Medical Device Daily West Coast Editor

With approval pending in the U.S. — and soon in Europe — for Synvisc-One to relieve osteoarthritis knee pain, Genzyme (Cambridge, Massachusetts) hardly was hurt by last week's news that its pivotal study with hylastan for the same indication failed to beat steroids.

"We had two products [under development] in the same time frame and same area," said Bo Piela, spokesman for Genzyme. "This one was relatively low profile."

The pivotal trial of hylastan, an injected joint lubricator, enrolled almost 400 patients at 27 sites in North America and Europe, and randomized patients to get two intra-articular injections of hylastan administered two weeks apart, or a single injection of hylastan, or an injection of a corticosteroid. Patients were evaluated for 26 weeks after therapy.

Hylastan "did reduce pain, but so did steroids," Piela said. Results showed a statistically significant and clinically meaningful reduction in knee pain compared to baseline for each of the three treatment arms, but the difference in pain reduction between patients treated with hylastan and patients treated with a steroid was not significant, so the trial missed its primary endpoint of superiority.

"We need to determine whether there's a place for [hylastan] longer term, what would be required," Piela said. "But the reality is, we're focused on Synvisc-One," a combined-dose regimen of already marketed Synvisc (hylan G-F 20) provided in a single injection. Synvisc sold $234 million for Genzyme in 2006, and is expected to range between $250 million and $265 million this year.

Meanwhile, Genzyme's bread and butter remains in its lysosomal storage disorder franchise and in Renagel (sevelamer hydrochloride), the phosphate binder for kidney failure. Renagel sold $515 million in 2006, a number that Genzyme expects will rise to a range of between $580 million and $590 million this year.

Cerezyme (imiglucerase for injection), Genzyme's standard-of-care therapy for Type 1 Gaucher disease, sold $1 billion last year, and is expected to reap as much as $1.075 billion this year. Fabrazyme (agalsidase beta) for Fabry disease sold $359 million in 2006, and could go as high as $425 million in 2007.

In the pipeline are Renvela (sevelamer carbonate) for end-stage renal disease and chronic kidney disease, and tolevamer for C. difficile colitis.

Last month, Genzyme reported encouraging data from two studies comparing powder Renvela with Renagel tablets.

The move to buy Bioenvision (New York) goes on, despite a snag.

Last week, Genzyme said only 26% of partner Bioenvision shares had been tendered, and Genzyme already had agreements to control 24%.

On May 29, Genzyme agreed to acquire Bioenvision in an all-cash transaction valued at $5.60 an outstanding common share and $11.20 an outstanding preferred share (plus accrued but unpaid dividends), or about $345 million. Now, Genzyme extended the deadline until July 10, and still plans to buy as many shares as are tendered.

At the center of the deal is clofarabine, a nucleoside analogue. Bioenvision markets the drug as Evoltra in Europe for pediatric relapsed or refractory pediatric acute lymphoblastic leukemia.

Genzyme got North American rights through the $1 billion buyout of ILEX Oncology (San Antonio), which had licensed the rights from Bioenvision, and Genzyme markets the drug as Clolar in the U.S. and Canada, but does not separate the Clolar sales from the rest of the oncology lineup, which netted $22.4 million in the first quarter. Genzyme's plan is to expand clofarabine's label into adult acute myeloid leukemia, where the firm estimated peak sales could hit $600 million, mostly from overseas sales.

Piela had no additional comment on the progress of the Bioenvision deal.