AveXis’ OneGene Program to Smooth the Path for Families Wanting Zolgensma

More than 90% of rare diseases have no federally sanctioned treatment. So when news came of an approved new therapy for spinal muscular atrophy (SMA), Zolgensma, celebrations abounded. But as is often the case, questions soon followed: Where is the medicine available? How do we deal with insurance companies? Where do we turn for help?

To answer these questions and provide comprehensive support and guidance throughout the treatment process, Zolgensma’s developer, AveXis, created OneGene, a program the company is touting as “a team of highly trained and dedicated people who help ensure that every family has the support they need during their treatment journey and beyond.”

In fact, AveXis (now part of Novartis) lists connecting with OneGene as the second of five steps of treatment, right after “determining therapy eligibility,” and followed by “prepare for treatment day,” “treatment day,” and “after treatment with Zolgensma.”

AveXis declined to say how many U.S. families have signed with the program — or whether babies are being treated — although the therapy is reported to now be available at 36 administration sites in 24 states. Children’s Hospital of Philadelphia (CHOP) told SMA News Today that it expects to start commercial use of Zolgensma (onasemnogene abeparvovec-xioi) by month’s end.

This is how it works. To be eligible for Zolgensma, infants and toddlers must have an SMA diagnosis confirmed by a genetic test that indicates the SMN1 gene is missing or not working as it should. A patient’s physician must also perform an AAV9 antibody blood test to measure the amount of these antibodies in the child’s blood, among other tests. [Editor’s note: An article explaining these tests and why they are necessary is expected to soon follow this report.]

After the prescription and patient consent forms are received, a OneGene representative will phone the family to discuss resources available. Support is led by a designated patient resource manager, each family’s main contact. This person is to be available for expert guidance and emotional support at every step of treatment, and to answer questions about Zolgensma.

A field reimbursement manager will be provided to track delivery of Zolgensma to the treatment center, while a case coordinator acts as a go-between to connect families to needed resources, and assists doctors with insurance processes. The coordinator may also verify insurance benefits, and coordinate financial assistance programs for eligible patients.

Following treatment, the program will facilitate discussion between caregivers/families and care teams about long-term needs, possibly including additional therapies, accommodations, or other support to help manage and guide the patient. Zolgensma is not a cure for SMA; it cannot reverse damage done to motor neurons before treatment.

Just before treatment, children will be administered an oral corticosteroid for at least 30 days. Their liver function will be monitored for at least three months. Each child’s physician will determine when to gradually reduce the corticosteroid dose, and when to stop it.

“The OneGene program will also reach out periodically after treatment to offer support and answer questions,” the Zolgensma website states.

Asked whether all patients would be accepted into OneGene — provided they meet requirements such as a confirmed diagnosis and acceptable AAV9 antibody test, and necessary forms are filed by their prescribing physicians — AveXis did not provide a definitive answer.

In addition, while financial assistance is given as part of the program, it’s unclear whether a family’s ability to pay for treatment has anything to do with that family being accepted into OneGene.

A statement from AveXis’ communications office simply urged directing “caregivers, parents and professionals to OneGene to better understand the program and how it meets their individual needs.”

The list price for Zolgensma is $2.125 million, making it the world’s most expensive drug. Still, over a 10-year period, it’s half the price of Biogen’s Spinraza (nusinersen), which costs $750,000 the first year and $375,000 every year thereafter. An intrathecal (spinal) injection, Spinraza was approved in December 2016 for SMA patients of all ages. Zolgensma is the first and only gene therapy for pediatric SMA patients, approved as a one-time intravenous treatment.

AveXis has said it does not expect any family to pay the full price for this disease-modifying therapy. A “robust” portfolio of financial and non-financial support services, it added, is available via the company and other third parties to help with copays, blood work, travel, and other disease- and treatment-related costs.

AveXis has partnered with specialty pharmacy Accredo to offer a pay-over-time option of up to five years to states, small insurance companies, and select employers to help ease budget constraints, where necessary.

The game-changing gene therapy was approved by the U.S. Food and Drug Administration on May 24 to treat babies and toddlers, up to 2 years old, with all SMA types by a 60-minute intravenous (IV) infusion. It is designed to replace the function of the nonworking or missing gene (SMN1) that causes SMA, an autosomal recessive neurodegenerative disease that affects 1 in every 8,000 to 10,000 people. The disorder is characterized by progressive muscle weakness.

Zolgensma is currently only available in the United States. Requests for its approval are before regulatory officials in Europe and Japan.

Zolgensma’s arrival highlights the importance of newborn screening, say neurologists and researchers, some of whom were surprised by its broader U.S. label. AveXis had initially requested coverage for infants up to 9 months old with SMA type 1 — the most severe and common form of the disease. Its effectiveness in toddlers isn’t fully known, and a Phase 1 trial (NCT03381729) in patients up to age 5 (called STRONG) delivering the therapy by intrathecal injection is underway.

Several studies have shown that early treatment is critical to controlling SMA development and progression rates. The best approach has proven to be beginning treatment before the onset of muscle weakness and irreversible damage to nerve cells.

Currently, seven states — Indiana, Missouri, New York, Pennsylvania, Utah, Minnesota, and Vermont — routinely screen newborns for SMA. Some 16 other states have either passed laws requiring such screening, or are close to doing so.

Families interested in Zolgensma are asked to contact the OneGene Program weekdays, from 8 a.m. to 8 p.m. ET, at 855-441-4363 (855-441-GENE).