EC approves Biomarin rare genetic disorder drug

The European Commission’s decision rubber stamps a previous recommendation by the European Medicines Agency (EMA) for use of the enzyme replacement therapy, which received orphan drug status as part of its review.

This status provided BioMarin with certain development incentives and is down to the rarity of Morquio A syndrome, which is estimated to affect about 3,000 people in the developed world and causes progressive multi-organ dysfunction.

The symptoms of Morquio A syndrome can include heart and respiratory complications, leading to loss of endurance, with 30-45 per cent of people with the condition ending up in a wheelchair. People with Morquio A syndrome are also at risk of early death.

The disease is caused by a deficiency in the enzyme GALNS. Vimizim (elosulfase alfa) works by replacing this missing enzyme, and has demonstrated an ability to improve the endurance of patients during clinical trials.

Compared to placebo, people with Morquio A syndrome who were treated with a once-weekly infusion of 2 mg/kg of Vimizim for 24 weeks demonstrated they were able to walk an extra 22.5m during a six-minute walk test.

Dr Chris Hendriksz at Salford Royal NHS Foundation Trust, UK, said the approval was “fantastic news” for patients.

“The impact for patients will be significant as they are now able to slow the progression of this devastating disorder.”

The EU approval follows a similar recommendation made by the US FDA in February this year, where it was also assessed as an orphan drug.

Marketing applications are also underway in Brazil, Australia, Canada and Mexico.

Please view PMLive for original source.

Leave a Reply