The innovation factor of delivery and biologics, the ultra-rare nature of the disease, and the facilities required to manufacture the drug were all taken into account when pricing Brineura, the company said.
Brineura (cerliponase alfa) is an enzyme replacement therapy delivered via brain fluid infusion.
The treatment will launch in the US in early June.
Brineura is the first FDA-approved treatment for paediatric patients three years of age and older with CLN2 disease. It is administered into the celebrospinal fluid (CSF). The total process, to be completed every second week, takes 4.5 hours.
The infusion passes through a specific surgically implanted reservoir and catheter in the head (intraventricular access device).
Ultra-rare CLN2 disease
Ultra-rare CLN2 disease
CLN2 is categorised in the neuronal ceroid lipofuscinoses (NCLs) disease group, known as Batten disease. The condition is also known as tripeptidyl peptidase-1 (TPP1) deficiency. It is an ultra-rare, inherited disorder, which affects the nervous system.
The disease inhibits the development of essential motor skills, signs and symptoms of which typically become apparent between ages two and four. Affected children develop muscle twitches and vision loss, and typically do not live pas their teenage years.
CLN2 disease affects less than one million US residents.
Brineura is the only enzyme replacement therapy delivered via brain fluid infusion.
The treatment is clinically proven to slow the loss of essential motor skills of CLN2 disease.
The US Food and Drug Administration (FDA) has asked BioMarin to further evaluate the safety of Brineura in CLN2 patients below the age of 2 years. A long-term safety study will monitor Brineura treated CLN2 patients for a minimum of 10 years.
BioMarin CEO Jean-Jacques Bienaimé said the company hopes the subsequent monitoring of CLN2-treated patients will mean that in the future, all age groups will benefit from Brineura.
$702k price tag
Brineura has a wholesale price tag of $27,000 (€24,740) per 300mg carton. The recommended bi-monthly infusion increased the price to $702,000 annually.
BioMarin is offering a no cost support service via its RareConnection programme, a focus of which will be to inform patients and families about financial assistance programmes for the CLN2 treatment.
BioMarin Executive Vice President and Chief Commercial Officer, Jeff Ajer, expects that mandated discounts will reduce Brineura’s cost to around $486k (444k) per patient, per year.
BioMarin expects 35-60% of CLN2 patients to be eligible for Medicaid, he said.