority Review Voucher can be sold to another company. These vouchers have previously been sold for prices ranging from $67 million to $350 million, creating a significant financial incentive for companies to pursue treatments for rare diseases. - The developer of WASKYRA is Fondazione Telethon ETS, an Italian non-profit organization, making it the first cell and gene therapy from a non-profit applicant to be approved by the FDA. - The development of this therapy relied on tech-focused life science careers; for instance, bioinformaticians analyze the genetic data to understand the mutation, and computational biologists design the viral vectors for effective gene delivery. - Patient-facing roles are critical for delivering such a treatment. Genetic counselors educate families on the inherited condition and the therapy's implications, while clinical research teams manage the trials, monitor patients after the one-time infusion, and collect data on safety and efficacy.
