2019 global sales: $463 million
Generics expected: Oct. 1, 2020
BioMarin is gearing up for some big launches—especially its hemophilia A gene therapy—but on the flip side, it’s also getting ready to lose exclusivity for Kuvan.
First approved in 2007, Kuvan is used to lower blood phenylalanine levels for certain patients with phenylketonuria. It was the first medication ever green-lighted for the disease, which affects at least 50,000 patients in the developed world, BioMarin said in its annual SEC filing.
Kuvan generated $463 million globally last year, just shy of 30% of the company's overall revenue. The company didn’t report U.S. sales, but it noted that an increase in North American patients drove an increase for Kuvan revenues.
From 2014 to 2016, two generics companies filed applications with the FDA to market their copycats, and BioMarin sued for patent infringement, the filing says. Then, between 2015 and 2017, the companies settled. Now, BioMarin expects generic launches on Oct. 1, 2020.
Endo’s Par Pharmaceutical has an FDA-approved version, and Dr. Reddy’s has a tentatively approved version, according to FDA records.
Meanwhile, BioMarin continues to prep for its next stable of drugs and advance next-gen candidates. The company’s top pipeline prospect is valoctocogene roxaparvovec, a gene therapy intended to treat hemophilia A. Behind that is vosoritide for the treatment of children with achondroplasia.
The drugmaker is also advancing BMN 307, a gene therapy for the treatment of phenylketonuria.