Catabasis Pharmaceuticals Presents Positive CAT-1004 Data from Part A of the MoveDMDSM Trial at the 2016 Muscular Dystrophy Association Clinical Conference

by Admin 3/22/2016 8:57:00 AM
 

- Trial Demonstrated Favorable Safety, Tolerability and Pharmacokinetics in Patients -

- Results Support Initiation of Part B of Trial, Expected in First Half of 2016 -

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Mar. 21, 2016-- Catabasis Pharmaceuticals, Inc.(NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced positive data from Part A of the MoveDMD trial of CAT-1004 for Duchenne muscular dystrophy (DMD). The objectives for Part A were to establish safety, tolerability and pharmacokinetics in boys with DMD age 4 to 7 to support initiation of Part B of the MoveDMD trial, a 12-week trial to assess efficacy. All three doses of CAT-1004 tested were generally well tolerated with no safety signals observed. The majority of adverse events were mild in nature, and the adverse events observed were diarrhea (4 events), soft feces (3 events) and upper abdominal pain (2 events). There were no serious adverse events and no drug discontinuations. Six of the total of 9 adverse events were observed in the highest dose group. There were no trends or safety issues in labs, physical exams, EKG or vital signs.

From the pharmacokinetics data, we conclude that doses of 33 mg/kg given 2 or 3 times daily are predicted to achieve systemic exposures at which NF-kB inhibition was observed in adults. Pharmacokinetics demonstrated dose-dependent increases in exposure and meal composition had a modest effect. With single doses of 33 mg/kg, there were minimal differences in either area under the curve (AUC) or maximum serum concentration (Cmax) when CAT-1004 was administered either with a high-fat or a low-fat meal. Based on these results, the daily doses selected for Part B will be 67 or 100 mg/kg administered with food at 33 mg/kg either 2 or 3 times daily. From these results, Catabasis plans to initiate Part B of the MoveDMD trial in the first half of 2016, subject to regulatory approval of the proposed protocol. The Parent Project Muscular Dystrophy and the Muscular Dystrophy Association are providing funding to support participant travel for the MoveDMD trial.

“The favorable safety, tolerability and pharmacokinetic results in boys affected by DMD are encouraging and we look forward to starting Part B of the MoveDMD trial. CAT-1004 is a potential disease-modifying oral DMD therapy regardless of dystrophin mutation type,” said Joanne Donovan, M.D., Ph.D., Chief Medical Officer of Catabasis. “We have received significant support and enthusiasm for our MoveDMD trial and CAT-1004 from parents, advocacy groups, investigators and study staff, and we thank them for their participation and support.”

“The unmet medical need in DMD is profound and potential therapies that could make a meaningful difference are needed” said Richard Finkel, M.D., Division Chief, Division of Neurology, Department of Pediatrics at Nemours Children’s Health System. “Showing positive safety, tolerability and pharmacokinetics results is an important milestone in the development of CAT-1004. I look forward to the advancement of this novel potential therapy.”

CAT-1004 is an oral small-molecule that the Company believes has the potential to be a disease-modifying therapy for the treatment of DMD, regardless of the underlying dystrophin mutation. CAT-1004 is an inhibitor of NF-kB, a protein that is chronically activated in DMD as well as multiple other skeletal muscle disorders and rare diseases. In animal models of DMD, CAT-1004 inhibited NF-kB, reduced muscle degeneration and increased muscle regeneration.

