EMA orphan designation is intended to encourage development of medicines for the diagnosis, treatment, or prevention of life-threatening or chronically debilitating conditions that affect no more than 5 in 10,000 people in the EU. Medicines that receive orphan designation are eligible for a number of incentives, including assistance with development of the medicine; reduced fees for marketing-authorization applications; and extended market exclusivity once the medicine is authorized. Orphan designation is conferred following a positive opinion by the EMA's Committee for Orphan Medicinal Products (COMP).
About Resolaris
aTyr Pharma is developing Resolaris as a potential first-in-class intravenous protein therapeutic for the treatment of rare myopathies with an immune component. Resolaris is derived from a naturally occurring protein released by human skeletal muscle cells. aTyr believes Resolaris has the potential to provide therapeutic benefit to patients with rare myopathies with an immune component characterized by excessive immune cell involvement.
About LGMD2B
Limb girdle muscular dystrophy (LGMD) refers to a group of rare genetic myopathies, of which there are more than 20 different subtypes, none with approved therapies. LGMD affects an estimated 16,000 patients in the U.S., approximately 3,000 of whom have LGMD2B. LGMD2B is a recessive genetic disease caused by a toxic loss of function in the dysferlin gene. Patients experience progressive debilitating muscle weakness and atrophy as well as immune cell invasion in the skeletal muscle. To learn more about LGMD2B please visit http://www.jain-foundation.org.
About aTyr Pharma
aTyr Pharma is engaged in the discovery and clinical development of innovative medicines for patients suffering from severe, rare diseases using its knowledge of Physiocrine biology, a newly discovered set of physiological pathways. The Company's lead candidate, Resolaris, is a potential first-in-class intravenous protein therapeutic for the treatment of rare myopathies with an immune component. aTyr has built an intellectual property estate, to protect its pipeline, comprising over 80 issued or allowed patents and over 230 pending patent applications that are owned or exclusively licensed by aTyr, including over 300 potential Physiocrine-based protein compositions. aTyr's key programs are currently focused on severe, rare diseases characterized by immune dysregulation for which there are currently limited or no treatment options. For more information, please visit http://www.atyrpharma.com.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Litigation Reform Act. Forward-looking statements are usually identified by the use of words such as "anticipates," "believes," "estimates," "expects," "intends," "may," "plans," "projects," "seeks," "should," "will," and variations of such words or similar expressions. We intend these forward-looking statements to be covered by such safe harbor provisions for forward-looking statements and are making this statement for purposes of complying with those safe harbor provisions. These forward-looking statements, including statements regarding the potential of Resolaris, the ability of the Company to undertake certain development activities (such as clinical trial enrollment and the conduct of clinical trials) and accomplish certain development goals, and the timing of initiation of additional clinical trials and of reporting results from our clinical trials reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations, strategies and prospects as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a variety of risks and factors that are beyond our control including, without limitation, risks associated with the discovery, development and regulation of our Physiocrine-based product candidates, as well as those set forth in our most recent Annual Report on Form 10-K for the year ended December 31, 2015 and in our subsequent SEC filings including our most recent Quarterly Report for the quarter endedSeptember 30, 2016. Except as required by law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.
Contact:Mark JohnsonSr. Director, Investor Relations mjohnson@atyrpharma.com 858-223-1163
To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/atyr-pharma-receives-ema-orphan-drug-designation-for-the-treatment-of-limb-girdle-muscular-dystrophy-with-resolaris-300417615.html
SOURCE aTyr Pharma, Inc.
