The U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) for delpacibart zotadirsen (del-zota) for Priority Review in people with Duchenne muscular dystrophy (DMD) who have genetic variants that may be treated through exon 44 skipping.
According to an update from Avidity, a Novartis company, the FDA determined that the application was sufficiently complete to begin a substantive review. The application will also be evaluated under the Accelerated Approval pathway.
What Is Del-zota?
Del-zota is being developed as a potential treatment for people with DMD44, meaning patients whose specific genetic changes may be amenable to exon 44 skipping.
Exon skipping is a treatment approach designed to help cells produce a shorter but potentially functional form of dystrophin by helping the cellular machinery skip a specific exon during the processing of dystrophin RNA. Learn More: What is Exon Skipping Therapy?
However, the FDA has not yet approved del-zota. The current milestone means that the application has entered the FDA review process.
What Does Accelerated Approval Mean?
The FDA’s Accelerated Approval pathway can allow certain treatments for serious diseases to reach patients sooner when they address an unmet medical need. Approval can be based on a surrogate endpoint that is considered reasonably likely to predict clinical benefit, while additional studies are conducted to confirm that benefit. Read More: What does BLA mean?
This means that even if the FDA grants accelerated approval, further clinical research will still be important to confirm the expected clinical benefit.
Phase 3 SAFARI44 Trial
The BLA includes data from the Phase 1/2 EXPLORE44 and EXPLORE44-OLE clinical trials.
A global Phase 3 SAFARI44 trial has also been initiated as a confirmatory study. According to Avidity, the trial is intended to further evaluate the long-term safety of del-zota and help confirm its clinical benefit. The study will be conducted outside the United States.
The company said that the development program would not have been possible without the participation of patients, caregivers, families, advocacy organizations, investigators and clinical research teams. Learn More: Explore Duchenne Clinical Trials
What Happens Next?
The FDA will now conduct its review of the del-zota application. The September 8 announcement does not represent an FDA approval.
Avidity said it will provide additional updates as they become available and advised patients and families with questions about del-zota or the clinical trials to speak with their healthcare providers.
Are You Eligible for Exon 44 Skipping? Check Now!
