BRUCE’S UNBELIEVABLE GIFT TO HELP HIS PATIENT

Bruce Babington, an Osteopath, based in Ste-Agathe-des-Monts, Québec, is taking on the challenge of cycling across Canada in July 2018. He was inspired by his patient, Anakin Lacasse, who is afflicted with Duchenne muscular dystrophy. Why riding across Canada? To spread knowledge about DMD and raise money for research, the only hope for children like Anakin…

Riding across Canada for hope!

Bruce plans to cycle an average of 200 KM from Mont-Tremblant to Vancouver and complete the journey in 24 days. When he first started to treat Anakin, he didn’t know much about DMD, but it became clear to him very fast that there was no quick fix! He promptly made the decision offer him free services for life. He also had the idea in the back of his mind that someday he would like to go across Canada by bike. He also learned about Fondation La Force, the organization inspired by Anakin, which helps him and other people afflicted with Duchenne to get closer to a cure. At one point, the idea of cycling across Canada for La Force crossed his mind, and he made the decision. He will be taking one month off in July of 2018, with the goal of cycling 200 KM a day and complete the distance between Mt-Tremblant and Vancouver within 24 days.

 

Go big or go home!

“You know, the challenge I’ll face in that 24-day period is nothing like the challenge Anakin is going to face every single day of his life. What I can really do for him is try as much as I can to help promote La Fondation La Force and really spread the knowledge of what Duchenne really is” said Bruce Babington to our team.

 

About Duchenne Muscular Dystrophy (DMD)

A degenerative disease of the muscles for which there is no treatment

Duchenne muscular dystrophy (DMD) is a disease that almost exclusively affects boys and whose incidence is 1 in 3,500. It is extremely rare that Duchenne muscular dystrophy (DMD) will affect girls. Those affected are usually diagnosed around the age of five, but symptoms may be visible from early childhood. It is a degenerative disease of the muscles caused by a genetic mutation. The Duchenne muscular dystrophy (DMD) – for which no treatment is currently available – directly affects skeletal muscles. Without treatment, the consequences of the disease are dire for those afflicted and their families. The normal evolution of the disease inevitably brings about premature death between the ages of 18 and 25.

 

We need your donation:

We have an ongoing campaign to finance Bruce’s ride. In 2018, our team will work hard to give the visibility Bruce needs to unite people around DMD and raise awareness in the general public about the disease.

Thank you in advance to all of our friends in the DMD community, for encouraging Bruce!

 

Don’t forget, sharing our stories is also an excellent way to support our cause.

MAKE A DONATION:  HERE BRUCE CAMPAIGN

 

 

Solid Biosciences Initiates Clinical Trial for Gene Transfer

Last week we had the privilege to meet in person with the team at their headquarter in Boston. They presented their very robust preclinical trial data suggesting that SGT-001, has the potential to slow or stop the progression of DMD, regardless of genetic mutation or disease stage. Now they are announcing the initiating of SGT-001, their investigational microdystrophin gene transfer for DMD and we are so excited as this might become a life-changing treatment for people with DMD.

 

SGT-001 Clinical trial is an Incredible milestone

This is an incredible milestone for this startup company of four years old. This accomplishment is the reflection of years of ongoing collaboration with the top experts in gene therapy. SGT-001 is based on pioneering research in dystrophin biology by Dr. Dongsheng Duan of the University of Missouri and Dr. Jeffrey Chamberlain of the University of Washington.

 

In case you don’t remember the specifics about the microdystrophin and gene therapy we invite you to watch the interview we conducted last year in London with Dr Jeffrey Chamberlain:

 LINK TO VIDEO

 

About the clinical trial:

The Phase I/II clinical trial, called IGNITE DMD, is a randomized, controlled, open-label, single ascending dose study that will evaluate the safety and efficacy of SGT-001 in both ambulatory and non-ambulatory patients with DMD.  IGNITE DMD, adaptive in nature, will allow Solid Biosciences to adjust dose and number of patients as the study progresses to efficiently characterize the safety and efficacy of SGT-001. The patient screening will begin at our first participating study in one location in the United States in the coming days. We are working to bring on additional sites in the United States and abroad.

