Bill seeks to add to list of newborn screenings in Arkansas; disease now treatable, lawmaker says

FILE - Rep. Julie Mayberry, R-Hensley, addresses the House Committee on State Agencies and Governmental Affairs at the Arkansas state Capitol in Little Rock, Ark., Wednesday, Feb. 4, 2015.
FILE - Rep. Julie Mayberry, R-Hensley, addresses the House Committee on State Agencies and Governmental Affairs at the Arkansas state Capitol in Little Rock, Ark., Wednesday, Feb. 4, 2015.

An often fatal degenerative disease would be added to the list of genetic disorders for which newborns must be screened under a bill that cleared a legislative committee Tuesday.

Rep. Julie Mayberry, the sponsor of House Bill 1074, said the Department of Health hasn't been screening infants for spinal muscular atrophy, or SMA, because it wasn't treatable until recently.

The U.S. Food and Drug Administration approved Spinraza, the first treatment for spinal muscular atrophy, in late 2016.

Last year, federal Health and Human Services Secretary Alex Azar approved adding the disease to the list of disorders for which newborns should be screened.

Three states are already screening for the disorder, and 11 others are planning to add it, Mayberry, R-Hensley, said.

The disease is the leading cause of death from a genetic disorder for infants under age 2, she said, but all children treated with Spinraza before showing symptoms have survived.

[RELATED: Complete Democrat-Gazette coverage of the Arkansas Legislature]

"You all as legislators today have an incredible opportunity to save lives," Mayberry said.

Those speaking in support of the bill included the mothers of two children with disabilities caused by spinal muscular atrophy and one of the children's grandmothers. Josh and Crystal Turner of Benton whose daughter, Briley Faith, died of the disease when she was 2 months old, also spoke for the bill.

"We just feel it's a no-brainer," Crystal Turner said.

Ann Purvis, the Health Department's deputy director for administration, said three or four children are diagnosed with the disease in the state each year.

Children born with the disease lack a protein needed to keep alive a spinal nerve cell, known as an anterior horn cell, said Greg Sharp, a child neurologist and chief medical officer at Arkansas Children's Hospital.

As a result, the cells die, leading to debilitating and often fatal muscle weakness.

Spinraza, marketed by Biogen of Cambridge, Mass., targets a gene that isn't affected by the disease to help produce more of the protein, according to a website about the drug.

"If diagnosis is made at birth, we can implement therapy as early as possible and not only save a child's life, but cause them to have a more normal and productive life," Sharp said.

Amit Agarwal, a pulmonologist in Children's Hospital, said treatment with the drug costs about $750,000 the first year and $250,000 a year for subsequent years.

The Swiss drug company Novartis also has applied for FDA approval of a gene replacement drug designed to cure spinal muscular atrophy with a one-time treatment.

Mayberry said treatment of the disorder is already covered by private insurance plans and the state Medicaid program.

The bill wouldn't affect coverage for the treatment, but would require insurers to cover testing for spinal muscular atrophy without adding to patients' out-of-pocket charges.

Purvis said the testing would increase what the department charges the hospitals by $9.10. Currently, testing costs $121 per sample. Hospitals pass the charges along to patients or their insurance plans.

Blood samples for testing are collected at hospitals by pricking the heel of the baby and collecting drops of blood on a piece of filter paper. The samples are then sent to the Health Department's laboratory in Little Rock.

The House Committee on Public Health, Welfare and Labor recommended passage of the bill in a voice vote, with no members dissenting. Mayberry said she expects it to go to the full House on Monday.

Calendar

The calendar of public events of the 92nd General Assembly for today, the 10th day of the 2019 regular session.

COMMITTEES

9 a.m. Joint Budget Committee, Room A, Multi-Agency Complex.

10 a.m. Senate Education Committee, Room 207.

10 a.m. Senate Judiciary Committee, Room 171.

10 a.m. Senate Public Health, Welfare and Labor Committee, Room 272.

10 a.m. Senate Revenue and Taxation Committee, Old Supreme Court chamber.

10 a.m. House Aging, Children and Youth, Legislative & Military Affairs Committee, Room 130.

10 a.m. House City, County and Local Affairs Committee, Room B, Multi-Agency Complex.

10 a.m. House Insurance and Commerce Committee, Room 149.

10 a.m. House State Agencies and Governmental Affairs Committee, Room 151.

Noon. House Rules Committee, Room B, Multi-Agency Complex.

3 p.m. Senate Judiciary Committee, Room 171.

3 p.m. Joint Committee on Energy, Room 149.

SENATE

1 p.m. Senate convenes.

HOUSE

1:30 p.m. House convenes.

A Section on 01/23/2019

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