Croatia is completing preparations for the introduction of screening of newborns for spinal muscular atrophy (SMA), after the government recently gave consent to the KBC Zagreb hospital for the procurement of medication for SMA at the expense of the state budget in the period 2023-2027.

Evidence shows that early diagnosis through newborn screening and early intervention with available treatments lead to better outcomes.

“In the coming months Croatia will introduce the newborn screening for SMA. All technical and organisational preparations are nearing completion,” the KBC Zagreb has informed Hina.

Newborn screening identifies conditions that can affect a child’s long-term health or survival. Early detection, diagnosis, and intervention can prevent death or disability, and enable children to reach their full potential.

The medicine, onasemnogene abeparvovec (Zolgensma), is used as gene therapy for spinal muscular atrophy, a rare and serious condition that causes muscles to be weak and waste away.

Zolgensma is given once as an infusion (drip) into a vein.

The total amount for the procurement of the medicine, slightly more than HRK 35 million (VAT included), is supposed to be paid in six installments, and the contract also stipulates the monitoring of the use of the medicine and a revision of contractual obligations related to the payment of the final three installments if the medicine does not have the expected effect, said Health Minister Vili Beroš in early November.

Currently there are 47 children in Croatia diagnosed with SMA and 57 adults registered as SMA patients.

Apart from Zolgensma, also Spinraza (nusinersen) and Evrysdi (risdiplam) are used in the treatment of SMA. They are special medications, and the state budget allocated HRK 100 million for the use of these medications in 2022. Spinaraza is directly delivered to the central nervous system (CNS) where motor neuron loss begins. After 4 initial loading doses, Spinraza is given 3 times a year.

Evrysdi (risdiplam) is an oral daily therapy.

In Croatia, seven underage patients receive Spinraza, in three cases Zolgensma has been applied, and the remaining children with SMA receive Evrysdi. Of those 54 adults, 14 receive Spinraza, and 40 Evrysdi.

(€ 1 = HRK 7.5)