 

About SGT-001

SGT-001 is a novel adeno-associated virus* (AAV) vector-mediated gene transfer under investigation for its ability to address the underlying genetic cause of DMD. SGT-001 is a systemically administered* candidate that delivers a synthetic dystrophin gene, called microdystrophin, to the body. This microdystrophin encodes for a functional protein surrogate that is expressed in muscles and stabilizes essential associated proteins.

  • Adeno-associated virus > is a small virus which infects humans and some other primate species. Link

  • Systemically administered > Systemic forms of administration affect the whole body (in general).

 

To know more

Open letter de Solid Biosciences

 If you are interested in learning more about this trial (IGNITE DMD), further information will be available on clinicaltrials.gov shortly.

Gene transfer has the potential to address the genetic cause of Duchenne muscular dystrophy.

Press release

Feeling bad about the holidays shopping frenzy?

Giving Tuesday is your opportunity to give back… This is a day dedicated to generosity just as Black Friday and Cyber Monday are dedicated to shopping. Contributing to our cause will help someone afflicted Duchenne muscular dystrophy (DMD). It’s the moment in Canada and the whole world to celebrate giving back and supporting causes that matter to you. We are part of the 3,200, charities and organization celebrating this day in Canada.

 

Help someone afflicted Duchenne muscular dystrophy (DMD)

What is Duchenne? It’s a degenerative muscle disease, afflicting mostly young boys 1-3500. It’s a fatal disease with no cure, taking muscles strength away and leaving young adults in a wheelchair and premature death in their twenties. Many treatments are on the horizon, but we need your help to raise awareness and funds… Because there is no time to lose for people afflicted with DMD. Our organization raise funds for promising research and raise awareness to have a unified DMD community across Canada to access new treatment quickly.

 

Give to La Force here

About GivingTuesday

GivingTuesday was launched in Canada in 2013 by GIV3 and CanadaHelps.org and lists 15 founding partners. It’s a day for the entire charitable sector and encourages all Canadians to join the movement and give and volunteer for charities of their choice. GivingTuesday was initially founded in 2012 in the US by 92Y and the UN Foundation.

How to contribute to our cause:

There is no small amount!

Our organization aim to unite the DMD community to raise awareness around a common objective: that of providing access to new treatments as fast as possible and to participate in the funding of promising research projects. We also raise funds for two promising research based in Canada.

 

Every dollar is taking us closer to find better treatments and the cure.

Give to La Force here

 

Other ways to support us:

  • Organize a fundraiser or a sporting event
  • Buy our promotional items to make gifts
  • Share our stories, video and article
  • Display our colours during your outings and events.

 

http://givingtuesday.ca/fr/partners/18244

News!

2017, November 17th

On a regular basis, team La Force will relay breaking news to the DMD community in News! our latest blog. We provide news highlights to give you quick-and-easy access to the most recent information about what’s happening worldwide in DMD advocacy and, more importantly, advances in therapy and the availability of new treatments. The short news alerts will also appear in our newsletter and, for members of our DMD community, on the Sign-in page.

News! helps you to stay informed, become aware and share what’s happening in the DMD community to bring us closer to a cure.

 

Be aware. Get involved. Share.

 

Jesse’s Journey & Max’s Big Ride fund STAT3 inhibitor research

On October 31, 2017, Jesse’s Journey and Max’s Big Ride – two non-profit groups founded by parents of children with Duchenne muscular dystrophy (DMD) – presented $336,000 in funding to Dr. Patrick Gunning, Professor of Chemistry, Canada Research Chair in Medicinal Chemistry  Tier II, and head of the Gunning Group, a research laboratory at the University of Toronto Mississauga.