The MoveDMD trial is being conducted in two sequential parts, Part A and Part B. In Part A of the MoveDMD trial, 17 ambulatory boys between ages 4 and 7 with a genetically confirmed diagnosis of DMD across a range of dystrophin mutations received CAT-1004. The boys were steroid naive or had not used steroids for at least six months prior to the trial. Part A of the trial was conducted at three sites in the U.S., and assessed the safety, tolerability and pharmacokinetics of CAT-1004 in patients at three dosing levels (33 mg/kg/day, 67 mg/kg/day and 100 mg/kg/day) during seven days of dosing. Part B will be a randomized, double-blind, placebo-controlled trial to evaluate the safety and efficacy of CAT-1004 in DMD over a 12-week period at 5 clinical trial sites in the U.S. at two dosing levels, 67 mg/kg/day and 100 mg/kg/day, subject to regulatory approval. The boys that participated in Part A of the MoveDMD trial will be asked to participate in Part B, and additional participants will also be enrolled for up to approximately 30 boys. We are currently identifying additional patients who are interested in participating in Part B of the trial. Entry criteria are expected to be similar to those in Part A.

More information about the MoveDMD trial can be found on the clinical trials page of the Catabasis website and on ClinicalTrials.gov under trial identifier NCT02439216.



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Rome holds host to Italian Duchenne Conference and UPPMD meeting

by Admin 2/15/2016 10:19:00 AM

  

On Friday 12th until Sunday 13th, Rome Italys was host to a gathering of patient organisations, Industry and patients with a single common goal of progressing care and therapies for Duchenne Muscular Dystrophy.

Friday's meeting saw Insutry and Patient advocate organisations from over 20 nationalities as far away as China arrive to share ideas on progressing drug development, patient care and access to approved therapies.

Saturday and Sunday saw local and international speakers discuss latest discoveries in biomarkers, endpoints, outcome measures and care. The full program of those that presented and topics of discussion can be seen here

 (pictured are members of UPPMD and other conference delegates and organisers)



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Today - September 7th 2015 is World Duchenne Awareness Day

by Admin 9/7/2015 1:24:00 AM

 

Tell someone about Duchenne and spread the word to support research  

  • Tell your school about Duchenne
  • Tell your colleagues in work about Duchenne
  • Talk to your parents about Duchenne.
  • Change your facebook page to a red baloon
  • Post the Duchenne Manifesto to your social media
 

    

 



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VBP15 To Treat Duchenne Muscular Dystrophy - Clinical Trial Update

by Admin 4/26/2015 9:18:00 AM

In this interview, Eric Hoffman, PhD, president and chief executive officer of ReveraGen Biopharma, provides an update on the phase 1 and phase 2(a/b) studies either underway or planned for the use of VBP15 to treat patients with Duchenne muscular dystrophy. VBP15 is hoped to be a proposed replacement for corticosteroids such as prednisone or deflazacourt with the hope to have fewer side effects.

The Key take away is that the IIa is to start this year and targeted at steroid naive boys with Duchenne from 5-7 years of age in the US only via CINRG sites. Phase IIb to follow internationally.



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Phrixus Pharmaceuticals announces European Access Program for Carmeseal- MDTM (P-188 NF) for patients with Duchenne muscular dystrophy

