BioMarin Pharmaceutical Inc's new drug application for drisapersen as a treatment for Duchenne muscular dystrophy has been rejected by the FDA.
BioMarin Pharmaceutical Inc’s new drug application for drisapersen (Kyndrisa) as a treatment for Duchenne muscular dystrophy has been rejected by the FDA.
The pharmaceutical company announced it received a complete response letter on January 14, 2016.
In the letter, the FDA stated that Kyndrisa was not ready for approval in its current state because there was not enough substantial evidence of its effectiveness.
BioMarin said it would work with the FDA moving forward to determine the next steps.
Ongoing Kyndrisa extension studies are expected to continue, and patients receiving Kyndrisa BMN 044, BMN 045, and BMN053 will remain on therapy.
At the moment, there is no FDA-approved therapy to treat Duchenne, which affects around 1 in every 3500 to 5000 male children and is the most common fatal genetic disorder diagnosed in childhood.
Patients affected by Duchenne muscular dystrophy begin to show muscle weakness between the ages of 2 and 5, and the disease can eventually weaken the skeletal or voluntary muscles in the arms, legs, and trunk.
Marketing approval for Kyndrisa is currently under review in the European Union.
BioMarin said it expects to receive a response from the European Medicines Agency in the first half of 2016. A final decision is expected to come from the European Commission before the end of the year.