Eteplirsen (Exondys 51) for Duchenne Muscular Dystrophy (DMD) approved

Last Updated on

September 19, 2016 – This post presents below a virtually unedited announcement by the American Food & Drug Administration (FDA) concerning Duchenne Muscular Dystrophy (DMD) therapy  option which gives much hope for at least some of the affected patients. However, be aware of the associated conditions on the approval. Please read on below:

The American Food & Drug Administration (FDA) just approved Eteplirsen (Exondys 51) injection, the first drug approved to treat patients with Duchenne Muscular Dystrophy (DMD). Eteplirsen (Exondys 51) 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.

“Patients with a particular type of Duchenne muscular dystrophy will now have access to an approved treatment for this rare and devastating disease,” said Janet Woodcock, M.D., director of the FDA’s Center for Drug Evaluation and Research. “In rare diseases, new drug development is especially challenging due to the small numbers of people affected by each disease and the lack of medical understanding of many disorders. Accelerated approval makes this drug available to patients based on initial data, but we eagerly await learning more about the efficacy of this drug through a confirmatory clinical trial that the company must conduct after approval.”

DMD is a rare genetic disorder characterized by progressive muscle deterioration and weakness. It is the most common type of muscular dystrophy. DMD is caused by an absence of dystrophin, a protein that helps keep muscle cells intact. The first symptoms are usually seen between three and five years of age, and worsen over time. The disease often occurs in people without a known family history of the condition and primarily affects boys, but in rare cases it can affect girls. DMD occurs in about one out of every 3,600 male infants worldwide. People with DMD progressively lose the ability to perform activities independently and often require use of a wheelchair by their early teens. As the disease progresses, life-threatening heart and respiratory conditions can occur. Patients typically succumb to the disease in their 20s or 30s; however, disease severity and life expectancy vary.

Eteplirsen (Exondys 51) was approved under the accelerated approval pathway, which provides for the approval of drugs that treat serious or life-threatening diseases and generally provide a meaningful advantage over existing treatments. Approval under this pathway can be based on adequate and well-controlled studies showing the drug has an effect on a surrogate endpoint that is reasonably likely to predict clinical benefit to patients (how a patient feels or functions or whether they survive). This pathway provides earlier patient access to promising new drugs while the company conducts clinical trials to verify the predicted clinical benefit.

The accelerated approval of Eteplirsen (Exondys 51) is based on the surrogate endpoint of dystrophin increase in skeletal muscle observed in some Eteplirsen (Exondys 51)-treated patients. The FDA has concluded that the data submitted by the applicant demonstrated an increase in dystrophin production that is reasonably likely to predict clinical benefit in some patients with DMD who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping. A clinical benefit ofEteplirsen (Exondys 51), including improved motor function, has not been established. In making this decision, the 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, the FDA is requiring Sarepta Therapeutics to conduct a clinical trial to confirm the drug’s clinical benefit. The required study is designed to assess whether Eteplirsen (Exondys 51) improves motor function of DMD patients with a confirmed mutation of the dystrophin gene amenable to exon 51 skipping. If the trial fails to verify clinical benefit, the FDA may initiate proceedings to withdraw approval of the drug.

The most common side effects reported by participants taking Eteplirsen (Exondys 51) in the clinical trials were balance disorder and vomiting.

The FDA granted Eteplirsen (Exondys 51) fast track designation, which is a designation to facilitate the development and expedite the review of drugs that are intended to treat serious conditions and that demonstrate the potential to address an unmet medical need. It was also granted priority review and orphan drug designation. Priority review status is granted to applications for drugs that, if approved, would be a significant improvement in safety or effectiveness in the treatment of a serious condition. Orphan drug designation provides incentives such as clinical trial tax credits, user fee waiver and eligibility for orphan drug exclusivity to assist and encourage the development of drugs for rare diseases.

The manufacturer received a rare pediatric disease priority review voucher, which comes from a program intended to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases. This is the seventh rare pediatric disease priority review voucher issued by the FDA since the program began.

Eteplirsen (Exondys 51) is made by Sarepta Therapeutics of Cambridge, Massachusetts.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

Tags: , , , , , , , ,
About the Author
thassodotcom Ph.D.; Professor in Pharmacology and Toxicology. Senior expert in theragenomic and personalized medicine and individualized drug safety. Senior expert in pharmaco- and toxicogenetics. Senior expert in human safety of drugs, chemicals, environmental pollutants, and dietary ingredients.

Your opinion

Comment

No comments yet

thasso: conditions

thasso: tweets

thasso post: magazine

View my Flipboard Magazine.

thasso: categories

thasso: archives

thasso: simple chat

You must be a registered user to participate in this chat.

  • New tumor-driving mutations discovered in the under-explored regions of the cancer genome January 18, 2020
    In an unprecedented pan-cancer analysis of whole genomes, researchers at the Ontario Institute for Cancer Research (OICR) have discovered new regions of non-coding DNA that, when altered, may lead to cancer growth and progression.
  • Loss of function in key Y-chromosome genes increases cancer risk in men January 17, 2020
    Numerous studies have shown that men are more susceptible to cancer than women; however, the reason for this difference remains poorly understood. A new study by researchers from the Barcelona Institute for Global Health (ISGlobal) has identified a key biological mechanism that puts men at higher risk of cancer: loss of function in certain genes […]
  • Why we differ in our ability to fight off gut infections January 17, 2020
    Τhe ability of the immune system to fight off bacterial, viral and other invading agents in the gut differs between individuals. However, the biological mechanism by which this happens is not well understood, but at least part of this difference may be explained by genetic factors.
  • Low doses of radiation used in medical imaging lead to mutations in cell cultures January 16, 2020
    Common medical imaging procedures use low doses of radiation that are believed to be safe. A new study, however, finds that in human cell cultures, these doses create breaks that allow extra bits of DNA to integrate into the chromosome. Roland Kanaar and Alex Zelensky of Erasmus University Medical Center and Oncode Institute and colleagues […]
  • Progress in unraveling the mystery of the genomics of Parkinson's disease January 16, 2020
    The International Parkinson Disease Genomics Consortium (IPDGC) has now been in existence for ten years. In an article published in the Journal of Parkinson's Disease the consortium reviews the progress made over the past decade in the genomics of Parkinson's disease (PD) and related disorders including Lewy body diseases, progressive supranuclear palsy, and multiple system […]
Top