BioMarin Pharmaceutical Inc. BMRN announced that the European Commission has approved Brineura (cerliponase alfa) for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2).
CLN2 is also known as tripeptidyl peptidase 1 (TPP1) deficiency, a form of Batten disease and ultra-rare Brain disorder in children. Brineura is the first approved treatment in the EU for CLN2 and the drug can be administered to children of all ages since birth.
The approval does not come as a surprise since the Committee for Medicinal Products for Human Use (CHMP) had given a positive opinion on the same in Apr 2017, following an accelerated review procedure.
So far this year, BioMarin’s share price has risen 8.6%, better than a gain of 1.0% for the Zacks classified Medical - Biomedical and Genetics.
The recent European approval was based on data from an open-label, dose-escalation study evaluating Brineura in children with CLN2 disease between 3 and 8 years of age along with an open-label extension study. We remind investors that the FDA had approved Brineura in Apr 2017 to treat CLN2 by slowing the loss of ambulation in symptomatic pediatric patients 3 years of age and older.
Per BioMarin’s press release, CLN2 disease is a rapidly progressing, fatal neurodegenerative disease that impairs the ability to walk and talk in the majority of affected children by the time they are around six years of age.The company estimates CLN2 disease to affect approximately one in 200,000, with nearly 1,200 to 1,600 children in the company’s commercial territories.
BioMarin’s orphan disease drugs – Vimizim and Kuvan – continue to do well, backed by strong underlying patient demand trends. The approval of Brineura in the U.S. and EU will further boost the growth prospects of the company.
Zacks Rank & Stocks to Consider
BioMarin currently carries a Zacks Rank #3 (Hold). Better-ranked stocks in the health care sector include VIVUS, Inc. , Zoetis Inc. ZTS and Regeneron Pharmaceuticals, Inc. REGN. VIVUS sports a Zacks Rank #1 (Strong Buy) while Regeneron and Zoetis carry a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
VIVUS’s loss per share estimates narrowed from 50 cents to 39 cents for 2017 over the last 30 days. The company posted positive earnings surprises in all the four trailing quarters with an average beat of 233.69%.
Regeneron’s earnings per share estimates increased from $10.17 to $10.52 for 2017 and from $10.90 to $12.10 for 2018 over the last 30 days. The company posted positive earnings surprises in two of the four trailing quarters with an average beat of 0.45%.
Zoetis’ earnings per share estimates increased from $2.32 to $2.34 for 2017 over the last 30 days. The company posted positive earnings surprises in each of the four trailing quarters, with an average beat of 9.82%.
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