BioMarin, the pharmaceutical company, recently announced that it will stop the development of a muscular dystrophy treatment that was undergoing in the European market. The recall came after the European Medicines Agency offered a negative opinion on the effectiveness of the cure.
BioMarin Market Tentatives
The company, which already tried and failed to enter the US market with the new treatment, has also received a negative review from the European legislators. It is thus forced to discontinue the treatments and to retract the existing medicines from the market.
BioMarin will also have to stop the projects of three other products that are currently in Phase II of the development.
The pharmaceutical company decided to join efforts with regulatory authorities, doctors, and patient groups in order to offer a transition plan for patients that are currently following the soon-to-be stopped Kyndrisa treatment.
The decision had an adverse impact on the company’s stocks, which dropped 29% in comparison with the same period of the last year.
The company is intending to continue the development of oligonucleotides that are used in the cure of Duchene Muscular Dystrophy, as it is in a continuous fight to protect its patent. Last fall, BioMarin just fought and won Sarepta on an initial ruling concerning this matter.
This new treatment was a project based on the 2014 BioMarin acquisition of Prosensa. Soon after the tests showed that the pills had little to no effects, GlaxoSmithKline refused to continue the collaboration with BioMarin. Still, the company continued the cure development by itself and tried to find a treatment solution that would delay the disease progression.
Treatments for Muscular Dystrophy
MDM is a genetic disorder involving muscle degeneration and weakness in boys. The prevalence is one in 3,500 – 5,000 cases. DMD is an effect of dystrophin absence. The symptoms appear in early childhood, and by adolescence, it gets to affect the heart and the respiratory muscles.
Despite the fact that the medicines were withdrawn from the EU market, BioMarin expects to have excellent results in prospecting different solutions for MDM in 2017.
Another pharmaceutical company that intends to obtain approval for an MDM treatment is Sarepta Therapeutics. Last week, the company announced that the Food and Drug Administration has prolonged the term of issuing an opinion on its new medicine, eteplirsen. Sarepta Therapeutics still has hopes that the approval will come soon.
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