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There are many promising trials in the pipeline that may bring big news for major therapeutic areas.
Clinical trials are essential for establishing the efficacy of new drugs, observing patients’ reactions to treatments, and representing populations of people that may not always be discussed in everyday conversation. Although there are many diseases and disorders that are waiting for treatments, there are many promising trials in the pipeline that may bring major news for these conditions sooner than we think, even if there are some minor setbacks.
In the gene therapy space, Sarepta Therapeutics is looking to get an approval for Elevidys (delandistrogene moxeparvovec-rokl) through the EMBARK trial. This drug may be a breakthrough for patients with Duchenne muscular dystrophy who have been waiting for a treatment for many years. After receiving an accelerated approval for the drug for patients between 4 and 5 years of age, it has been the company’s mission to expand from the narrow population and address the doubts surrounding Elevidys. Although the trial is ongoing with 126 patients in a controlled environment, the results will either lead to regulators growing the age restrictions or having to remove the drug from the market (1).
We have seen very quick removals from the drug market before. An example of this is FDA’s decision to pause new patients on Merck KGaA’s multiple sclerosis drug, evobrutinib. Evobrutinib falls within the Bruton’s tyrosine kinase (BTK) inhibitors class, aimed at slowing the progression of nerve disease multiple sclerosis, but as of April 2023, a partial clinical hold on the Phase III trials was issued by the FDA due to “laboratory results which suggested drug-induced liver injury during Phase III studies.” Although a Phase III trial for evobrutinib is set to continue as planned, Merck has had to alter their study that involves patients who have been taking the drug for fewer than 70 days (2).
The growing number of clinical trials is very exciting, and I am looking forward to continuing to hear about the latest treatments being tested in the mental health space, cancer space, and any other therapeutic area that takes up the pharmaceutical industry. Even with the setbacks we hear from some of the trials, I am positive that we will get the best results, and help for so many patient populations will finally be achieved.
Jill Murphy is Editor at BioPharm International.
Vol. 36, No. 9
When referring to this article, please cite it as Murphy, J. Clinical Trials and their Impact on the Pharma Industry. BioPharm International. 2023 36 (9) 6.