Blog authored by:
Flavio Dormont, PhD, MBA, Therapeutic Area Head of Clinical Development RWE, Sanofi
Francesca Frau, PhD, Development Real World Evidence Lead, Sanofi
Drew Roberts, PharmD, PhD, Senior Director, Science, Aetion
How can real-world evidence support a label expansion for adolescent patients experiencing chronic rhinosinusitis with nasal polyps?
On September 13, 2024, Regeneron Pharmaceuticals and Sanofi announced that the U.S. Food and Drug Administration (FDA) approved Dupixent® (dupilumab) for adolescents aged 12 to 17 with inadequately controlled chronic rhinosinusitis with nasal polyps (CRSwNP). This approval, granted under Priority Review, follows the initial approval in 2019 for adults with CRSwNP and marks a significant advancement in treatment options for the adolescent population with this condition.
CRSwNP, driven by type 2 inflammation, can severely impact quality of life, causing blocked nasal passages, breathing difficulties, and reduced sense of smell. While standard treatments like steroids and surgery can provide relief, many adolescents—about 9,000 in the U.S.—continue to struggle with symptoms. Dupixent has shown effectiveness in alleviating these symptoms, decreasing the need for corticosteroids or surgery, as evidenced by pivotal trials (SINUS-24 and SINUS-52).
This expanded indication is a game-changer, making Dupixent the first biologic specifically approved for adolescents with CRSwNP. It solidifies Dupixent’s role as a leading treatment for conditions related to type 2 inflammation, including asthma and atopic dermatitis.
Harnessing Real-World Evidence
Real-world data (RWD) was instrumental in supporting this label expansion. By leveraging de-identified claims data from the Optum Clinformatics Data Mart (CDM)—Sanofi demonstrated similarities between the adolescent and adult CRSwNP patient populations in terms of baseline characteristics, CRSwNP disease course, and CRSwNP treatment patterns in routine care. This real-world evidence (RWE) was paired with clinical findings showing comparable Dupixent pharmacokinetic and pharmacodynamic (PKPD) profiles between adolescent and adult patients, while Dupixent’s established safety profile reinforced the positive benefit-risk ratio for this younger population.
Aetion’s Support
Using Aetion® Substantiate, built on the Aetion Evidence Platform®, this study emphasized transparency, reproducibility, and speed, delivering regulatory-grade evidence. Sanofi and Regeneron Pharmaceuticals came to Aetion to support the study design and execution using fit-for-purpose Optum CDM data, already connected to Substantiate. The Aetion Science team quickly developed and iterated the study to meet regulatory objectives, incorporating feedback throughout rapid study execution.
Following the delivery of the results, Aetion provided an expert review of key sections in the FDA submission to ensure accuracy and help optimize the framing of RWD for this regulatory purpose. Aetion's depth of experience in using RWE aided in the successful support of the regulatory submission.
With this robust evidence base, Sanofi can confidently offer a new treatment option for adolescents with CRSwNP, paving the way for improved health outcomes in this vulnerable population. Speaking about the experience of collaborating on this study, Development Real-World Evidence Lead at Sanofi Francesca Frau remarked, "It has been a pleasure collaborating with the Aetion team on this project. Leveraging RWE has been key to the timely approval of Dupixent for treating CRSwNP in adolescents, making a significant impact on their lives.”
Interested in how Aetion’s experts can support you in your own label expansion or other regulatory-grade activities? Contact us here.