Multiple sclerosis drug alemtuzumab has continued to show strong efficacy with no new or worsening side effects, according to data from a mid-stage trial, presented at the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) on Thursday.
Eighty seven percent of patients treated with alemtuzumab (also known as Campath), experienced no worsening of symptoms of disability after five years.
By comparison, only 62 percent of patients taking Rebif, a rival drug made by EMD Serono, an affiliate of Germany's Merck KGaA and Pfizer Inc, experienced no worsening of disability.
For the Genzyme Corp, the manufacturers of alemtuzumab, the trial results are a shot in the arm.
Most analysts and investors seem to agree that the drug is highly efficacious. The question is whether it is sufficiently safe to win approval and achieve widespread commercial success.
"The most remarkable thing about this data is that it hasn't changed much from the four-year data," said Michael Panzara, who oversees clinical development of Genzyme's multiple sclerosis and immune system drugs.
Initial three-year results, reported in 2008, showed that six patients developed immune thrombocytopenic purpura (ITP), an autoimmune disease in which low blood platelet levels can lead to bruising and bleeding. One patient in the trial died of a cerebral hemorrhage. The remainder were diagnosed more quickly and successfully treated.
Encouragingly, no new cases of the condition were reported in the latest data.
Multiple sclerosis is an autoimmune disease in which white blood cells, which are supposed to fight infection or disease, misguidedly attack the body's own cells, causing damage to the myelin sheath that protects nerves in the brain and spinal cord.
The disease, which some groups estimate affects between 350,000 and 400,000 patients in the United States, can cause a variety of symptoms including impaired mobility, fatigue, cognitive, speech and other difficulties.
Genzyme Corp believes alemtuzumab, if approved, could become the new standard of care for treating the disease and gain a significant portion of a market the company estimates will be worth roughly $13 billion by the end of this year.
Earlier in June U.S. Food and Drug Administration (FDA) had granted the drug Fast Track status. Consequently alemtuzumab for MS became eligible for Priority Review, and the FDA may consider for review portions of the marketing application before the submission of a New Drug Application (NDA) is completed.
"We are extremely pleased that our alemtuzumab development program has been assigned Fast Track status, and look forward to working closely with the FDA to expedite the program's review process," said Henri Termeer, Genzyme's chairman and chief executive officer. "Alemtuzumab is a potentially transformative therapy for the treatment of multiple sclerosis, and an important part of our future."
Alemtuzumab for the treatment of MS is currently being evaluated in two pivotal multi-center, multi-national trials, known as CARE-MS SM (Comparison of Alemtuzumab and RebifŪ Efficacy in Multiple Sclerosis). The company's CARE-MS I Phase 3 trial is a randomized study comparing alemtuzumab to the approved therapy Rebif (high-dose interferon beta-1a) in early, relapsing-remitting multiple sclerosis (RRMS) patients who have received no prior therapy. The second Phase 3 trial, CARE-MS II, is comparing alemtuzumab to Rebif in RRMS patients who had active disease while on other MS therapies.
Both trials are fully enrolled and data are expected to be available in 2011.