Baby denied life-saving drug by Louisiana Medicaid
BATON ROUGE - The most expensive drug in the world could be a possible cure for a 16-month-old boy.
Axel Dennis was diagnosed with Spinal Muscular Atrophy Type 1 at five weeks old. SMA is a genetic disease the affects muscle movement. Axel's mother, Andrea James, says her son's muscles aren't getting messages from his brain.
"We were told one to two years and bring him home and enjoy him the best we could, but there were no guarantees of his life span," James said.
In January, he lost his ability to smile.
Earlier this year, the FDA approved Zolgensma for children with SMA under the age of two. The infusion halts the disorder in its tracks and doesn't allow it to progress further. The single-dose drug has been praised by the SMA community but it's incredibly expensive at more than $2 million.
Axel's Louisiana Medicaid has refused to cover the cost.
"We've been denied twice and now we're applying for the State Fair Hearing," James said. "I think that they just don't want to pay the price."
In his treatment denial letter, it says Axel doesn't meet certain criteria to receive the dose. James says it's because he has a trach tube, which is used to help expand Axel's lungs.
It's now a race against time. The family has eight months before Axel turns two. James has heard and seen what the drug can do and wishes it for everyone.
"The money's been spent to prove that it'll benefit these kids," she said. "It will save their lives, as close to a cure as we've come and as what anything could be really. I say to the state, save his life, give him a chance to walk, give him a chance to earn his smile back."
Two On Your Side reached out to the Louisiana Department of Health regarding Axel's Zolgensma denial.
LDH says its heart goes out to parents who learn their child has a life-threatening disease such as spinal muscular atrophy, or SMA. Louisiana Medicaid covers all treatments for SMA that have been approved by the federal Food and Drug Administration and also reviews cases to make sure that no child is denied care if they meet the medical necessity requirements. For the newest treatment, Zolgensma, the FDA developed a guideline to make sure that the treatment is only used in children where it will be safe and effective.
LDH says in this situation, its medical professionals have extensively reviewed the case using the FDA’s guidelines for Zolgensma. It says the child’s Healthy Louisiana plan has contacted the parents and informed them of whether this treatment would be safe and effective for their child based on the FDA guidelines.
While Axel's family fights for his life, he continues his treatment with Spinraza. Spinraza, approved by the FDA in 2016, is given every four months through spinal injection. Each time, Axel must go under anesthesia to receive the treatment. It can often be incredibly traumatizing.
James and the rest of Axel's family are hoping someone will listen and get him the best treatment available. A GoFundMe has been set up for Axel here. To learn more about Axel and SMA, visit his Facebook page.