New treatments for Duchenne muscular dystrophy: a stalled process

Initial Duchenne muscular dystrophy diagnoses usually come about around age 4 or 5 years, mostly in young boys. Duchenne muscular dystrophy often referred to as “DMD”, is the most severe form of muscular dystrophy.

It is a degenerative disease that gradually weakens every muscle in the body, and that inevitably leads to premature death when afflicted kids reach their twenties. The corticosteroid-type treatments currently available – used for anti-inflammatory purposes – can prolong certain muscle functions; however, the side effects of these products are devastating in the long run!

A difficult approval process for effective treatments

For the first time in the history of Duchenne muscular dystrophy, innovative treatments that directly address the genetic causes of the disease are currently undergoing the FDA (Food and Drug Administration) approval process in the United States: Ataluren (PTC Therapeutic), Eteplirsen (Sarepta Therapeutics) and Drisapersen (BioMarin).

Ataluren is used in cases where on children happen to have a so-called “nonsense” genetic mutation, while the action of the other two, eteplirsen and drisapersen, has to do with the “exon skipping” principle.

Earlier this year, Sarepta Therapeutics, the pharmaceutical company developing Eteplirsen, was informed that there would be a delay in the process; the most recent news is that the matter is to be re-evaluated by a committee. Less than a month ago, it was PTC Therapeutics’ turn to be told, much to their surprise, that certain elements of information were missing, despite the fact that its drug, Ataluren, had been approved in Europe (May 2014 – read more).

Undue delays for children who have no time to wait

The FDA’s decisions influence those of all other jurisdictions. Failure to get approval for these pioneering treatments could have the effect of discouraging other pharmaceutical companies from developing alternative treatments for Duchenne muscular dystrophy. Worthy of mention is the fact that, although the data is currently deemed insufficient by the authorities, clinical trials have clearly demonstrated the positive effects of these treatments on children, with very few side effects.

These delays sometimes cause pharmaceutical companies to lose significant value in the stock market, which is unfortunate. But even worse are the years of life the kids lose due to a blocked approval process that prevents them get the treatment they need.

In fact, the community has but one choice, and that is to take action… Otherwise, no treatment!

Knowledge before action!

Of course, we do not at present have all the answers: Team La Force’s mission is to develop expertise in the field and broadcast to the Canadian Duchenne Muscular Dystrophy community any relevant information pertaining to access to treatment.

We’ve given ourselves the mandate to inform the DMD community because we believe that disseminating knowledge is a great way to change things. With this in mind, we are announcing with great pleasure that La Force will be attending the FDA-AdComm conference to be held in Washington in late April. Our goal is to acquire as much knowledge as we can about the North American treatment approval processes and keep you informed.

What should you do as a parent of a child afflicted with Duchenne muscular dystrophy?

If your child is suffering from DMD, we suggest you do the following:

  1. In consultation with competent medical personnel, determine your child’s specific genetic condition, find out the exact reason why he/she is afflicted with DMD; it will guide you as to what treatments, existing or in development, are appropriate to his/her situation;
  2. Learn about these treatments and work to get access to them: it’s a good idea to consult with us, because, with time we’ve learned a lot on the subject matter;
  3. Communicate with the people around you and share the information in order to raise awareness about the disease, about the availability of new treatments and about the long and laborious approval processes;
  4. Follow us on the web and on social networks in order to stay abreast of developments and benefit from the support of the DMD community.

Link about New treatments for Duchenne muscular dystrophy

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Sarepta Therapeutics

Eteplirsen – The Facts About Eteplirsen – Uptick Analyst – FDA Approval Status – FDA: Please accept Sarepta’s New Drug Application (NDA) for Eteplirsen

 

Biomarin

Drisapersen – FDA Approval Status (Drisapersen (Kyndrisa) – FDA Decision on Drisapersen for Duchenne Muscular Dystrophy

 

PTC Therapeutics

Fierce Biotech – PTC: With no evidence of Duchenne MD efficacy, FDA barred regulators’ doors to ataluren

Results from Phase 3 ACT DMD Clinical Trial of Translarna™ (ataluren) – Ataluren at Health Canada – Recommandation pour le remboursement NICE (UK)

 

FDA Draft Guidance on Duchenne

The Wall Street Journal