Sweden approves subsidy for children’s achondroplasia medicine

Children with achondroplasia will gain a subsidised medicine in Sweden from 1 November, after the medicines-pricing regulator accepted a higher treatment cost for the very rare condition.

TLV announced on Tuesday that Voxzogo will enter the pharmaceutical benefits scheme. The treatment is approved from four months of age while the skeleton is still growing.

Achondroplasia is a genetic condition that affects bone growth and causes disproportionate short stature. It can also cause serious complications, including pain, mobility difficulties and breathing problems. TLV assessed its severity as very high.

Voxzogo contains vosoritide and is given by daily injection. The regulator says no other approved treatment is available for this condition and assessed the medicine alongside existing supportive care.

The decision reflects TLV’s newer approach to medicines for rare conditions. It accepted a higher cost than it would usually consider reasonable for a very severe condition because achondroplasia is very rare and the treatment offers clinically relevant benefit.

A rebate agreement between manufacturer BioMarin and Sweden’s regions reduces the treatment cost and forms part of the decision. The published list price does not represent the negotiated net cost or an individual patient’s bill.

TLV can revisit the subsidy if the conditions underpinning the agreement change.

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