The FDA rejects drug for Duchenne’s Muscular Dystrophy (DMD). Eteplirsen is the name of the drug developed by Serapta which has been rejected by the FDA at only a week after kyndrisa, a similar drug developed by BioMarin has also been rejected.
The neurology division of the FDA has reviewed the eteplirsen and concluded that the amount of dystrophin it produces is insignificant to actually improve the condition of the patients. Dystrophin is the critical protein missing from the muscles of boys suffering of DMD.
DMD is a disease affecting boys, who become dependent of wheelchairs before the teenage years and killing them on average at the age of 25. The disease is affecting over 15,000 boys in the United States and until now there is no medicine which can at least slow its progression.
The hundreds of thousands of patients were waiting for Sarepta and BioMarin to release their drugs on the market, hoping they will be provided with the necessary dystrophin lacking from their bodies.
Next Friday an advisory committee formed from outside experts will hear the FDA’s report along with testimonies from Serepta and DMD experts and patients. After hearing all the parts the panel will issue their recommendations about eteplirsen to the FDA that might change its decision and finally approve the drug.
In most of the cases the FDA accepts recommendations made by the advisory panel but it is not obligated to. The agency will decide whether or not they will grant accelerated approval to the new drug by the end of February.
Pharmaceutical companies can request accelerated approval for certain drugs developed to respond to serious unmet needs. This procedure implies skipping the extensive clinical trials usually requested by the FDA.
The FDA declared that even if they are ready to show flexibility with respect to this devastating illness without any treatment available it cannot give approval for a drug without substantial evidence of effectiveness.
Calculations show that the effectiveness of the drug has been calculated to an average of only 0.9 of dystrophin increase after 180 weeks of treatment. It is not yet known the level required for clinical significance but experts say it should be a lot more than 1 percent.
However, even if the drug won’t be approved this year it still has chances to get approval in 2017 as Sarepta has already begun the third phase of the drug’s study which is expected to produce results by the end of 2017.
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