Max’s Big Ride raised $35,000 for Max’s Big Fellowship, a new Ph.D. fellowship at the Gunning Group. It was awarded to Yasir Raouf, who will study STAT3 inhibitors in DMD. Earlier this year, Jesse’s Journey approved a $300,000 grant to Dr. Gunning to fund an exciting, first-time, preclinical study of STAT3 and other protein inhibitors to treat DMD.

For more information about the study and how you can get involved: read here

 


Duke University and Sarepta hunt for gene-editing therapy

Muscular Dystrophy News Today reports that Sarepta Therapeutics has teamed up with Dr. Charles Gersbach’s biomedical engineering laboratory at Duke University to develop gene-editing therapies for Duchenne muscular dystrophy (DMD).

Dr. Gersbach is a pioneer in the use of CRISPR/Cas9 technology, a gene-editing tool that can remove abnormal parts of the dystrophin gene. Mutations in this gene cause DMD. Gerbach says that cutting out specific exons (regions that contain the genetic code or “blueprint” for the dystrophin protein) “has the potential to correct a majority of DMD mutations.”

His team has already shown, in mice models of DMD, that removing abnormal regions of the dystrophin gene restores muscle function. The edited gene was able to produce a normally functioning protein that improved muscle strength in mice.

For more information: read here

 


New clinical trial of exon-51 skipping therapy for DMD

Wave Life Sciences has begun the first clinical trial of WVE-210201, an exon 51-skipping therapy, in patients with Duchenne muscular dystrophy (DMD). The multicenter, double-blinded, placebo-controlled, dose-finding, Phase 1 clinical trial will study which intravenous doses of WVE-210201 are safe and tolerable in a subgroup of patients with DMD. About 13% of patients with DMD have exon 51 mutations in the dystrophin gene.

The study will enroll up to 40 patients between 5 and 18 years of age, either able or unable to walk. It will begin in the USA, then expand to Europe and other regions. Results are expected in late 2018.

WVE-210201 is an antisense oligonucleotide that throws a cog in the wheels of protein manufacturing. It forces the cellular machinery to skip over a damaged exon – a specific section of genetic code – then resume regular “reading” of the protein’s “blueprint.” The exon 51-skipping process produces an incomplete but functional dystrophin protein, which may restore some muscle function in patients with this mutation.

For more information: read here

 

Ride for a cure with Dystrospin

On Sunday, November 19, from 7 am to 5 pm, Dystrospin and La Force invite you to spin for Duchenne Muscular Dystrophy. Since 2016, La Fondation La Force has been associated with this event, organized by the Morand family for over five years. All proceeds from the Dystrospin will be donated to two researchers based here in Québec. They are Dr. Jacques Tremblay for his research on CRISPR-Cas9 and Dr. Jérôme Frenette for his research on Osteoprotegerin.

 

For the love of his son…

Jean-Philippe Morand, whose 11-year-old son, Victor, has DMD, will be directing 10 consecutive spinning sessions. The bravest participants will have the opportunity to pedal 10 in a row.

 

An event for everyone

Whether you’re a beginner or experienced, it’s sure to be a pleasurable experience, and above all, a unique chance to test your limits. On site, volunteers will help you adjust your bike and explain the training instructions. For the young ones, daycare service will be offered free of charge for the duration of your participation in the event. Inflatable games, drawing, ball games, etc.: you can bet they’ll have a good time! The little ones are also more than welcome.

 

Every action counts

Duchenne muscular dystrophy is a degenerative disease of the muscles that mainly affects young boys and is 100% fatal. The progression of the disease is unforgiving: the body’s muscles weaken little by little, leaving boys in a wheelchair at the age of 12, with a life expectancy of 20 to 30 years. Nowadays, there is hope through research, and several new treatments are on the horizon. This hope motivates Jean-Philippe to exceed his limits for his son. Every gesture made to raise awareness for the disease will help us fund research and access to new treatments.

