After spending $680 million and working on a drug to treat Duchenne muscular dystrophy for two years, BioMarin Pharmaceutical has decided to cut its losses and stop development. Drisapersen was intended to treat Duchenne syndrome, a muscular dystrophy condition that causes muscles to quickly degenerate. Currently, there is no effective treatment for Duchenne, so most patients diagnosed with it gradually lose their ability to move and die by the time they reach their mid twenties. Despite their efforts to develop an effective way of halting the fatal progression of Duchenne, BioMarin has been unable to get drug approval from either United States or European markets.
The Controversial History of Drisapersen
Drisapersen was designed to treat muscular dystrophy by patching the mutated genes that cause muscular degeneration. In 2014 BioMarin took a huge gamble and paid $680 million for drisapersen. It was bought during a buyout for Prosensa, a pharmaceutical company from Netherlands that lost its value after after drisapersen failed phase 3 clinical trials. Though the drug had previously failed trials, BioMarin was hoping to be able to rework the drug and somehow gain approval from the FDA.
BioMarin Faces FDA
BioMarin used Prosensa’s data and their own resources to develop the Duchenne drug further. When they bought the drug, they were confident that it would be effective for a unique subset of patients with Duchenne, even though it did not seem to work when applied to all categories of people with Duchenne. BioMarin CEO, Jean-Jacques Bienaime claimed that the Duchenne drug would most likely be approved, simply because there is an unmet need for any sort of Duchenne treatment, even one that does not cure the condition entirely. However, when this reasoning was explained to the United States Food and Drug Administration, it did not succeed. Ultimately, in January of 2016, the FDA rejected requests for approval. The main reason behind this rejection is that large doses of drisapersen are toxic, and the FDA believes that doses small enough to avoid toxicity do not provide any sort of treatment.
BioMarin Finally Gives Up on Drisapersen
BioMarin continued to try for drisapersen approval in Europe, citing studies that showed very low doses of the medicine might slow the disease progression if started while patients were still children. However, on May 31, 2016, this attempt for approval was shot down during a meeting with the European Committee for Medicinal Products for Human Use. Though the meeting did not result in any sort of official declaration, the committee clearly stated that they would continue to regard drisapersen with disapproval. After discovering that European approval most likely would not happen, BioMarin decided to cut its losses and stop trying to develop the Duchenne drug.
The Future of Duchenne Treatments
After realizing that it would most likely be unfeasible to continue researching drisapersen and seeking approval in various countries, BioMarin is moving on to other potential Duchenne treatments. BioMarin claims that stopping development was a “difficult but necessary decision.” They are now pinning their hopes on treatments that alter RNA instead of DNA in an attempt to halt the miscoded genes from destroying muscular tissue. Patients who were in clinical trials using drisapersen will be transitioned from the drugs to other treatments. Currently, the only other Duchenne treatment that seems to be more favorably viewed by the FDA is eteplirsen, created by Sarepta Therapeutics. The need for an effective Duchenne treatment is so great. If Sarepta beats BioMarin in getting a drug approved, BioMarin may face significant financial difficulties.
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