Ireland’s HSE Faces Renewed Calls for Action Following NHS Deal for Critical Spinal Muscular Atrophy Drug

According to a story from, Ireland’s Health Services Executive (HSE) is facing renewed pressure from activists and patient advocates for a reasonable coverage decision regarding Spinraza, which is currently the only available treatment for spinal muscular atrophy that can reverse or halt the effects of the disease. Spinal muscular atrophy is ultimately lethal without treatment. These renewed calls come in the wake of a recent deal struck by the NHS which allows temporary access to the drug for the most severely affected patients.

About Spinal Muscular Atrophy (SMA)

Spinal muscular atrophy is a type of neuromuscular disorder in which the motor neurons are destroyed, leading to muscle wasting. In many cases, the disease is lethal. This disorder is linked to genetic defects of the SMN1 gene. This gene encodes a protein called SMN, and when not present in certain amounts, neurons are unable to function. There are different kinds of spinal muscular atrophy that are categorized by when symptoms first appear. These symptoms may include loss of reflexes, muscle weakness and poor muscle tone, problems with feeding and swallowing, developmental delays, respiratory muscle weakness, tongue twitching, and a bell shaped torso. The most effective treatment currently available for the disease is called Spinraza. To learn more about spinal muscular atrophy, click here.

Spinraza is Expensive

The main obstacle to coverage for the HSE is the extremely high price of the drug. In effect, the HSE determined that the cost of the drug would not be worth the benefit. There are around 26 known patients in the country that would benefit from the use of the drug. TD Alan Kelly of the Labour Party, along with some other politicians, have expressed strong support for covering the drug.

Senator Rose-Conway Walsh says that the price appears to have lowered, even in the latest offer to the HSE. This signals that Biogen, the manufacturer of Spinraza, is willing to negotiate. The company plans to submit a proposal to the HSE that would be similar in nature to the deal struck with the NHS. Since its introduction, thousands of spinal muscular atrophy patients around the world have benefited from the use of the drug.

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