PPMD is excited to learn that the FDA has granted accelerated approval to Vyondys 53 (golodirsen) injection to treat Duchenne patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping. With the approval of VYONDYS 53, another 8% of Duchenne families will have a therapy we hope will continue to slow the progression of Duchenne.

What Does Accelerated Approval Mean?

The FDA Accelerated Approval Program was established in 1992 to allow for products that meet regulatory rigor based on efficacy of a surrogate endpoint that is reasonably likely to have clinical benefit for select patient communities in which there is significant unmet need.

By definition, limited clinical data for products approved through accelerated approval will exist at the time of approval and in early stages of the Phase 4 environment. In these circumstances, this lack of clinical data is not a reflection of the robustness of the therapy, but rather the regulatory review pathway agreed upon by the product developer and the Food and Drug Administration.

VYONDYS 53 is now an approved therapy and is no longer considered an investigational product.

VYONDYS 53 has been approved utilizing the Accelerated Approval pathway on the surrogate outcome measure of dystrophin production and the assessment by regulatory experts that dystrophin production in a disease caused by its absence is ‘reasonably likely to have clinical benefit’.

This demonstration of clinical benefit will depend upon product availability to amenable patients enabling the collection of data on long term outcomes. PPMD and the Duchenne clinical expert community – as convened by the leaders of the Certified Duchenne Care Centers – assert that coverage of a drug must reflect the FDA approval status of a drug.

Navigating Access

Following the approval of EXONDYS 51, PPMD launched an Access & Coverage Resources section of our site in order to inform the community about the process of accessing approved therapies for Duchenne. Upon the commercial launch of Emflaza by PTC, the site was expanded to include information about access to Emflaza. In addition, we have recorded webinars about navigating the access ecosystem and the many stakeholders involved in this new access and coverage landscape. These webinars feature speakers with decades of experience in payer, pharmacy, access, and reimbursement issues, and provide community members with a deep understanding of access considerations, an overview of current policy issues with potential impact coverage, and tools to empower personal access.

We will immediately begin folding VYONDYS 53 into our resource pages in an effort to curate answers to our community’s questions regarding access, coverage, and pricing. PPMD is committed to working towards community access to all approved products and will continue to work with all relevant stakeholders to ensure a favorable access environment for our community.

For 25 years, PPMD has been working with researchers, clinicians, industry and the Duchenne community to find treatments for all people living with Duchenne. And while we need to ensure that these approved therapies are accessible and affordable for patients, today we celebrate this approval and thank Sarepta for their continued leadership in the fight to end Duchenne.