 

How to participate

The activity will take place at PerformanceC2. The Centre is located at Place St-Nicolas, 845, Marie-Victorin in St-Nicolas. Since the number of stationary bikes is limited, you must reserve your bike at for a time that suits you.

Registration and online payment on our highly secure site can be found here> https://www.jedonneenligne.org/fondationchudequebec/frm_detail.php?FrmUID=134

 

If you can’t participate in this activity? You can still donate here

 

Learn more:

The FDA closes the door to the approval of ataluren

The FDA has opted not to approve a treatment for DMD, ataluren from PTC Therapeutics. The FDA requires the PTC company to conduct yet another clinical trial in order to consider approval of the treatment. Stuart W. Peltz, CEO of PTC Therapeutics Inc., responded that a formal request called “dispute resolution” will be filed with the FDA next week.

 

This is the third time the FDA has rejected the PTC’s application. This last refusal arrives a month after AdCom, an advisory committee of experts and parents. Here is what Stuart W. Peltz, CEO of PTC, communicated to the DMD community:

 

“We are extremely disappointed for the Duchenne community and strongly disagree with the agency’s conclusions,” said Stuart W. Peltz, Ph.D., chief executive officer of PTC Therapeutics. “We believe that this decision fails to consider the benefit-risk of ataluren and the high unmet medical need. Therefore, we plan to file a formal dispute resolution request next week.”

-Stuart W. Peltz, CEO of PTC Therapeutics Inc.

 

PTC Therapeutics continues to distribute ataluren to the families

The CEO, also reassured families currently receiving treatment through several clinical trial extension programs that they will continue to receive ataluren, throughout the “dispute resolution” appeal. A final decision about the distribution will be made following the outcome of the dispute resolution process. Regarding distribution for Canadian families, there is no information to suggest that there will be any change.

 

For more information, please consult:

Also in the same subject :

 

#AtalurenNow #laforcedmd

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FDA Advisory Committee (AdCom) hears the voice of the DMD community

On September 28, 2017, the U.S. Food and Drug Administration (FDA) held an Advisory Committee (AdCom) meeting to discuss the new drug application for ataluren for oral suspension (PTC Therapeutics Inc.). This committee allows stakeholders to present additional data, information and views on treatment. At the end of this hearing, the AdCom voted that more data is needed to prove that ataluren works in children with Duchenne muscular dystrophy (DMD).

About the results of the vote

The FDA Peripheral and Central Nervous System Drugs Advisory Committee voted overwhelmingly that efficacy data, supporting the PTC Therapeutics Inc. application for marketing approval of ataluren, was inconclusive (10 of 11 votes). Only one vote supported the position that clinical research data showed that ataluren was effective in children with DMD due to a nonsense mutation in the dystrophin gene.

Overall, this vote means that the FDA will continue their conversation with PTC Therapeutics Inc. The information presented to the AdCom will be considered as part of the company’s total submission for marketing approval.

La Force meets the DMD community

The La Force team was present at the AdCom meeting. We interviewed many families whose children with DMD have been on ataluren. They were all very positive about the benefits, and they felt that the FDA heard them. This blog gives an overview of what they had to say about the event, the benefits of treatment, and their reactions to the decision.

The role of families at the AdCom

Being able to complete a Lego set. Having more global energy to study. These incalculable benefits rarely emerge from clinical research data. To go beyond the data, the AdCom heard patient and expert testimonials.

 

Betty Vertin, mother of three boys with DMD

“Basically, it is a meeting that gives us an opportunity to put a case together in front of the FDA. They looked at the data, for them, and the data did not make a convincing argument that ataluren worked. So, they pulled in this group of experts. Basically, they’re giving us our day in court and we can put a face to the data and show what benefit really looks like.”

 

About overall benefits

Many benefits cannot be calculated by clinical research data. For example, for a boy with DMD, having more global energy to study is life changing. This is the reason why testimonials from children and young adults are very important at AdCom meetings.

