Carmeseal-MD Nick Catlin Thursday, 11 December 2014 No Comment

An early access programme was announced for Carmeseal-MD™ (Poloxamer 188, NF) recently ( with the headline:

"Phrixus Pharmaceuticals announces European Access Program for CarmesealMD™ (P-188 NF) for patients with Duchenne muscular dystrophy"

Treat NMD and UPPMD have published this very good guideline for families

It is interesting to note here how it is possible to gain early access to medicines within current legislation for a drug that has not been tested in clinical trials for Duchenne. Again an excellent review of these procedures by Treat NMD . It would appear that in this case access to Carmeseal would be on a "Special" "named patient" basis where the doctor would contact the company directly and take responsibility for administering a drug. It was not very clear in their press release if Phrixus have clinicians already in mind or if they expect patients to consult their own MD's.

The second issue is that there appears to be evidence from the Treat NMD review that Carmeseal-MD has side effects that might lead to muscle weakness. Phrixus also report that they are planning clinical trials in Duchenne patients.

It is worth keeping in mind that if a company chooses to offer their drug for use on the basis that it might do some good then individual clinicians can administer it to patients even without it having been tested in specific DMD clinical trials. In my own view the test should always be to consider if there is enough evidence to show that the benefits of taking a drug are more than likely to outweigh risks or side effects in the long term. This evidence might possibly come from animal studies, usage in other patient groups, clinical practice etc; but we know that clinical trials conducted in Duchenne patients are the best way to collect data to determine the benefit/risk ratio and establish the efficacy of medicines.

So it might be the case here that patients can find clinicians that Phrixus are willing to offer Carmeseal to for administration for Duchenne. If that is the case for you then look carefully at the research data highlighted by Treat NMD and ALWAYS first consult your own neuromuscular expert team before agreeing to take any new medication.

79 is hosted by Nick Catlin

Nick Catlin has a son Saul who has Duchenne Muscular Dystropy. He is a founding member and previous CEO of the Charity Action Duchenne. Nick now works with young people with Duchenne and their families at Decipha assessing for Special Education Needs and advising on reviewing Education Health and Care Plans.

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