UCLP Bosses speak out against the actions of Genzyme

Thompson AJ, Giovannoni G. Removal of access to alemtuzumab for patients with aggressive multiple sclerosis. BMJ. 2013 Jan 18;346:f275. doi: 10.1136/bmj.f275.

Team G speak out. 


Although some treatments are now available for multiple sclerosis, better treatments are needed for people with aggressive disease. The need to find agents that can prevent or slow disease progression is particularly challenging and requires a concerted global effort that combines academic and pharmaceutical expertise. Although such partnerships are rare, the story of alemtuzumab has shown, inspirationally, that academic and pharmaceutical prowess can be combined to deliver an agent with real promise. Alemtuzumab, which has been used successfully off label to treat active relapsing-remitting multiple sclerosis, is a humanised monoclonal antibody that targets the CD52 antigen on leucocytes. Not surprisingly, there was therefore an outcry when Genzyme, now a Sanofi company, surrendered the licence for all currently licensed preparations of alemtuzumab, with effect from 8 August 2012, meaning that it will no longer be available as a licensed product in the United Kingdom once existing supplies run out.


This withdrawal was not related to product safety, efficacy, or supply, but was part of Genzyme’s plans for bringing alemtuzumab forward as a treatment for multiple sclerosis. It was done in agreement with the Medicines and Healthcare Products Regulatory Agency (MHRA). When stocks run out, alemtuzumab will still be available through a named patient access programme for its cancer and transplant indications. But what does this mean for people with multiple sclerosis in the UK?

It means that off-label use of alemtuzumab in patients with multiple sclerosis has come to an abrupt end. Patients and clinicians will have to wait for the new formulation (Lemtrada) to be licensed by the European Medicines Agency and be approved by the National Institute for Health and Clinical Excellence before it can be accessed on the NHS. (Years away)

The removal of access to off-label use of alemtuzumab is a complex matter. It will not surprise anyone if the new formulation of alemtuzumab that is specifically for multiple sclerosis (Lemtrada) costs more than the old formulation (Mabcampath) when launched. A cynical perspective is that the old formulation was withdrawn simply to allow the manufacturer to increase the price of the new formulation. Of course the argument is that Genzyme needs to recoup the costs of running the multiple sclerosis clinical development programme for Lemtrada and make money for its shareholders. If we want the drug industry to repurpose existing drugs for new indications it needs an incentive to do so. However, until we know what Lemtrada will actually cost any further debate on this matter is moot. (Bet you it will cost alot if word on the street is correct)

Mabcampath is not entirely safe. Serious, albeit treatable, adverse events have occurred in people with multiple sclerosis who have received alemtuzumab.These have included several life threatening autoantibody mediated autoimmune diseases, the most serious of which are immune mediated thrombocytopenia and Goodpasture’s syndrome. If licensed for the treatment of multiple sclerosis, alemtuzumab will almost certainly have an intensive and rigorous post-marketing surveillance programme. Patients treated with alemtuzumab for multiple sclerosis will probably need monthly blood and urine tests performed for at least five years after treatment to monitor for these complications. It would be difficult for Genzyme to implement this programme if an unlicensed formulation of alemtuzumab (Mabcampath) was simultaneously being used.

What about the ethical problem of patients who have already been treated with alemtuzumab who will no longer have access to it? This is important because alemtuzumab is an induction therapy—it is given as a course of five daily infusions at the beginning of year one and three daily infusions at the beginning of year two.(Ironically, patients with multiple sclerosis will be offered a third, or subsequent, course of the drug only if their disease is active—if they have a relapse or have evidence of disease activity on magnetic resonance imaging, which is counter intuitive.) Genzyme and the relevant healthcare providers must come to an interim solution that allows patients with multiple sclerosis who have had a course of alemtuzumab infusions but need additional courses to receive the drug on a named patient basis before it is brought to market for the new indication. The onus will then be on the treating clinicians to make sure that patients are monitored appropriately for any delayed complications of alemtuzumab treatment.

The withdrawal of alemtuzumab has generated a backlash from the multiple sclerosis community. However, we would encourage a broader debate on the questions that this and other related cases raise about the role of the drug industry in drug development—particularly in the repurposing of existing drugs—including relations with academia and the industry’s role in the wider economy. On a positive note, the alemtuzumab multiple sclerosis development programme is an excellent example of how academia and the drug industry can work together to find new treatments, with benefits on both sides. What started as an academic initiative in the early 1990s was successfully transferred to industry and has resulted in a promising treatment for people with multiple sclerosis. Perhaps future models might be based on partnership throughout the entire drug delivery pipeline.


I think this is a sorry state of affairs, but companies want to make money that is clear......however it is right for BigAl and ProfG to raise questions. Will Teva make a citizens petition to the FDA against it or maybe Novartis and Biogen will make a case as it will compete with their product?

This is why we need the BPA (see above), that can even move into the Big Pharma space. 

Maybe the As from Cambridge are a bit embarrassed about this whole process of profiterring, because if seems that at least one of the As is keeping an unusually low profile, with regard publishing the effects of alemtuzumab or is that Lemtrada-which is less alemtuzumab at ten times the price?

Also they are lucky it is a biological because had it been a chemical, the patent life would be gone by now and the me-toos would be on the table?

CoI: Written by Team G

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