Duchenne muscular dystrophy (DMD)

Sarepta’s Unusual Journey

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Before approving an investigational drug, safety and effectivity needs to be proven. Something different happened with Sarepta’s Muscular dystrophy drug Eteplirsen, as its luck changed completely when the U.S. Food and Drug Administration on 19th September 2016 approved Sarepta Therapeutics’ Exondys 51 (eteplirsen) injection to treat patients with Duchenne muscular dystrophy (DMD). Exondys51 is specifically indicated for patients who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping, which affects about 13 percent of the population with DMD. This is the first drug to get approved for this rare disease.

Earlier in March, 2016, FDA had declared that it was not able to decide on Sarepta’s eteplirsen and needed more time to review. In making this decision, FDA considered the potential risks associated with the drug, the life-threatening and debilitating nature of the disease for these children and the lack of available therapy. Under the accelerated approval provisions, FDA required Sarepta Therapeutics to conduct a clinical trial to confirm the drug’s clinical benefit. In a complete turn of events, FDA approved Exondys 51 (eteplirsen) for the treatment of Duchenne muscular dystrophy (DMD), leading the stock to more than double in price in just a few days.

Sarepta’s drug has many health insurance providers are on board with it, others like Anthem Inc are still questioning whether it will do its customers any good — given the drug’s accelerated approval. Additionally, on October 5, Sarepta Therapeutics and Summit Therapeutics partnered to advance the development of novel therapies for the treatment of Duchenne muscular dystrophy.

Insight by:
Sadaf Javed
Associate Analyst
DelveInsight Business Research


DelveInsight’s Genetic disorders based Gene Therapy Reports

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Gene Therapy to “fix” Genetic disorders!

“We used to think that our fate was in our stars, but now we know that, in large measure, our fate is in our genes, “quotes James Watson. This fate and our future is locked in our genes, and Gene therapy is unlocking these doors. Many physicians are predicting that in twenty years gene therapy may change the practice of medicine from a treatment-based to a prevention-based practice.

About Gene Therapy

Gene therapy is a novel therapeutic branch of modern medicine. Its emergence is a direct consequence of the revolution heralded by the introduction of recombinant DNA methodology in the 1970s. Gene therapy is still highly experimental, but has the potential to become an important treatment regimen. Basically, it allows the transfer of genetic information into patient tissues and organs in order to eliminate or restore the normal functions of the diseased genes.

Genetic disorders affect millions of people world-wide. Scientists have currently identified more than 4000 different genetic disorders. These are the harmful effects on an individual caused by inherited genetic diseases or mutations. Usually genetic disorders are recessive, so they are only expressed in a small percentage of the population, but a much larger percentage are carriers. When expressed in the homozygous recessive individual, they often code for the wrong protein or amino acid sequence. Gene therapy aims to get rid of these genetic conditions at their source.

Gene Therapy: Mending Genetic disorders

Gene therapy has made important medical advances in less than two decades. Within this short time span, it has moved from the conceptual stage to technology development and laboratory research to clinical translational trials for a variety of deadly diseases. During this period molecular genetic technologies have been spectacularly successful in identifying and characterizing novel disease genes, and in devising novel diagnostic tests for inherited disorders. Current and possible candidates for gene therapy in the field of genetic disorders include Severe Combined Immune Deficiency (SCID), Adenosine deaminase (ADA) deficiency and Duchenne muscular dystrophy (DMD) etc.

Gene therapy is likely to be most successful with diseases caused by single gene defects which is due to the absence of complex interplay between different genetic loci and/or environmental factors. However, some genetic disorders may not be so easy to treat as others and within the single gene disorder category differing pathogeneses means that certain single gene disorders will be more amenable to gene therapy approaches than others. However, low efficiency of gene transfer & expression and low longevity of gene expression are the acting limitations in this field. Inspite of this, there is much optimism and confidence in the potential of Gene therapy to develop an effective treatment for genetic disorders in the near future.

Gene Therapy: The Market Scenario

Many companies are investing in and researching on this field using gene therapy due to its promising effects. Large Pharmaceutical and Biotech giants, such as GlaxoSmithKline, Sanofi, and Bluebird Bio etc., are operating in the field of Genetic disorders in the gene therapy domain. A growing number of partnership between companies in drug development for example between Oxford BioMedica and Sanofi etc., are driving the new gene therapy research. The collective pipeline of industry is brimming with 300+ therapies of which genetic disorders collectively accounts for 11 % of gene therapies in pipeline. The global market opportunities for gene therapy have risen drastically and continue to be of great interest for many companies globally.

DelveInsight’s Gene Therapy Reports

DelveInsight’s Gene Therapy Reports cover the entire gene therapy market insights for genetic disorders including technology assessments, licensing opportunities, collaborations, market trends, pipeline coverage and competitive landscape. The report essentially provides DelveInsight’s proprietary market and pipeline analytics which identifies the front runners in this therapeutic area. It also identifies the potential market movers and future regulatory landscape.