 
Darrell Knight, father of Jack Knight, a 17-year-old with DMD

“Like I said, it varies from boy to boy, but most boys will usually lose their ability to walk and be in a wheelchair by the time they’re 8 years of age. Roughly around that area. My son was 14, almost 15 years of age. Did that have to do with the drug? We believe so.”

 

Making more treatments available

Eteplirsen is only approved for a small fraction of patients with DMD. The only other FDA-approved therapy, the corticosteroid Emflaza™ (deflazacort), isn’t a cure. Every treatment that gets marketing approval helps to move towards a cure and encourages the pharmaceutical industry to invest in finding new DMD therapies.

Betty Vertin, mother of three boys with DMD

“Getting one more through the door, getting one more approved, opens up the door. More companies are going to invest in Duchenne and be willing to go to bat for us, and those boys deserve that opportunity. So, even though this is a win for a small population of boys who will get access to the drug, it would be an even bigger win for the community.”

Darrell Knight, father of Jack Knight, a 17-year-old with DMD

“I think the FDA is looking for that home run ball, that in-the-park, grand-slam home run. For us, it’s not that at all. For us, it’s base hits. If we get a base hit here, a base hit there, we’re going to be in the ball game. We have to have the base hits, where they’re looking for the home runs. I think, if we can get them convinced that a base hit is worth it, then I think, we’re going to be moving on and I think it’s going to be very encouraging.”

 

Voices from the DMD community

The AdCom listened to voices from the DMD community. What they heard was compelling. Even if the clinical data didn’t convince them of this drug’s efficacy, their words – and message – had a positive impact.

Angela Willette Knight, mother of Jack Knight, a 17-year-old with DMD

“They gave us the opportunity to go and get more information. And they confirmed that what we do have is compelling. So, I think the fact that it is compelling is very huge. And we were heard, and that was part of the totality of the data and part of what went into the decision process.”

 

Anything could happen

The FDA typically follows the advice of its advisory panels. But, last year, an exception occurred when the FDA approved, for the first time, Exondys 51 (eteplirsen; Sarepta Therapeutics), a drug for children with a specific cause of DMD.

Our team feels very good about the positive feedback that the FDA gave to the DMD community. The story will continue on October 24, 2017, when the FDA will make a final decision. At this point, anything could happen.

Interesting links

https://seekingalpha.com/news/3298268-fda-ad-com-says-data-ptcs-translarna-inclusive-needed-prove-efficacy

https://pharmaphorum.com/news/fda-reviewers-slam-ptc-dmd-drug-ahead-key-meeting/

http://www.xconomy.com/new-york/2017/09/28/fda-panel-pans-ptcs-audacious-plan-to-force-vote-on-duchenne-drug/

Emflaza

Exondys 51 (eteplirsen; Sarepta Therapeutics)

Translarna (ataluren; PTC Therapeutics Inc.)

AdCom for Translarna (ataluren) by the FDA

Advisory Committee (AdCom) for Translarna (ataluren) by the FDA

On September 28, the US Food and Drug Administration (FDA) will hold an AdCom meeting for PTC Therapeutics Translarna (ataluren). This committee allows stakeholders to present additional data on treatment. It is important to ensure an in-depth analysis because Translarna (ataluren) acts on a small population of patients with DMD. It is effective on nonsense mutations, accounting for 13% of people with the disease.

An important event for the DMD community

This is an important opportunity for the DMD community to demonstrate its support for the cause. La Force will table a written testimonial of the effects of the drug on a number of Canadian patients being treated with Translarna (ataluren). Our team is participating in this advisory committee to support access to this treatment because approval for new drugs in Canada are closely linked to decisions made in the United States.

What is an FDA Advisory Committee?

FDA Advisory Committees present recommendations from independent experts on a range of complex scientific, technical and policy issues.

