BioMarin Will Stop Developing Lead Drug for the treatment of Duchenne Muscular Dystrophy

BioMarin Pharmaceutical plans to stop the development of Kyndrisa a drug which targets patients suffering from Duchenne Muscular Dystrophy. This decision came out because the European regulators threaten BioMarin that they will give a negative review of their lead drug.   This is a double-blow for the company after the US regulators rejected their drug four months ago.

Jean-Jacques Bienaime, Chief Executive Officer of BioMarin, said in a Press Statement, that the ominous denial of European Union, has forced the company to stop the development of Kyndrisa and three other related medications. Bienaime shared that it was a difficult decision for the company but it is necessary to do so.

As of now, there is no approved drug treatment yet for Duchenne Muscular Dystrophy or also known as DMD.  Duchenne is a deadly genetic disease that affects young boys. For years, biopharmaceutical companies have tried finding a cure for this disease but they failed.

Just last week, the Food and Drug Administration also delayed the approval of another experimental drug which is made by Sarepta Therapeutics Inc.

FDA rejected Kyndrisa last January after BioMarin failed to provide a statistically significant report that the drug is indeed effective in treating DMD. A month after, last February FDA also rejected another drug for DMD from PTC Therapeutics Inc.

But Bienaime said that BioMarin will still continue exploring other treatment options.  They are now looking at oligonucleotide as a possible area of research for DMD treatment.

Why Biotech Companies Have Difficulty Finding a Cure for DMD?

Duchenne Muscular Dystrophy is a form of X-linked recessive disorder that commonly affects 1 out of 3,500 males. It is a fatal skeletal and cardiac muscle disease caused by a mutation of the dystrophin gene.

DMD has a devastating disease progression where patients unavoidably transition towards total dependency requiring wheelchair at a younger age and ventilation support for survival. Most patients with DMD also suffer other mental health comorbidities like obsessive compulsive disorder and autism spectrum disorder.  DMD is a complex disease that requires multidisciplinary management with neuromuscular, respiratory, and cardiac specialist.  Patients also require help from physiotherapists and other healthcare professionals.

The difficulty of finding a cure lies in the fact that DMD is a genetic disease that needs genetic targeted therapy.  As of today, gene therapy is still a new and evolving field of science.  Molecular Biologist and scientist are still exploring this vast yet complicated field.

Clinical studies on gene therapy are also very expensive considering that they need to conduct a series of animal studies and other experimental trials.  It is also hard to get patients who are willing to undergo the experiment.

In addition, dystrophin is a vital component of the cardiac and skeletal muscles. Biopharmaceutical companies should be very careful in targeting receptors of these muscles because it may cause more harm than good.

One of the concerns of the regulators in approving the commercialization and marketing of the drug is its safety profile. Pharmaceutical companies should be able to prove that their drug is effective and safe for the patients.

Works Cited

Aartsma-Rus, A. (2016). The importance of genetic diagnosis for Duchenne muscular dystrophy. Journal of Medical Genetics , 145–151.

Landfeld, E. (2016). Quantifying the burden of caregiving in Duchenne muscular dystrophy. Journal of Neurology , 906–915.

Nowak, K. J. (2004). Duchenne muscular dystrophy and dystrophin: pathogenesis and opportunities for treatment. EMBO Rep , 872-876.

Parvatiyar, M. S. (2015). Sarcospan Regulates Cardiac Isoproterenol Response and Prevents Duchenne Muscular Dystrophy–Associated Cardiomyopathy. J Am Heart Assoc .


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