by Admin 12/1/2014 5:39:00 PM
Phrixus Pharmaceuticals announces European Access Program for Carmeseal- MDTM (P-188 NF) for patients with Duchenne muscular dystrophy
- Ethicor to distribute Carmeseal-MDTM (P-188 NF) in Europe for the treatment of respiratory and cardiac dysfunction in Duchenne muscular dystrophy (DMD) -
ANN ARBOR, MI – 2 December 2014 – Phrixus Pharmaceuticals, Inc. and Ethicor Pharma Ltd. today announced the initiation of their European Access Program (EAP) for Carmeseal-MDTM (P-188 NF). EAP is intended to make Carmeseal-MD available to patients with respiratory and cardiac deficits in DMD through their specialty physicians, primarily cardiologists and pulmonologists, as unlicensed medicinal product (‘Special’). In accordance with local regulations, Ethicor will make Carmeseal-MD available in January 2015 to such patients regardless of their genetic mutation. "For boys and young men with DMD and for their parents time is of the essence. We are committed to making Carmeseal-MD available to patients and their physicians using the most expeditious pathway available,” stated Thomas A. Collet, President and Chief Executive Officer of Phrixus Pharmaceuticals, Inc.
The underlying agreement provides Ethicor with the distribution rights to Carmeseal-MD in the European Union, with the possibility of an expansion to other regions of the world, excluding the United States, Canada and Mexico, prior to and until the registration of the product in the different countries covered by the agreement. Under the European medicines legislation (Directive 2001/83/EC, Article 5(1)), Ethicor will be able to supply, prior to regulatory approval, Carmeseal- MD as a “Special”. A “Special” may be requested by an authorized healthcare professional to meet the special needs of an individual patient under their direct responsibility. Specials cannot be actively promoted to healthcare professionals. Once Carmeseal-MD becomes an approved drug in a given country, the marketing rights to the approved product in that country revert back to Phrixus.
About Carmeseal-MDTM
Carmeseal-MD (Poloxamer 188 NF) has been in clinical development for unrelated indications and has been used as excipient for several decades. In animal models of DMD, it has been shown to improve the efficiency of damaged hearts and to improve
- more -
the performance of damaged diaphragms with once-a-day subcutaneous administration at low doses. When infused into the bloodstream, it encounters and binds to microscopic tears in the muscle. This prevents the pathological leakage of calcium into the cells, which causes calcium overload and keeps the muscle from performing as required. Carmeseal-MD is expected to have its effect in patients with DMD irrespective of the genetic defect that causes the disease.
About Duchenne muscular dystrophy (DMD)
DMD is the most devastating of the muscular dystrophies. It is a genetic disease that affects about 20,000 boys and young men in the United States and a comparable number in Europe. The hallmarks of DMD are skeletal muscle weakness, followed by respiratory distress and heart failure. As a degenerative disease it inevitably leads to premature death, most commonly through respiratory failure but now increasingly through heart failure.
About Phrixus Pharmaceuticals, Inc.
Phrixus Pharmaceuticals, Inc. is developing Carmeseal as Carmeseal-MDTM (P-188 NF for subcutaneous injection) for DMD and as Carmeseal-HFTM (P-188 NF for intravenous administration) for acute decompensated heart failure. Phrixus has assembled the leading global patent portfolio for the use of poloxamers in DMD, heart failure and respiratory dysfunction. For more information: Thomas A. Collet, thomas.collet@phrixuspharmaceuticals.com orwww.phrixuspharmaceuticals.com.

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World Duchenne Awareness Day - 7th of September 2014

by Admin 9/6/2014 11:08:00 AM

 

On the 7th of September 2014, the world will come together for world Duchenne awareness day. The 7th of the 9th has been chosen to represent the 79 exons in the dystrophin gene Dystrophin is the protein missing in boys and girls with Duchenne. Duchenne Charities around the world will raise awareness for boys and young men living with Duchenne muscular dystrophy.This day will be a focal point for the worldwide Duchenne Muscular Dystrophy community with the aim to bring this cruel condition to the attention of a wider global audience. We want to tell you about Duchenne Muscular Dystrophy, how it devastates the lives of the boys who suffer from it and the impact it has on their families and carers. We want to show you why we need to improve the situation for all Duchenne patients who urgently need better access to good care and facilities.

On the 7th of September there will be events and activities all over the world and we have also made a short movie entitled ‘The Many Faces of Duchenne’. This movie which is available on a dedicated website (www.worldduchenneawarenessday.org) highlights the many challenges Duchenne patients are facing. However, in contrast to the physical weakness which Duchenne patients have to cope with, the movie also shows their incredibly strong will to live and take part in life to the greatest extent possible.

Duchenne Muscular Dystrophy affects approximately 250.000 children around the world. The vast majority of Duchenne patients are boys with one in every 3500 newborn boys suffering from Duchenne; only very few girls have this rare disease. Duchenne boys first lose the ability to walk during their childhood days and as they grow into adolescents they also lose the ability to use their arms, carry out everyday activities such as writing or feeding themselves, and their ability to breathe deteriorates as well. As they grow into young men Duchenne boys finally succumb to the disease and die due to respiratory problems or heart failure. But many don't reach adulthood in the first place.

