BioMarin Pharmaceutical Inc. is wasting little time getting to the market with its newly approved enzyme-replacement therapy, Naglazyme, for patients with mucopolysaccharidosis VI.
The FDA granted marketing approval for Naglazyme (galsulfase) six months after receiving the biologics license application, and the Novato, Calif.-based company expects to launch the product in 30 days.
"It's really a great day for patients of MPS VI," said Emil Kakkis, senior vice president of business operations, calling the FDA's approval "an important transformation for a population of people who have been sitting alone in the dark."
The FDA recommended changing the name of the drug, formerly Aryplase, and Naglazyme now becomes the first product approved for MPS VI. It previously was granted orphan drug status and earns seven years of market exclusivity. If approved in Europe, the product could receive 10 years of exclusive sales there. Kakkis said he expects the European regulatory agency to approve Naglazyme during the coming first quarter.
"I've been in the business in MPS diseases since 1991, and no one thought we'd get any treatments approved," he told BioWorld Today, and now "we've got a second product."
BioMarin received FDA approval in 2003 for Aldurazyme (laronidase), a treatment for MPS I that is partnered with Cambridge, Mass.-based Genzyme Corp.
Described as a lysosome disorder similar to MPS I or Gaucher's disease, MPS VI - also known as Maroteaux-Lamy syndrome - is a rare, genetic disease that causes a deficiency of an enzyme needed for the body to break down sugar. As a result, that sugar builds up in tissue and can affect lungs, liver and bones before ultimately causing death. Many patients with MPS VI die in their teens or early twenties.
Naglazyme is designed to replace the missing enzyme, "basically producing it and giving it back," Kakkis said. "The enzyme gets through the bloodstream, gets bound to cells and then taken up into the tissues, where it reverses some of the storage that's observed with complex carbohydrates."
BioMarin estimated that MPS VI afflicts about 1,100 patients worldwide, and the company already has identified more than 250 in preparation of the drug's launch. Kakkis said he expects the market to grow over time.
"The ability to make a diagnosis and identify patients is really driven by the presence of a treatment," he said. "Having our sales force out talking to doctors and making them aware will lead to earlier diagnoses" and earlier treatment.
The company has a 60-person U.S. sales force that primarily handles Orapred, an oral product for severe asthma, but also has been working on patient identification in both MPS I and MPS VI. In addition, Kakkis said, BioMarin is working with three medical science liaisons to set up sites for patients to receive weekly Naglazyme treatment in four-hour infusions.
BioMarin will market Naglazyme itself in the U.S. Though the company has considered taking on a partner for overseas sales, the relatively small patient population might make it "more beneficial for BioMarin in the long term to commercialize ourselves," Kakkis said, adding that the company's new CEO, Jean-Jacques Bienaime, has a background in sales and marketing and "believes we can take on the task."
As an enzyme therapy, Naglazyme is expected to be expensive, averaging between $200,000 and $300,000 per patient per year, but Kakkis said the company does not anticipate difficulty in getting the drug to patients since "other enzyme therapies have been reimbursed."
Once Naglazyme hits the market, it will be evaluated in a single post-market study in patients less than the age of 1 in skeletal dysplasia. A clinical surveillance protocol also will be in place to collect additional data on Naglazyme for doctors.
The FDA's approval was based on results of a Phase III trial involving 39 patients who received weekly intravenous infusions of either Naglazyme or placebo solution. After 24 weeks of treatment, patients receiving Naglazyme showed a statistically significant improvement in endurance compared to patients in the placebo group, measured by the change to baseline in the distance walked within 12 minutes. Naglazyme-treated patients showed further improvement following another 24-week period. (See BioWorld Today, June 4, 2004.)
Naglazyme is BioMarin's third approved product since the company was founded in 1997. Earlier this year, it began a Phase III trial of Phenoptin in phenylketonuria (PKU), and last month BioMarin signed a potential $257 million deal licensing to Geneva-based Serono SA overseas rights to Phenoptin and PKU drug Phenylase. (See BioWorld Today, May 17, 2005.)
BioMarin also recently settled its proxy contest with OrbiMed Advisors with respect to the election of directors at the company's annual stockholder meeting. Under the terms of the agreement, the nominees for election to BioMarin's board will consist of current members Bienaime, Franz Cristiani, Elaine Heron, Pierre Lapalme and Erich Sager, and proposed new members Joseph Klein and Alan Lewis. The company agreed to appoint an additional member at the stockholders' meeting that is acceptable to OrbiMed.
Shares of BioMarin (NASDAQ:BMRN) lost 17 cents Wednesday to close at $6.63.