Within the framework of this specific committee, testimonials from patients benefiting from treatment will be presented, as well as those of patient associations, foundations, and independent specialists. This opportunity offered by the FDA allows for the evaluation of data added to those usually observed. In the case of a rare disease such as DMD, it is necessary to evaluate in depth, as only a small population of patients is concerned.

Prior to any advisory committee meeting, it is possible to present a verbal or written request indicating that you wish to intervene at the meeting. In general, advisory committees consist of a chair, several members, consumers, industry stakeholders, and sometimes, patient groups. Other specialists with knowledge in specific areas may be invited to individual committee meetings, as required. Finally, the committees present their recommendations to the agency, and the final decisions are made by the FDA.

Our team will be on-site on September 28 to share the results of this day with you live. Stand by and join our community for fresh news about this advisory committee.

Interesting links:

February The Rare Disease Month

Did you know that February is the Rare Disease Month?

 

DMD is also a Zebra

Because it manifests itself only rarely, Duchenne muscular dystrophy (DMD) falls within the rare diseases category. It is also a treatment orphan.

Since this is February, it seemed normal for us to tell you about it.

The zebra has been used as a symbol for rare diseases since about 1940. This comes from a quote by Dr. Theodore Woodward: “When you hear the sound of hooves behind you, do not expect to see a zebra”. This is the metaphor Dr. Woodward used to teach students basic concepts about the diagnosis of a disease: when examining a patient’s symptoms, it’s better to think of a horse rather than a zebra. It’s a fact that horses are hoofed animals more commonly encountered than zebras, so you should automatically assume that if you hear the sound of hooves, it should be a horse, not a zebra, right?

Conversely, if we can’t confirm a common disease, it may be because it is a zebra, and continue the research among the approximately 7,000 known rare diseases. This inevitably results in very long diagnosis delays, along with a lot of consequences this entails.

A few distinctions …

A rare disease is defined as a disease that affects less than 1 in 2000 (DMD affects 1 in 3500-5000, which makes it a rare disease).

The RQMO estimates that, in Québec, nearly one in 20 is afflicted by or carrier of a rare disease, for a total of nearly 500,000 Quebecers. Many rare diseases are chronic, progressive and fatal. The CORD estimates approximately, 3 million Canadians and their families face a debilitating disease that severely impacts their lives.

Nearly 75% of these diseases affect children and about 80% of them are genetic.

A few examples of rare diseases: cystic fibrosis, Duchenne muscular dystrophy, Huntington’s disease, autoimmune myasthenia and Angelman Syndrome.

The term “orphan disease” is often confused with the term “rare disease” because the vast majority of rare diseases are orphans in many ways.

There are approximately 350 orphan drugs for about 7000 rare diseases.

A medical condition is referred to as an orphan disease if there is no treatment for the disease other than treating the symptoms, as is the case for DMD: for example, prednisone is a medication that has an effect on the symptoms but not on the cause of the disease.

The rarity of these diseases creates obstacles and needs for afflicted people both in our health system and in society in general.

The Quebec Coalition for Orphan Diseases (RQMO) works to provide information and support to patients, their families, and healthcare professionals. Their website is full of relevant information both for professionals and caregivers. Being rare, these diseases are often of little interest to researchers and organizations who fund research. The RQMO aims to advance knowledge about the various rare and orphan diseases by promoting exchanges between patients and researchers.

The Canadian Organization for Rare Disorders (CORD) is Canada’s national network for organizations representing all those with rare disorders. CORD provides a strong common voice to advocate for health policy and a healthcare system that works for those with rare disorders. CORD works with governments, researchers, clinicians and industry to promote research, diagnosis, treatment, and services for all rare disorders in Canada.

February 28Th is the Rare Disease Day to raise awareness amongst the general public and decision-makers about rare diseases and their impact on patients lives.

We wish to emphasize that the information contained in this article come mainly from the RQMO website and the CORD website.