Duchenne patients urgently need good medical care. They need access to specialized centers and multidisciplinary care as well as diagnostics. But we also need to improve the situation concerning the reimbursement of costs for medical care, drugs and/or equipment. Finally, it is absolutely essential that Duchenne boys get improved access to education and increased support to overcome hurdles related to physical and to learning issues. Access to good care and facilities improves not only the quality of life for Duchenne patients but it is also proven to prolong lifespans by between 10 and 20 years. The knowledge about good medical care is widely available, but the majority of Duchenne boys still doesn't have access to good care and facilities. We have to address this and find ways to improve the situation for all Duchenne patients.

There is currently no cure for Duchenne Muscular Dystrophy but for the first time ever there are several drugs under development. In Europe the first drug already received Conditional Approval. Now we have to make sure that all Duchenne patients will benefit from new developments as soon as possible! 

Find out more about the medical care required for those who have Duchenne Muscular Dysptrophy by reading the imperatives of DMD which is now available in 17 different languages. 

 

Dedicated Awareness Website and Video 

We would like to invite you to visit our dedicated World Duchenne Awareness Day website on www.worldduchenneawarenessday.org

 

 

 

Facebook 

Apart from the video you will also find a lot of information on Duchenne Muscular Dystrophy and the activities around the world there. Our Duchenne boys really need your help. The more you know about their plight the more you may be able to support them, directly or indirectly, to improve access to good medical care and facilities. 

You can also like the facebook page here: https://www.facebook.com/worldduchenneawarenessday

Twitter

Please tweet about "World Duchenne Awareness Day" using the hastag #WDAD14 

Duchenne out of this World 

If you would like to show your support, one idea from a Duchenne organisation in Spain is to ‘send a message to the sky’ . At 1pm on Sunday 7th September, send an object into the sky – this could be a balloon, paper aeroplane, sky lantern or a kite – with the message ‘Duchenne out of this world’ written on it. Take a picture and post it with the hashtag #WDAD14.



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PTC Therapeutics Receives Conditional Approval in the European Union for Translarna™ For the Treatment of Nonsense Mutation Duchenne Muscular Dystrophy

by Admin 8/4/2014 11:35:00 AM
August 4, 2014

PTC Therapeutics Receives Conditional Approval in the European Union for Translarna™ For the Treatment of Nonsense Mutation Duchenne Muscular Dystrophy

- The first treatment approved for DMD –

 

SOUTH PLAINFIELD, NJ – August 4, 2014 – PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the European Commission has granted conditional marketing authorization for Translarna™ (ataluren), in the European Union (EU) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients aged five years and older.

"We are delighted that Translarna was approved for the treatment of nonsense mutation Duchenne muscular dystrophy. By targeting the underlying cause of DMD, it has the potential to change the course of the disease. We are moving rapidly to make this product available to patients in the EU as we continue our global efforts to fulfill our vision of making Translarna available to all the boys it may benefit," stated Stuart W. Peltz, Ph.D., CEO of PTC Therapeutics, Inc. "We are grateful to the patients, families, advocacy groups and physicians who have supported PTC Therapeutics through many years of research and development of Translarna. The DMD community has been waiting a long time for treatment options and this conditional approval marks an important day for us all."

The authorization allows PTC to market Translarna in the 28 countries that are Member States of the European Union, as well as European Economic Area members Iceland, Liechtenstein and Norway. As part of the conditional marketing authorization, PTC is obligated to complete its confirmatory Phase 3 trial in nmDMD (ACT DMD) and submit additional efficacy and safety data from the trial.

