Jun 01, 2020

Dear SMA community,

In January we launched a global Managed Access Program (MAP) for Zolgensma® (onasemnogene abeparvovec). Under this program, we are making up to 100 doses available in 2020 to eligible patients with SMA under the age of two in countries where Zolgensma is not approved. Already, the program has enabled children across Asia, Australia, Europe and North America to receive treatment who would not have otherwise had access to the therapy.

With only a single production location currently licensed to produce Zolgensma, we committed to offering the global MAP as soon as we had the available doses to do so. To our knowledge, the Zolgensma managed access programs represent the first time that a one-time transformative therapy is being made available to patients prior to regulatory approval via a free program. When designing the program last year, we recognized it would be challenging to implement due to supply constraints, logistical hurdles and the complexity of gene therapy manufacturing. Since then we’ve also encountered the challenges presented by the COVID-19 pandemic, which has caused institutions to temporarily lockdown due to the pandemic.

More importantly than the challenges of administering the program, we asked ourselves whether it was appropriate to implement a global MAP that could only offer a limited number of doses. We knew this would be an emotional issue but we believed that, even with limitations, putting in place a program to help SMA-affected children was the right thing to do.

We based the program design on three principles: fairness, clinical need and global accessibility. In close partnership with bioethics advisors, we debated questions such as: Do we offer Zolgensma first to patients for whom no other option is available or effective? Only to the youngest patients (who in theory stand to benefit most), or to patients about to age out of the program prior to a pending regulatory approval? Should we simply say, ‘first-come, first-served’?

The most difficult decisions were around the program’s allocation system, especially when you consider regional differences in access to therapies, healthcare infrastructure and cultural norms. We believed it was important to empower the discretion of clinicians in determining who would be eligible for the program, factoring available treatments and the patient’s health status. Under conditions where the program is over-subscribed, we wanted to ensure that all eligible children had a chance to receive therapy, and did not believe it was appropriate for us to ascribe value to the life or anticipated benefit of one child over another. We also anchored heavily on the principle of “global accessibility,” avoiding any process that would favor children from more advanced healthcare systems and ensuring truly global access to therapy.

Strengthening the global MAP

The program launch was met with sincere appreciation in some regions of the world, and with criticism in others. We take the concerns of the community seriously, and in retrospect recognize that we had prioritized a sense of urgency over inclusiveness in rolling out the program – and for that, we sincerely apologize. On hearing the feedback, we committed to collecting more input, and engaged in extensive and deep discussions with additional SMA community members.



Based on what we’ve heard through dozens of conversations with advocates, physicians and patient families, we are adjusting some elements of the global MAP. Starting on July 6, 2020, we are now requiring physicians to attest that their patient does not have access to, or is not medically eligible for, available treatment alternatives. June 22, 2020 is the last day that patient registration forms under the existing treatment plan will be accepted. There will be a 2-week transition period where patient registration forms will not be accepted to allow time for us to process the existing registrations, focus on operational readiness and prepare for accepting the new registration forms.

In regard to allocation process, following extensive consideration, at this time we will continue offering Zolgensma on a blinded allocation basis in situations where the number of requests outweigh available supply. We understand that this approach has been a focus of concern. However, we have not found an alternative approach that would fully address the community’s concerns and meet the principles that underscore the program. Ultimately, we believe that the updated entry criteria are sufficiently narrow to establish medical eligibility and need, guided by clinician judgement, and that it is not appropriate for us to further prioritize which child is more deserving within this context.

Additionally, we have made several operational improvements to the program in line with the feedback we have received, and we streamlined and clarified the registration forms and process.

Committed to patients and the SMA community

Ground-breaking new paradigms such as gene therapy present huge challenges in how we ensure access to innovation. We reaffirm our commitment to creating multiple access pathways so that as many children as possible around the world can benefit from Zolgensma. For example, we are creating innovative programs that enable and incentivize early reimbursement, including by offering discounts and sharing risk with payers. We are also working closely with advocacy partners to enhance data collection via registries, accelerate newborn screening, and to conceive sustainable access solutions in low- and middle-income countries.



The global MAP is only one small step toward faster and broader access to Zolgensma. The other pioneering initiatives we are pursuing stand to deliver even greater impact for SMA, while paving the path for future gene therapies. We appreciate the passionate opinions on the global MAP that have led to the changes we are implementing. We intend to advance our broader set of access initiatives with focus and determination, while also learning from this experience and committing to engaging the community more proactively. Our goals are ambitious, and we can only achieve them by working together. We are grateful for the community’s input on the global MAP and look forward to collaborating on new programs that provide more promise for more patients.


The AveXis Team