Genzyme General and BioMarin Pharmaceutical Inc., through their joint venture, filed the first portion of a rolling biologics license application for Aldurazyme, and now have started the approval process in the U.S. as well as overseas.
In March, a marketing authorization application was submitted to the European Agency for the Evaluation of Medicinal Products to sell the drug in the European Union.
“It’s a great accomplishment,” said Dan Quinn, manager of public relations at Cambridge, Mass.-based Genzyme. “[Beginning the rolling BLA] coincides with the EU submission we announced a couple of weeks ago. It’s just one step closer to bringing this drug to market, which is important to the patients who are waiting for it.”
Aldurazyme (laronidase) is an enzyme replacement therapy designed to treat patients with mucopolysaccharidosis I (MPS I). The disease is caused by a deficiency of the enzyme alpha-L-iduronidase that leads to an accumulation of complex carbohydrates in the lysosomes of cells. Symptoms of the disease can include impaired cardiac and pulmonary function, delayed physical development, skeletal and joint deformities, and reduced endurance.
Quinn said the companies anticipate having the BLA filing complete “sometime in the third quarter.” At this point, the information filed on both sides of the Atlantic comes from the Phase III trial that had positive preliminary data late last year. (See BioWorld Today, Nov. 5, 2001.)
“Thus far, [the filing includes] essentially the data from November, which was the first six months of the trial,” Quinn told BioWorld Today.
All patients who received the drug in the Phase III study elected to continue taking it in the extension study that is ongoing. The companies will file data from that trial as they become available. In the meantime, Novato, Calif.-based BioMarin and Genzyme are planning two other clinical trials with Aldurazyme one will enroll children younger than 5 years old, while the other will enroll patients with more advanced clinical manifestations of MPS I. Those trials should help bring the disease into focus, Quinn said.
“[The two trials] are to add to our body of knowledge about the efficacy of the drug,” he said. “It’s a very heterogeneous disease; we want to have as complete a picture of it as we can.”
SG Cowen Securities Corp. analysts wrote in a research note that “the companies anticipate to receive an FDA response in [the first half of 2003],” and those at Cowen “envision that the companies will submit six-month extension data to the EMEA as it becomes available and will receive a reply in [first half of 2003], in line with a 12-month review timeline.”
The joint venture between BioMarin and Genzyme was formed in 1998 through a deal valued at up to $30 million for BioMarin. BioMarin will have manufacturing responsibility for Aldurazyme and Genzyme will commercialize the product if and when it is approved. “We took the lead in the EU [filing] and [BioMarin] took the lead for the FDA,” Quinn said, noting the joint venture has gone smoothly. (See BioWorld Today, Sept. 16, 1998.)
“We had to work very closely on the filings,” he said. “It’s been great. This has been a very collaborative thing and it’s a good milestone for both companies.”
Genzyme’s stock (NASDAQ:GENZ) rose $1.22 Monday to close at $43.32. BioMarin’s stock (NASDAQ:BMRN) fell 61 cents to end the day at $7.39.