Shire Prepares To Compete With Genzyme In Gaucher Market With 15% Price Discount

During its 2009 earnings call, the company also reported significant sales increase for its Replagal drug for Fabry disease.

Shire is preparing to capitalize on Genzyme's manufacturing woes with quick, aggressive launches of two drugs that would compete directly with Genzyme's top-sellers in Gaucher disease and Fabry disease.

During its 2009 earnings presentation on Feb. 19, Shire officials said they hope to win regulatory approval this year for velaglucerase alfa for Gaucher disease in the U.S. and EU, and for Replagal for Fabry disease in the U.S. Replagal has been available in Europe for eight years and saw its market share in western Europe increase from 44 percent to 53 percent last year after Genzyme's Fabrazyme (agalsidase beta) faced supply shortages due to a manufacturing plant shutdown for viral decontamination (Also see "Genzyme Institutes Further Rationing Of Cerezyme, Fabrazyme To Build Inventory" - Pink Sheet, 17 Feb, 2010.).

Also affected by that shutdown was Genzyme's Gaucher drug Cerezyme (imiglucerase), currently the only approved therapy for the rare lysosomal storage disorder. When the June 2009 shutdown of Genzyme's Allston Landing, Mass., plant for six weeks led to shortages of both Cerezyme and Fabrazyme, FDA permitted velaglucerase and Replagal to be provided to patients under an emergency access treatment protocol.

During the investor call, Sylvie Gregoire, president of Shire's Human Genetic Therapies (HGT) division, noted that Replagal (agalsidase alfa) net sales increased 10 percent last year to $194 million, due to the increased market share in Europe, which accelerated upon the shortage of Fabrazyme. Quarterly sales increased 26 percent from the third quarter to the fourth quarter last year, she added.

Shire filed a BLA for Replagal last December and expects FDA to accept the filing within the next week, with hopes of launching the drug this year (Also see "In Brief" - Pink Sheet, 4 Jan, 2010.). More than 300 patients started Replagal therapy in 2009 - most new patients generally are treatment-naive, Gregoire said, but patient switches to Replagal soared in the fourth quarter.

Replagal gained 150 patient switches in fourth quarter

Roughly 60 percent of the drug's new 2009 patients came onboard during that quarter, with about 150 being switches from Fabrazyme, a trend that continued into January. Genzyme, which has shipped only 30 percent of demand for Fabrazyme during the shortage, announced on Feb. 17 that rationing would continue through May due to a slower-than-expected manufacturing process as well as a plan to build an inventory buffer of the drug.

"In Q4, there [was] a dramatic increase in the number of new patients on Replagal, and the vast majority of them [came] from switching from Fabrazyme," Gregoire said during the call. "There [was] also, though, a nice increase in [treatment] naive patients... physicians tend to start the new patients now on Replagal since there is the availability of that product as opposed to the competitor product."

Shire also is preparing for the possible imminent launch of velaglucerase - to be marketed under the brand name VPRIV - as its FDA filing has a Feb. 28 action date. A Marketing Authorization Application also has been filed in the EU and accepted under accelerated assessment, a process that is similar to FDA's priority review.

The company hopes to increase the pressure on Genzyme by pricing VPRIV at $1,350 for a 400-unit vial, about 15 percent less than the wholesale price for an equivalent dose of Cerezyme. In addition, Shire will provide co-payment assistance to financially eligible Gaucher patients under its OnePath initiative.

Since the FDA treatment protocol and similar programs in other countries began, about 400 Gaucher patients have received velaglucerase, Gregoire said, including about 10 percent of the U.S. patient base. In addition, the drug has claimed 5 percent to 6 percent of the eastern European market and "a significant amount" of patients in Israel, she said.

What's more, Shire has the capacity at present to treat about 200 additional patients, Gregoire said. "[We] have ample supply to support several hundred more patients this year, and they can be assured that we will not have to worry about interruptions in their treatment," she added.

Velaglucerase manufacturing moved up 18 months

In an interview prior to the earnings call, Sue Bruhn, Shire's senior VP of strategic planning and program management, explained that before the Cerezyme shortage, the company planned to submit its velaglucerase BLA in late 2009. But with the need to accelerate clinical trials and provide the drug to patients under the treatment protocol, Shire moved up its manufacturing timelines about 18 months from the prior plan.

The company did so by shifting manufacturing schedules at a Cambridge, Mass., plant where it also makes Replagal and its Hunter disease drug Elaprase . "It really just meant moving our manufacturing schedule around to focus on velaglucerase manufacturing earlier than we would have," Bruhn said. "We have plenty of supply of our other products so it was an easy switch to make."

Gregoire noted that U.S. manufacturing and clinical site inspections already have received FDA clearance, and that Shire will be adding more production capacity with a Lexington, Mass., plant expected to go online in July. The plant will offer four 2,000 liter bioreactors and an alternate site for making VPRIV or the other HGT products.

Overall, Shire announced that it earned $2.69 billion in net product sales in 2009, a 2 percent decline from 2008. However, when the impact of generic competition to top-seller Adderall XR is factored out, the company's other products brought in $2.07 billion, a 25 percent increase over 2008. Adderall XR, which has faced generic competition from Teva and Impax since last April, netted $627 million in sales last year, a 43 percent decline from 2008.

-Joseph Haas ([email protected])