The approval is based on the safety and efficacy results from a randomized double-blind multicenter study in 174 nmDMD patients for 48 weeks and our additional retrospective analyses of study data. The primary endpoint evaluated the effect of Translarna on ambulation as assessed by the change in distance walked (six-minute walk distance - 6MWD) during a six-minute walk test (6MWT). The post-hoc analysis showed that from baseline to Week 48, patients receiving Translarna (40 mg/kg/day given in 3 doses) had a 12.9 meter mean decline in 6MWD, and patients receiving placebo had a 44.1 meter mean decline in 6MWD. Thus the mean change in observed 6MWD from baseline to Week 48 was 31.3 meters better in the Translarna group than in the placebo group (p=0.056). Furthermore, in more severely affected patients whose baseline 6MWD was less than 350 meters, the mean change in observed 6MWD from baseline to Week 48 was 68 meters better in the Translarna group than in the placebo group. Patients on Translarna also showed a slower rate of decline in ambulation based on an analysis of time to 10 percent worsening in 6MWD. The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency found that these results suggest that Translarna slows the loss of walking ability in nmDMD patients.

Additionally, based on a retrospective analysis, patients receiving treatment also trended better in secondary endpoints such as stair climb and stair descend time-function tests, which the CHMP also found to suggest slowing of nmDMD progression relative to placebo. Safety results showed that Translarna was generally well tolerated. Serious adverse events were infrequent, and none was considered to be related to Translarna. The most frequent adverse reactions at the recommended dose were nausea, vomiting, and headache. These adverse reactions generally did not require medical intervention, and no patients discontinued Translarna treatment due to any adverse reaction.

"The world's first approved treatment for the underlying cause of DMD marks a very important moment for patients and their families. It is our highest priority to make Translarna available to patients and we will be working with regulators, payers, physicians and patient organizations to make that a reality." stated Mark Rothera, Chief Commercial Officer, of PTC Therapeutics, Inc.



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Prosensa Announces Regulatory Path Forward for Drisapersen as a Potential Treatment for DMD

by Admin 6/3/2014 6:53:00 AM
June 3, 2014

Plans to Submit a New Drug Application to the FDA this Year; Dialogue with EMA continues with intent to seek approval

 

 

Leiden, The Netherlands, June 3, 2014 (GLOBE NEWSWIRE) -- Prosensa Holding N.V. (NASDAQ: RNA) the Dutch biopharmaceutical company focusing on rare diseases with a high unmet medical need, today announced that the United States Food and Drug Administration (FDA) has outlined a regulatory path forward, under an accelerated approval pathway, for drisapersen, the Company's lead program for the potential treatment of Duchenne Muscular Dystrophy (DMD). In addition, the company has been interacting with the European Medicines Agency (EMA) and based on these interactions intends to file in Europe as well.

Following the positive feedback from the FDA, Prosensa has confirmed that it will pursue a New Drug Application (NDA) filing for drisapersen with theFDA, under an accelerated approval pathway based on existing data. The company plans to submit a file later this year and will commit to the initiation of two confirmatory post-approval studies. "Given the urgent need to find effective therapies for boys afflicted with this devastating disease, we could not be more pleased with this favorable outcome and with the regulatory authorities' willingness to advance investigational products for the treatment of DMD" said Hans Schikan, Chief Executive Officer of Prosensa.

"We have been diligently preparing for multiple scenarios since acquiring the rights back from GSK in January and completing our more detailed analysis of the drisapersen dataset. The progress announced today is remarkable news for boys and their families, and we remain dedicated to enabling long term patient access to drisapersen and our follow-on products as novel treatments for DMD" he said.

The FDA has outlined the following approaches for confirmatory trials, which the Company is urged to initiate both as soon as possible, as quoted from the guidance letter:

1.       "A historically-controlled trial might be acceptable to confirm clinical benefit following accelerated approval. We note that a historically-controlled study is likely to provide interpretable evidence of efficacy only if the beneficial effect of drisapersen is large, by clearly showing that performance is better in drisapersen-treated subjects than could be reasonably expected, based on knowledge of the natural history of the disease. The effect size would have to be sufficient to overcome the uncertainty inherent in historically controlled trials, and motivational factors that can affect the results.

