An early access programme was announced for Carmeseal-MD™ (Poloxamer 188, NF) recently (http://www.phrixuspharmaceuticals.com/news.php). This early access programme is intended to make Carmeseal-MD™ available to patients with Duchenne muscular dystrophy with respiratory and cardiac deficits through their specialty physicians as an unlicensed medicinal product (a so-called ‘Special’). The following information is provided to help people understand a bit more about this drug and what such a programme means.
This type of access is also called ‘named patient’. Different forms of early access to medicines in development were explained recently on the TREAT-NMD website. When early access is sought to drugs being tested in clinical trials for the disease in question, this can be done through compassionate use if approved through a national system, where regulations apply on e.g. the data to be collected on side effects and efficiency. By contrast, for a ‘named patient’ programme a clinician directly requests a supply of a medicine from the manufacturer. This medicine does not have to be under evaluation in clinical trials and there are no regulations in place on the collection of data for patients who are treated, since treatment is the responsibility of the clinician. There is no set mechanism for payment; it can be through health insurance systems, the company or patients themselves. It is not yet known what mechanism will be used for Carmeseal-MD™.
Carmeseal-MD™ has not yet been tested in clinical trials for Duchenne. It has been tested in animal models for Duchenne muscular dystrophy (mice and dogs), where systemic treatment resulted in improved heart and diaphragm function (e.g. http://www.ncbi.nlm.nih.gov/pubmed/21575230, http://www.ncbi.nlm.nih.gov/pubmed/20234088), but a recent paper showed treatment resulted in loss of skeletal muscle strength (http://www.ncbi.nlm.nih.gov/pubmed/24642557).
One of the reported side effects of Carmeseal-MD™ treatment is muscle weakness https://www.spectrumchemical.com/MSDS/P1169ANSI-P1169AGHS.pdf).
Carmeseal-MD™ has been reviewed by the TREAT-NMD Advisory Committee for Therapeutics (TACT) in 2010. In the most recent update by Phrixus on the DuchenneConnect website (https://www.duchenneconnect.org/en/clinical-trials/study-faq-sheets/650-carmeseal-md-poloxamer-188-nf.html, posted may 2014), clinical trials for Carmeseal-MD™ were being planned.