January 12, 2016
By Alex Keown, BioSpace.com Breaking News Staff
SAN RAFAEL, Calif. – This might be the year that BioMarin Pharmaceuticals Inc. finally shows a profit. That’s what Jean-Jacques Bienaimé, BioMarin’s chief executive officer, told the San Francisco Business Times Tuesday night.
In an interview, Bienaimé said he expects this to be a good year for BioMarin with pending approval for its experimental treatment for Duchenne muscular dystrophy and also plans to file for approval of a treatment for late-infantile Batten disease, a genetic disorder that typically kills children by the time they reach 12 years of age. The company also opened a new North Bay research center that is expected to house 700 employees.
BioMarin is expecting the U.S. Food and Drug Administration to rule on its Duchenne muscular dystrophy treatment early this year and is hoping the full regulatory body will overlook a critical analysis an advisory committee gave drisapersen in November. The FDA granted drisapersen, also called Kyndrisa, Orphan and Fast Track status, as well as Breakthrough Therapy designation. Bienaimé told Reuters the drug is also up for approval in Europe, where there are approximately 5,000 patients eligible for the drug. The European regulatory body is expected to make a ruling on Kyndrisa this summer.
If the FDA rules against Kyndrisa, it would open the door for rival drugmaker Sarepta Therapeutics , which is developing its own treatment for DMD, eteplirsen. Sarepta is scheduled to appear before an FDA panel on Jan. 22. BioMarin beat Sarepta to the FDA with a New Drug Application filed in April for drisapersen. Sarepta filed its NDA for eteplirsen in June. Both drugs utilize “exon skipping” technology, which allows the damaged cells’ repair mechanisms to fix the specific genetic mutation that affects about 13 percent of individuals with the disease. Additionally, Pfizer is developing a myostatin inhibitor, PF-06252616 to treat DMD. The pharmaceutical giant is currently conducting a mid-stage clinical trial with results expected in early 2017. Pfizer’s drug may have a wider reach than the treatments being developed by Sarepta and BioMarin, which will only impact about 15 percent of patients with DMD.
If the drug is approved, it could mean big dollars for BioMarin, but even if it is rejected, Bienaimé told the Times BioMarin could pass the $1 billion revenue threshold this year—due to its orphan drug treatments.
This week, during the J.P. Morgan Chase Healthcare Conference, BioMarin announced interim results for its Phase II clinical trial for reveglucosidase alfa, a treatment for late-onset Pompe disease. Reveglucosidase alfa is a fusion protein of insulin-like growth factor 2 and acid alpha-glucosidase (IGF2-GAA). The drug replaces the enzyme (GAA) that prevents the glycogen build up that causes Pompe disease. Interim results show patients improved respiratory muscle improvements, which the company said may indicate a possible halt in decline or improvement in lung capacity and endurance. The positive results buoyed BioMarin’s stock, which shot up more than 3 percent, hitting a high of $96.64 on Monday. BioMarin’s stock has had its ups and downs over the past year, hitting a high of $149.13 in July.
BioMarin is expected to report Phase III results for pegvaliase, for the treatment of Phenylketonuria (PKU), a rare inherited disorder that “causes an amino acid called phenylalanine to build up in your body. BioMarin also has a number of other treatments for rare diseases in its pipeline, including Vimizim, its enzyme replacement therapy for patients with Morquio A Syndrome patients, experimental treatments for dwarfism and a gene therapy for hemophilia A, the Times said.