2.       A randomized, placebo-controlled trial of another exon-skipping drug with a similar mechanism of action, directed at a different exon (e.g., PRO044 or PRO045), with demonstration of a correlation between dystrophin protein production and definitive clinical benefit on 6-minute walk or another measure, could provide confirmatory evidence of drisapersen's clinical benefit if approval were based on a surrogate endpoint."

In conjunction with commencing confirmatory post-approval studies of drisapersen, Prosensa will continue with its plans to re-dose an initial cohort of boys in the third quarter of 2014 who have previously participated in clinical studies with drisapersen. Based on the guidance, one option is  to enroll a number of previously treated patients in these confirmatory studies. Moreover, Prosensa's natural history study, which has enrolled 250 patients, can serve as a historical control.

"Improved understanding of the natural history of DMD and developing effective therapies takes the commitment of pioneers such as Prosensa, and we welcome the news that the FDA is committed to accelerating the pathway for approval of drisapersen and potentially for follow-on exon-skipping therapies" said Dr. Nathalie Goemans, Head of the Neuromuscular Reference Center for Children at the University Hospitals Leuven (UHL) in Belgiumand one of the key investigators in various drisapersen studies.

Prosensa has the most comprehensive pipeline of targeted RNA-based products in development for the treatment of DMD, with three other exon skipping therapies in Phase I/II clinical studies and two programs in pre-clinical development, all of which have been granted orphan drug designation in the US and EU.  Dose finding studies for PRO044 have been completed and are ongoing for PRO045 and PRO053.



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United Parent Project Muscular Dystrophy to host webinar on Translarna (Ataluren)

by Admin 5/30/2014 7:39:00 AM

June 3, 2014 at 1:00pm EST (7pm CEST)

 

In a webinar hosted by United Parent Project Muscular Dystrophy, Robert Spiegel, MD, FACP, chief medical officer of PTC Therapeutics, will present an overview of the positive opinion by the Committee for Medicinal Products for Human Use of the European Medicines Agency regarding the company’s application for a conditional marketing authorization of TranslarnaTM(ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy, and what this decision means for families living in and outside the EU.

 

Additional Information:


To Participate:

  1. Visit ReadyTalk.com and use participant code 9449985. (Be sure to test your computer beforehand)
  2. Audio Dial-In Information:
    • U.S. & Canada: Dial 866.740.1260 and use the Access Code 9449985
    • Outside the U.S. and Canada: Lookup your number


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PTC THERAPEUTICS RECEIVES POSITIVE OPINION FROM CHMP FOR TRANSLARNA™ (ATALUREN)

by Admin 5/23/2014 1:28:00 PM
May 23, 2014

PTC THERAPEUTICS RECEIVES POSITIVE OPINION FROM CHMP FOR TRANSLARNA™ (ATALUREN)

- The first treatment for the underlying cause of Duchenne muscular dystrophy -

 

SOUTH PLAINFIELD, NJ – May 23, 2014 – PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that following its request for re-examination, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion regarding the company's application for a conditional marketing authorization of TranslarnaTM (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients aged five years and older.

"We are very pleased with the outcome of the CHMP review of Translarna's marketing authorization application (MAA) and the level of engagement we experienced with CHMP members throughout the review process," said Robert J. Spiegel, M.D., Chief Medical Officer of PTC Therapeutics, Inc. "We are grateful to the patients, families, advocacy groups and physicians who have supported PTC Therapeutics through many years of research and development of Translarna. It is important to note that this journey continues through the completion of our Phase 3 Translarna confirmatory trial in nmDMD (ACT DMD) which is a high priority for PTC and the DMD community."

