BioMarin Could Launch Brineura Concurrently In US And Europe
Executive Summary
The EU’s top advisory panel, the CHMP, has given a green light to BioMarin’s late-infantile Batten disease therapy, Brineura, one of the first therapies for the ultra-rare and fatal disease, a decision that comes just days before a US FDA decision is expected on the replacement enzyme therapy.
A positive EU CHMP opinion for the use of BioMarin Pharmaceutical Inc.’s Brineura (cerliponase alfa) in CLN2 disease, the late-infantile form of Batten disease, has come just days before a US PDUFA date of April, 27, 2017 for the product, suggesting US approval for the drug could be closely followed by European approval and near-concurrent launch of the product on two major markets.
BioMarin says it is ready to commercialize the orphan drug; “We will be able to commercialize Brineura within two weeks of EU approval, and in the US within six weeks of approval, setting the company up for concurrent launches in both regions,” said company chairman and CEO Jean-Jacques Bienaime. The European good news contrasts with the disappointment a year ago when the US FDA rejected the company’s potential muscular dystrophy drug, Kyndrisa (drisapersen); the company discontinued development of drisapersen in June 2016.
Bienaime told analysts he expected Brineura to be priced at the “high end” for an ultra-rare indication, without giving further details, and would be an important contributor towards the company’s goal of growing revenues by 15% through to the end of the decade. He was speaking during an analysts’ call held on April 21, after the release of the CHMP’s recommendation.
European approval usually comes within a couple of months of a positive opinion, and is expected by the end of the 2017 second quarter, one quarter earlier than expected. Brineura is one of the first products to use the revised EU process for accelerated assessment, and is one of the first therapies for the ultra-rare and fatal brain disorder, neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency or late-infantile Batten disease, the company noted.
Under Four Years In Clinical Trials
Cerliponase alfa is a recombinant replacement therapy for the enzyme deficient in CLN2 disease, tripeptidyl peptidase 1, and is intracerebroventricularly administered every 14 days. The first child was treated less than four years ago, and the EU approval recommendation was based on an open-label dose-escalation study in 24 patients aged between three and six years of age, and an open-label extension study. The CHMP is recommending a full approval under exceptional circumstances.
The commercial strategy for Brineura will involve “raising awareness of the disease to more physicians, both geneticists and pediatric neurologists, so as to facilitate early diagnosis,” Bienaime remarked during the call. “The goal is to have patients screened and diagnosed early while they retain a good level of function, as these will be the best candidates to benefit from Brineura.”
The CHMP has included all ages in its labeling recommendations, and the company has agreed to collect additional data on patients from birth to two years of age, and to maintain a patient registry. BioMarin’s clinical study to achieve the approval recommendation was in children aged three years or older; 20 of 23 patients who received Brineura for 48 weeks had either a slower than expected progression of the disease, a stabilization of the progression of the disease, or some improvement in their motor and language abilities. 15 out of 23 Brineura-treated patients had no overall decline in CLN2 score irrespective of baseline score, and two patients increased their score by one point.
Company executives reported the US FDA review of Brineura was substantially completed and they were in labeling discussions with the regulators. Credit Suisse analysts noted there are around 1,200-1,600 patients in the world with Batten disease, and Brineura has sales expectations of around $500m annually at its peak.