Sarepta Therapeutics Inc. (SRPT) made public its partnership with the NASDAQ-listed Catabasis Pharmaceuticals Inc. (CATB) to find a cure for a rare illness called Duchenne muscular dystrophy (DMD). The drug implemented by these two companies is Exondys 51 (eteplirsen). Using the partnership, Sarepta considers implementing Exondys 51 combined with an anti-inflammatory drug called CAT-1004 (edasalonexent). They want to analyze whether this combination of medication will combat the muscular damages provoked by the rare illness.
The CAT-1004 drug is assumed to impair the degradation process of the muscle. The testing of this new drug will be conducted on mice. Catabasis protects the orphan drug and the identification of rare pediatric illnesses from the FDA in order to use CAT-1004 for DMD’s treatment.
This partnership represents the first time two big pharmaceutical companies manage to test a mixture of investigational treatments to cure Duchenne muscular dystrophy. The financial report that monitors the costs was not made public.
Sarepta stated that their company is ready to investigate new cures for other rare illnesses, developing their drugs. This new plan may bring a benefit to Exondys 51 which could be used to treat similar DNA mutations.
This drug is effective in creating a dystrophin protein, which helps in confronting the Duchenne disease. Sarepta is regarding the possibility of using other medicines that show the same ability to produce similar proteins. For example, those drugs that produce utrophin protein are as useful as the dystrophin protein created by Exondys 51.
This drug manufacturing deal may seem odd for other specialists, and even dangerous. In 1962, officials had enhanced the process for authorization of new medicines. The new studies that wanted an approval not only needed to pass the law which involved evidence of safety but also required a substantial proof of the drug’s effectiveness. Nevertheless, the trials conducted to show the effectiveness of dystrophin protein revealed no significant progress in subjects’ performance. The question is how this new drug got its approval from FDA.
Moreover, FDA specialists discovered severe problems in the effectiveness of the tests meant to show a change in the level of dystrophin. Despite all these facts, why did Dr. Janet Woodcook, the director of FDA’s Center for Drug Evaluation and Research, give her approval? Don’t you think this is a violation of the regulations?
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