The Burrill Report


Sanofi’s Genzyme unit said the U.S. Food and Drug Administration would not approve its supplemental application to market Lemtrada, alemtuzumab, for the treatment of relapsing forms of multiple sclerosis. According to Genzyme, the FDA has told the company that based on the late-stage study evidence submitted the company had not produced adequate evidence to demonstrate the benefits of Lemtrada outweigh its serious adverse effects. The agency told Genzyme that one or more additional active comparator clinical trials of different design and execution would be needed prior to the drug’s approval for the indication. Genzyme said it strongly disagreed with the FDA’s conclusions and planned to appeal the agency’s decision. “We strongly believe that the clinical development program, which was designed to demonstrate how Lemtrada compares against an active comparator as opposed to placebo, provides robust evidence of efficacy and a favorable benefit-risk profile, said David Meeker, Genzyme president and CEO. “This evidence was also the basis for the approvals of Lemtrada by other regulatory agencies around the world.” Sanofi said it did not expect the CVR milestone of U.S. approval of Lemtrada by March 31, 2014 to be met.

Israeli biotech Can-Fite BioPharma subsidiary OphthaliX’s investigational compound for dry-eye syndrome, DF101, failed to meet its primary or secondary endpoints in a late-stage study. The 24 week, placebo-controlled involving 237 patients with moderate-to-severe dry eye syndrome who were treated with its licensed drug CF101, an A3 adenosine receptor agonist. However, it was found to be well tolerated. CF101 is being developed by Can-Fite for anti-inflammatory indications and the company has reported positive data from a mid-stage clinical trial in rheumatoid arthritis. In addition, Can-Fite is conducting a phase II/III clinical study in patients with Psoriasis. OphthaliX is also developing CF101 for the treatment of glaucoma and uveitis, with interim data from an ongoing mid-stage study as a treatment for glaucoma expected to be released during 2014.

Swedish biotech Axelar said final results from a mid-stage study in patients with non-small cell lung cancer showed no statistically significant difference in rate of progression-free survival between the patients treated with AXL1717 compared with the group treated with docetaxel, confirming previously communicated preliminary data. Despite these results, the company, which is backed by Karolinska Development, plans to investigate AXL1717’s potential to be developed for patients that have relapsed after treatment with docetaxel. “There is increasing evidence that AXL1717, in addition to the IGF-1R pathway inhibition, also suppresses tumor cell division by arresting cells in mitosis through a non-IGF-1R-dependent mechanism,” said CEO Mikael von Euler. “This proposed additional mechanism of action would explain the differences in efficacy and side effect profile compared with other substances inhibiting the IGF-1R pathway.” The new data will be offered at scientific meetings in 2014.

At the start of the new year, Germany’s Institute for Quality and Efficiency in Healthcare, or IQWiG, told three pharmaceutical companies that their drugs offered no additional benefit to therapies currently in use. It said GlaxoSmithKline’s Tafinlar provided no additional benefit versus dacarbazine to treat unresectable or metastatic melanoma in patients with a BRAF V600 mutation. The opinion was based on a late-stage trial of the oral BRAF protein kinase inhibitor compared to dacarbazine that had not yet reached a stage for a final analysis of overall survival data. IQWiG told Sanofi that Aubagio had no additional benefit versus interferon beta-1a to treat relapsing-remitting multiple sclerosis, but side effects of the two drugs were different. IQWiG also found no additional benefit for Bayer’s Eylea compared to Lucentis to treat visual impairment caused by macular edema secondary to central retinal vein occlusion. The agency said that neither drug was used according to its European label in the data submitted by Bayer. Comments for all three drugs are due by January 23 and a final assessment from Germany’s Federal Joint Committee is expected in mid-March.

January 02, 2014
http://www.burrillreport.com/article-fda_rebuffs_genzyme%e2%80%99s_lemtrada_for_relapsing_ms.html