SAN RAFAEL, Calif., Nov. 20, 2015 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) today announced data analysis for Kyndrisa demonstrating consistent evidence of efficacy in comparable patients across three randomized, placebo-cont...
- 152 DMD patients now on Translarna™ (ataluren) commercial therapy - - Strong sales growth with YTD 9/30 Translarna revenues of $21.0M - - Phase 3 ACT DMD clinical trial results confirm clinical benefit of Translarna - - SMA program expected to resume clinical development in early 2016 -
Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage drug development company built on a pathway pharmacology technology platform, today announced that the European Commission (EC) has granted orphan medicinal product designation to CAT-1004 for the treatment of Duchenne muscular dystrophy (DMD). CAT-1004 is designed to inhibit activated NF-kB, a protein that plays an important role in muscle health. CAT-1004 is a potential disease-modifying therapy being developed to reduce muscle inflammation and degeneration and promote muscle regeneration in patients affected by DMD regardless of the underlying dystrophin mutation. The U.S. Food and Drug Administration (FDA) has previously granted Fast Track, Orphan Drug, and Rare Pediatric Disease Designations for CAT-1004 for the treatment of DMD.
After yesterday’s news regarding the Ad Comms for both BioMarin and Sarepta, the community continues to ask questions. PPMD has been working around the clock…
After yesterday’s news regarding the Ad Comms for both BioMarin and Sarepta, the community continues to ask questions. PPMD has been working around the clock to get some answers and will continue to share information with you as it becomes available. One topic of discussion is the question of ‘unmet need’ – if one therapy is approved does that hurt the potential of the other therapy because there is no longer the urgency of unmet need?
Dr. John Porter and Kathi Kinnett from the PPMD team recently attended the World Muscle Society (WMS) meeting in Brighton, UK and will be reporting back over…
PPMD's Dr. John Porter and Kathi Kinnett report back with updates from Days 2-3 of the World Muscle Society Congress, including:
► Highlights from symposiums sponsored by PTC & BioMarin ► Clinical Duchenne poster topics ► Work on putative biomarkers and modifier genes ► Updates on therapy development programs (Translarna, eteplirsen, HT-100, & more)
"Together with the recently completed patient and caregiver survey prepared by PPMD, which underlined the importance of treating pulmonary complications in patients with DMD, we have now all data we wanted to include in our regulatory filing dossiers according to our previous discussions with regulators. The expected shorter review timelines due to the fast track status allowing for priority review in the US and a filing as a Type II variation of the existing marketing authorization in the EU should lead to regulatory decisions next year. The new study we plan to start soon addresses a clear medical need for the treatment of respiratory function decline also in patients using glucocorticoid steroids and follows requests by physicians and the DMD community. If successful, we will seek to extend the label accordingly to include this substantial group of DMD patients."
- PTC plans to complete rolling NDA submission to the FDA by end of 2015 - - Totality of clinical data demonstrate Translarna's ability to slow disease progression for patients with nonsense mutation Duchenne muscular dystrophy - - 15 meter benefit observed in 6MWD in overall study population (p=0.213, n=228) - - 47 meter benefit observed in 6MWD in pre-specified subgroup of 300 - 400 meters at baseline (p=0.007, n=99) - - Pre-specified meta-analysis of combined ACT DMD and Phase 2b studies demonstrated benefit in Translarna-treated patients across primary (p=0.015, n=291) and key secondary efficacy endpoints - - Strong safety profile of Translarna confirmed, consistent with previous studies -
PTC Therapeutics has announced promising results from the Phase 3 ACT DMD Clinical Trial of Translarna™ (ataluren).
Today we learned that the FDA will host an Advisory Committee (Ad Comm) meeting on November 24 to discuss the NDA for BioMarin’s drisapersen compound. Tomorr…
We all have many questions about what the January Ad Comm date for Sarepta, after the PDUFA date for BioMarin may mean. And PPMD is working with our regulatory experts to seek those answers and to understand how we can continue to work together as a community to ensure that both products are offered regulatory pathways to success so that patients ultimately may have access to either product, if approved. Read the latest blog from PPMD President Pat Furlong to learn more.
This is our final update from the 2015 World Muscle Society meeting in Brighton, United Kingdom (click here for updates from previous days). Before providing…
PPMD's Dr. John Porter and Kathi Kinnett share their final update from the 2015 World Muscle Society Congress – an exciting meeting for Duchenne! There were four major symposia, all of which included information about Duchenne (with the BioMarin, PTC Therapeutics, and Sarepta symposia very much dominated by Duchenne) and at least five Duchenne-intensive poster sessions that included preclinical studies, mechanistic research, therapy development, and patient care topics.
Throughout the week, several presenters mentioned specific PPMD activities, including our efforts in newborn screening, developing the FDA guidance, benefit risk, and the certified Duchenne care centers. Total attendance at the meeting was said to be 750 and there were many opportunities for us to interact with company representatives and academic researchers to emphasize the PPMD mission and strategies to end Duchenne.
Dr. John Porter and Kathi Kinnett from the PPMD team are attending the World Muscle Society (WMS) meeting in Brighton, UK and will be reporting back over the…
Yesterday was a big day for Duchenne at WMS in Brighton, UK. The morning sessions included several talks, and both afternoon poster sessions covered Duchenne gene and imaging, and functional outcome measures, plus the symposium by Sarepta.
PPMD is excited to today announce a collaboration with BioMarin Pharmaceutical Inc., PTC Therapeutics, and Sarepta Therapeutics, Inc. in the next phase of the…
PPMD is proud to announce a collaboration with BioMarin Pharmaceutical Inc., PTC Therapeutics, and Sarepta Therapeutics, Inc. in the next phase of the highly successful Decode Duchenne program. Thanks to the collaboration and generous support of these partners, Decode Duchenne will continue to provide genetic testing, interpretation, and counseling at no cost to eligible patients who are unable to access testing due to barriers such as a lack of insurance or insufficient insurance coverage. Identifying people with Duchenne muscular dystrophy is critical in our fight to end this devastating disorder so that optimal care is provided as soon after diagnosis as possible, as well as connecting patients to the most relevant clinical trials or approved therapies. We could not be more appreciative or proud to collaborate with three exceptional industry partners who continue to prove their commitment to the Duchenne community by supporting important programs like Decode Duchenne. The program will be administered by PPMD through DuchenneConnect. Visit DuchenneConnect.org today to learn more!
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced that Edward Kaye, MD, Sarepta’s interim chief executive officer and chief medical officer will provide a corporate update and report on recent data from the Phase IIb study of eteplirsen for Duchenne muscular dystrophy via a live webcast and conference call on October 1, 2015 at 7:00 AM EST. The update will be followed by a panel discussion with Duchenne muscular dystrophy experts Anne Connolly, MD; Eugenio Mercuri, MD, PhD; Jerry Mendell, MD; Perry Shieh, MD, PhD; and Steve Wilton, PhD, BSc.
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