1. A Phase II clinical trial involves a larger group of people ( 20- 300) to know how well the drug works and also to continue to test the safety levels of the drug (already done in Phase I trials).
2. Oral doses of FTY720 given as once a day pills of 5mg and 1.25 mg to 2 groups ( 281 patients per group) of relapsing remitting MS patients. The treatment was carried out for 3 years. This study was done in Canada and 10 European countries.
3. This trial gave positive results.
4. After 2 years of study 77% (5mg) and 75% (1.25mg) patients showed reduced rates of annual relapses of MS. The first 6 months trial showed a decrease of 50% in the treated patients compared to 30-35% decrease by the present therapies (first line treatments).
5. "On the basis of comparable efficacy and better safety profile" all the patients were shifted to 1.25mg dose of FTY720.
6. Three year data showed that 89% of the patients got free of active brain lesions which are seen in untreated MS patients as tested by MRI scans.
7. Side Effects - FTY720 was well tolerated in patients in 3 years. However, it showed the following side effects in 16 % of treated patients: Nasopharyngitis (inflammation of nasal passage and upper part of pharynx), fatigue, headache, influenza increase in liver enzyme levels ( alanine aminotransferase). Also, a small number of patients showed localized skin malignancies.
More on ALT- An ALT (alanine aminotransferase test) is done with blood samples to determine the liver damage. Therefore increased levels of ALT indicate some damage to liver.
So what is not clear from the Novartis report is which dose of FTY720 is causing the liver damage and to what extent? may be this is the reason for the shift from 5mg to 1.25 mg dose of FTY720.
8. Novartis is continuing with two very large Phase III clinical trials. They will also do regulatory filings by end 2009. However that does not mean that approval for sale is ensured.
Personal view- there is no harm in keeping a track of this drug for another year. It sounds promising. However, till FDA approval is obtained, nothing can be decided about it. It is very important that the dose and safety levels of the drug is thoroughly established. Immunomodulators and immunosuppressant drugs show very strong positive results very quickly but fail at safety levels.
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