PEARLAND, Texas Krista James is turning 2 years old this week. But unlike other toddlers, she cant walk, talk, or sit up on her own.

Krista has Spinal Muscular Atrophy, or SMA. The life-threatening condition severely impacts kids muscle movements.

(Doctors) said children with SMA dont live to be eight months or a year to two years, so its truly a blessing shes turning two Friday, said Misty James, the childs mother.

However, her upcoming birthday also means Krista is running out of time.

The FDA recently approved Zolgensma, a cutting edge gene therapy that treats the disease at the genetic level.

Children 2 years old and younger are eligible.

The treatment is priced at $2.125 million.

Despite her doctor telling Medicaid its what the toddler needs, the request for coverage was denied.

Last week when I got the third denial letter, I broke down, James said.

University of Houston professor Barbara Evans said the problem starts at the top, because jumping through the FDAs hoops to get approval can cost pharmaceutical companies billions of dollars per drug.

SMA is estimated to affect 400 babies per year. You take a high cost of development and divide it by 400. It leads to a high price, said Evans, Director of the Center for Biotechnology and Law at the University of Houston.

She explains it is a price that can even strain insurance companies budgets.

Zolgensma is sold by the Swiss drugmaker Novartis.

A spokesperson for AveXis, a Novartis Company, said in a statement, For any newly approved therapy, it takes time to set up agreements with commercial and government-based health plans and it is common for there to be an appeal process while insurers put coverage and utilization policies and procedures in place. To facilitate this, we are actively partnering with insurers to accelerate coverage decisions that will support access for eligible patients.

The statement added they are aware of Kristas situation and are in contact with multiple parties to help provide support and assistance.

In the meantime, Medicaid is covering a different, less expensive medicine for the little girl called Spinraza.

Spinraza comes with three spinal injections a year for the rest of her life.

Kristas family is grateful for the medication, but they believe this new drug can give their child the best shot at a normal life.

They feel helpless knowing their daughters future is in someone elses hands.

A spokesperson from Texas Health and Human Services confirmed an expedited fair hearing was held Thursday regarding Kristas case.

A decision, which can be appealed, is expected to be issued Friday, which is also Kristas second birthday.

To read the full statement from AveXis:

"The FDA approval of Zolgensma (onasemnogene abeparvovec-xioi), a gene therapy for spinal muscular atrophy (SMA) in pediatric patients less than 2 years of age, marked an important milestone within the SMA community. Understandably, many families are actively interested in accessing Zolgensma as soon as possible. Our goal is to support access for patients who need this one-time gene therapy. For any newly approved therapy, it takes time to set up agreements with commercial and government-based health plans and it is common for there to be an appeal process while insurers put coverage and utilization policies and procedures in place. To facilitate this, we are actively partnering with insurers to accelerate coverage decisions that will support access for eligible patients. We are pleased with our progress to date to support access for eligible children which includes many Medicaid state policies now in place, but our work with health plans is ongoing. While we cant discuss the specifics of any particular patient we are aware of this family and their efforts to access Zolgensma and we are in contact with multiple parties to help provide support and assistance."

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Originally posted here:
Pearland family fighting to get $2.1 million drug for toddler with rare genetic disease - KHOU.com

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