Dr. Craig McDonald, Professor of Physical Medicine and Rehabilitation at the University of California, Davis, who developed and validated the 6-minute walk test as a primary clinical endpoint in Duchenne muscular dystrophy (DMD) stated, "This is a historic day for the DMD community. Translarna is the first treatment for the underlying cause of nonsense mutation DMD to receive a positive opinion from the CHMP. The Phase 2b clinical trial provided strong evidence that Translarna slows disease progression as measured by the 6-minute walk test. A clinically meaningful 31.3 meter benefit in 6-minute walk distance, relative to placebo, was achieved in 48 weeks of treatment in patients five years and older and this was supported by positive trends in multiple secondary efficacy endpoints. In addition, in the prespecified group of patients with less than a 350 meter baseline 6-minute walk distance, a 68 meter benefit was observed in patients treated with 40 mg/kg Translarna given daily, relative to placebo. A conditional approval by the European Commission based on this positive opinion would allow children with nmDMD in the European Union to gain access to Translarna while PTC Therapeutics completes its ongoing confirmatory trial."

The CHMP opinion will form the basis for a European Commission (EC) decision as to whether to formally grant the conditional marketing authorization. The European Commission will review the positive opinion from the CHMP and generally delivers its final decision within three months. The conditional marketing authorization would authorize the company to market Translarna with unified labeling in the 28 countries that are Member States of the European Union, as well as European Economic Area members Iceland, Liechtenstein and Norway.

"We congratulate PTC Therapeutics on this landmark decision by the CHMP," stated Elizabeth Vroom, Chair of United Parent Project Muscular Dystrophy (UPPMD). "We applaud PTC for its dedication to the community and its perseverance in pursuing regulatory options to provide Translarna to patients as rapidly as possible. The company's pioneering work has paved the way and encouraged the scientific community to develop new therapies for DMD," she continued. "The EMA is to be commended for recognizing the great unmet need for novel treatments for this relentlessly progressive disease."

Filippo Buccella, President of Parent Project Italy and UPPMD board member, commented, "After thirty years since the discovery of the dystrophin gene, we are finally beginning to see a change in the landscape. For the first time in the history of Duchenne, we see the path to approval for a drug to treat the underlying cause of DMD. This positive result rewards the efforts of a company that has always believed in collaboration with patients and with clinicians. The perseverance of this community made it possible to realize a dream that can give hope to the boys affected with nmDMD in Europe. Our work as a community of patients is not yet finished and we will stand ready to participate with PTC in the ensuing stages of this process and, we hope, with many other companies with new therapies for Duchenne."

PTC requested a re-examination of the CHMP's negative opinion received in January 2014. The positive opinion is based on data and subsequent analysis submitted from a 48-week, 174-patient Phase 2b double-blind, placebo controlled trial which demonstrated that nmDMD patients treated with Translarna (40 mg/kg given daily) walked on average 31.3 meters farther than patients on placebo, as measured by the change in six-minute walk distance (6MWD) from baseline to Week 48. Patients receiving Translarna also demonstrated a slower rate of decline in ambulation, based on an analysis of time to 10 percent worsening in 6MWD. Safety results showed that Translarna was generally well tolerated. Serious adverse events were infrequent and none were considered to be related to Translarna. PTC's global Phase 3 ACT DMD clinical trial is ongoing with full enrollment expected mid-2014.

"The positive opinion from the CHMP recommending the conditional approval of Translarna in nonsense mutation Duchenne muscular dystrophy is a major milestone for the DMD community and we are extremely proud of this joint achievement in accelerating the access to Translarna for patients with nonsense mutation DMD," stated Stuart W. Peltz, Ph.D., CEO of PTC Therapeutics, Inc. "DMD is a progressive disease for which there are currently no approved treatment options. As previously disclosed, we expect to have all patients enrolled in our global Phase 3 ACT DMD by mid-2014. The outcome of this trial is critical for achieving full approval in the EU as well as the US. Assuming that the EC approves a conditional marketing authorization for Translarna in nmDMD, today's decision means that in parallel to this effort, we will be able to provide patients access to Translarna with the immediacy that DMD deserves."

Full Press release here



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