The following are the headline results of a press release from Merck-Serono:
- Following recent feedback from FDA and previous feedback from EMA Merck Serono has decided to no longer pursue the global approval process of Cladribine Tablets
- Merck Serono will focus resources on other projects bringing benefit to patients with MS
- The benefit/risk profile of Cladribine Tablets is unchanged – Ongoing clinical trials will continue
- One-time charge of EUR 20 million to be recorded in the second quarter
“What does this mean for PwMS? In essence oral cladribine will not become available for treating MS’ers.”
“Don’t underestimate the implications of this decision. What about other MS drugs in the pipeline with undefined or even defined risks?”
“How did cladribine fail? Some initial thoughts: (1) the drug development programme was designed in the pre-natalizumab-PML era; once PML emerged potential undefined risks of new drugs became the big issue and the cladribine development programme was not sufficiently large enough to satisfy, the insatiable, appetite of regulators for safety data. (2) Cladribine has an oncology legacy; oncology drugs are simply perceived as being toxic. (3) Emergence of potentially safer competitors; notably BG12 (see previous post). (4) Patent issues; I assume cladribine will have needed to be tested in another pivotal phase 3 trial, this would delay its launch by several years, eating up valuable time on the patent clock. This would make it difficult or impossible for Merck-Serono to recoup the development costs and turn a profit on the drug for its shareholders. At the end of the day the pharma industry is a business and is shaped by the market. Unfortunately, the market rarely, if ever, prioritises unmet medical need.”
“Undefined risk; at present we simply don’t know what the long-term safety profile of cladribine is. Is this a good reason to kill a drug with so much promise? In every set-back lies an opportunity. I wonder if the NHS would be prepared to take-up the baton and run a national study to compare cladribine (the cheaper generic version) against established DMTs? Some food for thought!”
“Finally if there are any other amateur of professional hacks out their with relevant information or opinions please feel free to join the debate. It may yet become the ‘SAVE CLADRIBINE FOR THE NHS’ campaign.”
6 thoughts on “Bad news: regulatory update on cladribine”
Prof G, rumor has it the that Merck-Serono could not be sure that they could protect the patent on the tablets in the US. Which would mean the possibility of a generic oral cladribine being launched. This would destroy the very very lucrative MS DMT market, including Rebif's. Why shoot the goose that lays the golden eggs?
Fine. No worries. If the powers that be are rejecting Claderibine tablets then that’s cool. Something better will come along that works x10 better and is x50 safer. I read some other comment in another post where the person said that they will fly off to Switzerland and top them self before their “dignitity (sic) is completely eradicated by the disease”, but that’s a loser’s way of looking at things. I say deal with it. Live your life man. MS is hard and it’s gonna get harder for all of us but let’s be more hopeful. I’m a young person too and it sucks having this disease but I wanna stick around. I’m new to this blog but there’s stuff I’ve read here that makes me think good progress in treatments is going to happen soon, even better than this Clabridine drug. Even if MS kills me I wanna stick around till the end. I’m not going any sooner than I have to because I think I’ll be alright. Life’s good and it’ll get better. Prove me wrong, I dare you.
@The Awesome MS SocietyI also believe that staying positive is very important. I too am young and some of the comments I read here do make me worry about what may happen in the future. But then nobody knows what's going to happen – MS or not. If anything, having MS has made me even more determined to live life to the full and make the most of what I can do while I can still do it! I have great faith in the work of people like Prof G and do count myself lucky that while there are still improvements to be made in terms of the drugs available, I'd rather have been diagnosed now than have been someone who was diagnosed 20-25 years ago when there was nothing really available. All too often I think MS'ers can forget just how lucky we are in that respect.
Cladribine come from a class of drugs called induction therapies or immune system rebooters. Not having cladribine removes this option for MS'ers. If the use of Alemtuzumab gets limited, because of costs and/or perceived risks the induction strategy will be hard to pursue in the near future.
"I wonder if the NHS would be prepared to take-up the baton and run a national study to compare cladribine (the cheaper generic version) against established DMTs? "It sounds like a good idea – the NHS would have an effective treatment available at low cost. Is there any chance that it will happen?
Re: ""I wonder if the NHS would be prepared to take-up the baton and run a national study to compare cladribine (the cheaper generic version) against established DMTs?"I agree, but it will depend on the safety analysis of the ongoing CIS and extension study. If there is an apparent increase in secondary malignancies I can't see investigators wanting to randomize their patients to a potentially dangerous drug.