We thank them.

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Utrophin production might be a good option for people with DMD

Utrophin could potentially replace dystrophin. In people with Duchenne muscular dystrophy (DMD), dystrophin does not function properly. Numerous media outlets have recently reported many exciting developments in the search for new treatments for Duchenne muscular dystrophy (DMD). Science, medicine, and technology are uniting to create significant results. This article is the first in a new series to shed light on promising, experimental treatment options. This first article will feature utrophin production.

What is utrophin?

The human body naturally produces utrophin, a protein, when a muscle is first forming or when a muscle is repairingAs a muscle matures, dystrophin replaces utrophin. However, in people with Duchenne muscular dystrophy (DMD), dystrophin does not function properly.

Utrophin is functionally and structurally similar to dystrophin. Preclinical trials that have stimulated sustained utrophin production have shown that it could potentially replace dystrophin in people with Duchenne muscular dystrophy (DMD). The replacement of dysfunctional dystrophin with functional utrophin might have a highly positive impact on muscle performance.

Summit Therapeutics believes that utrophin may slow or even stop the progression of DMD.

More information: PPMD  –  Wikipedia  – NCBI

What is ezutromid?

Ezutromid is an utrophin modulator.

It is an orally administered, small molecule, experimental drug, in development by Summit Therapeutics.

More information

How does it work?

Ezutromid stimulates the body to sustain production of utrophin.

This experimental therapy has the potential to work in people with all genetic profiles of Duchenne muscular dystrophy (DMD). Given that it doesn’t depend on a specific genetic profile, this treatment may be suitable for 100% of people with DMD.

Clinical Trial

Summit Therapeutics just announced that the first patients have been enrolled into trial sites in the US into PhaseOut DMD. Summit Therapeutics will be making updates about additional US sites and contact details to the clinicaltrials.gov record (https://clinicaltrials.gov/ct2/show/NCT02858362). As a reminder, PhaseOut DMD is a 48-week, open-label (meaning all participants receive ezutromid) Phase 2 trial, is ongoing in the UK and the US. Enrolment of approximately 40 patients continues, and they expect to complete trial enrolment in the second quarter of 2017.

For more information about PhaseOut DMD clinical trial of ezutromid: Utrophin Trials   –  Clinical trials

What’s the regulatory status of ezutromid?

Ezutromid is an experimental drug candidate in Phase 2 clinical trialThe U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have granted Orphan Drug designation to ezutromid. Orphan drugs receive a number of benefits, including additional regulatory support and a period of market exclusivity after marketing approval. In addition, the FDA has granted ezutromid both Fast Track and Rare Pediatric Disease designations.

Recently, Summit Therapeutics Plc applied to the MHRA and FDA regulatory authorities to proceed with the planned extension of PhaseOut DMD for patients currently enrolled in the trial. The extension phase is expected to last until ezutromid either receives marketing approval in relevant countries or its development is discontinued.

In addition to PhaseOut DMD, they plan to conduct a randomised, placebo-controlled trial designed with the potential to support accelerated and conditional approvals for ezutromid in the US and Europe. It is anticipated that this trial would start after positive interim data from PhaseOut DMD.

Summit to Extend Ongoing PhaseOut DMD Clinical Trial of Ezutromid in Patients with DMD: Press Release 

How is ezutromid administered?

Ezutromid is given orally, as a liquid.

Muscular Dystrophy News

A word from the manufacturer

“The Rare Pediatric Disease designation builds upon the Fast Track and Orphan Drug designations, which the FDA has already awarded to ezutromid, recognizing a significant unmet medical need in the treatment of DMD,” says Glyn Edwards, Chief Executive Officer of Summit.

“We plan to leverage these regulatory advantages in the continued clinical development of ezutromid, which is currently in Phase 2 clinical trial called PhaseOut DMD, to bring ezutromid to patients in need as quickly as possible.”

For more information about ezutromid click here

 

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