The key to Genzyme Corp.’s move to withdraw its leukemia drug, Campath, in anticipation of a multiple sclerosis indication seems to be working with the cancer community and establishing generous patient support.
Genzyme, the rare disease unit of Sanofi, is in the process of removing the Campath brand of alemtuzumab from commercial availability in about 50 markets, including the United States and the European Union – and the company has been transparent about its reasoning. It does not want Campath to be used off label in the multiple sclerosis setting when a lower-dose formulation of alemtuzumab obtains approval for multiple sclerosis.
But instead of courting controversy with the potentially contentious business decision, the firm has decided to make Campath available free of charge under a patient-access program. The leukemia treatment had reportedly cost in the range of $50,000-$60,000.
In an Aug. 9 letter to health care providers, Genzyme makes clear that it will stop selling Campath, not "for any reasons related to product safety, efficacy or supply, but as part of the company’s plan for bringing alemtuzumab forward as a treatment for a new indication." The company added that it "will continue manufacturing sufficient quantities of alemtuzumab to supply this patient access program."
Although sacrificing one patient community (and cancer, at that) in favor of a more lucrative one certainly seems as if it could provoke controversy, the steps Genzyme took appear to be enough to ease the company past public outcry.
Hildy Dillon, senior vice president of patient services for the Leukemia and Lymphoma Society, said that her organization was concerned when it first learned of Sanofi/Genzyme’s plans to stop selling Campath, but added that the companies had done a strong job of laying the groundwork for a transition from commercial product to one available for free under a compassionate-use protocol.
"There is an extensive patient-distribution system which they have shared with us," she said in an interview. "They’ve actually been quite supportive to us and our constituents by telling us what the distribution is and how they have communicated their plans to health care providers who treat our patients – hematology oncologists both in the community and in the major cancer centers.
"[Those doctors] are now aware of how they can access the drug since they no longer will be purchasing it, but they can contact the company directly to get whatever supply they need."
Campath Available to New Patients
Ms. Dillon said the Leukemia and Lymphoma Society was assured that Campath will be available for all patients who need it, including those who may be prescribed it for the first time after it is no longer available commercially.
"Genzyme was in touch with advocacy groups like ours to make sure we were aware of the change and how physicians will be accessing the drug," she said. "They’ve done a lot in their due diligence to inform both the physician community and the patients. ... It’s our understanding that there won’t be any shortage of supply and that the patients who need Campath will be able to get it."
By making it harder for alemtuzumab to be used in the MS setting off label, Genzyme also increases its chances of making the product (to be marketed as Lemtrada in the MS indication) much more lucrative. Campath, an anti-CD52 monoclonal antibody indicated for the treatment of chronic lymphocytic leukemia (CLL), yielded net sales of $76 million last year for Sanofi. The French pharmaceutical company bought out Genzyme for about $19.4 billion in early 2011, largely on the strength of the biotech company’s success in developing large-molecule products for rare disorders.
Industry analysts predict much bigger sales for Lemtrada, which will be provided in a 12-mg dose per treatment, compared with 30 mg for Campath. A supplemental Biologics License Application was filed at the Food and Drug Administration this past June, with a marketing authorization application sent to the European Medicines Agency in the same time frame, which means that regulatory decisions on Lemtrada are anticipated during the second quarter of 2013. Analysts widely predict initial-review approvals by the FDA and the EMA, a launch in late 2013, and the ramp-up to sales ranging between $336 million and $400 million in 2016.
No Similarity to Genentech AMD Dilemma, Genzyme Says
Sanofi/Genzyme’s dilemma regarding what to do with a useful but modestly selling drug that offers the potential to earn more in a second indication mirrors in some ways the challenge faced by Roche/Genentech Inc. with its vascular endothelial growth factor therapies Lucentis (ranibizumab) and Avastin (becavizumab). Both demonstrated potential in clinical trials for the treatment of wet age-related macular degeneration, but Roche never filed Avastin for AMD because it would undercut the Lucentis market in that indication. Lucentis costs about $2,000 per injection, while oncology drug Avastin is available in small-dose syringes for between $35 and $50 per unit.