Read the original:
aTyr Pharma Receives EMA Orphan Drug Designation for the Treatment of Limb Girdle Muscular Dystrophy with ... - PR Newswire (press release)
- Muscular .Dystrophy -Successful-treatment by acupressure , Ayurveda, Yoga [Last Updated On: May 10th, 2011] [Originally Added On: May 10th, 2011]
- Muscular Dystrophy Treatment - Todd Harrison Improving Balance [Last Updated On: May 16th, 2011] [Originally Added On: May 16th, 2011]
- Oculopharyngeal Muscular Dystrophy (OPMD): Exploring Causes and Treatment [Last Updated On: May 20th, 2011] [Originally Added On: May 20th, 2011]
- Muscular Dystrophy Treatment - Todd Harrison holding body weight [Last Updated On: May 21st, 2011] [Originally Added On: May 21st, 2011]
- David G VECTTOR Muscular Dystrophy Treatment double arm strength month 6 [Last Updated On: May 22nd, 2011] [Originally Added On: May 22nd, 2011]
- David Gould Becker Muscular Dystrophy VECTTOR Treatment [Last Updated On: June 2nd, 2011] [Originally Added On: June 2nd, 2011]
- Muscular Dystrophy Halo [Last Updated On: June 3rd, 2011] [Originally Added On: June 3rd, 2011]
- Muscular Dystrophy VECTTOR Treatment Documentary - Todd's 6 Month Journey [Last Updated On: June 7th, 2011] [Originally Added On: June 7th, 2011]
- Muscular Dystrophy Treatment Results - David Gould - New Footage [Last Updated On: June 8th, 2011] [Originally Added On: June 8th, 2011]
- Muscular Dystrophy - Rewriting History with VECTTOR [Last Updated On: June 10th, 2011] [Originally Added On: June 10th, 2011]
- Muscular Dystrophy - David Gould 5 month VECTTOR treatment for BMD [Last Updated On: June 12th, 2011] [Originally Added On: June 12th, 2011]
- Duchenne's Muscular Dystrophy Stem Cell Treatment - Reelabs India [Last Updated On: June 14th, 2011] [Originally Added On: June 14th, 2011]
- Muscular Dystrophy VECTTOR Treatment - Todd Harrison/Luau Presentation [Last Updated On: June 16th, 2011] [Originally Added On: June 16th, 2011]
- Dr. William Rader - Muscular Dystrophy Breakthrough [Last Updated On: June 16th, 2011] [Originally Added On: June 16th, 2011]
- Muscular Dystrophy patient at Xcell-center - Nabeel Mohamed Abdulhusain, 46 years [Last Updated On: June 17th, 2011] [Originally Added On: June 17th, 2011]
- Charlie's Story: Duchenne Muscular Dystrophy Part 4 [Last Updated On: July 18th, 2011] [Originally Added On: July 18th, 2011]
- Muscular Dystrophy VECTTOR Treatment - David Gould/Luau Presentation [Last Updated On: July 19th, 2011] [Originally Added On: July 19th, 2011]
- Testimonial 4 of Muscular Dystrophy after Stem Cell Therapy [Last Updated On: August 5th, 2011] [Originally Added On: August 5th, 2011]
- Drug Combo Dynamic in Muscular Dystrophy [Last Updated On: August 9th, 2011] [Originally Added On: August 9th, 2011]
- Testimonial for Fetal Stem Cell Treatment of Duchenne Muscular Dystrophy [Last Updated On: August 25th, 2011] [Originally Added On: August 25th, 2011]
- Improvement seen in Duchenne Muscular Dystrophy after Stem Cell Therapy [Last Updated On: September 11th, 2011] [Originally Added On: September 11th, 2011]
- Muscular Dystrophy STS/VECTTOR treatment results [Last Updated On: September 24th, 2011] [Originally Added On: September 24th, 2011]
- Muscular Dystrophy VECTTOR Treatment Documentary - Todd's One Year Journey [Last Updated On: October 2nd, 2011] [Originally Added On: October 2nd, 2011]
- Muscular Dystrophy Treatment Day 18 - Todd Harrison's Improvement [Last Updated On: October 2nd, 2011] [Originally Added On: October 2nd, 2011]
- Wang Yisheng - Muscular Dystrophy Adult Stem Cell Patient [Last Updated On: October 9th, 2011] [Originally Added On: October 9th, 2011]
- Becker Muscular Dystrophy Miracle TREATMENT [Last Updated On: October 11th, 2011] [Originally Added On: October 11th, 2011]
- Muscular dystrophy patient_Kleber_Brazil.wmv [Last Updated On: October 12th, 2011] [Originally Added On: October 12th, 2011]
- Testimonial 1 of Muscular Dystrophy after Stem Cell Therapy - Video [Last Updated On: October 18th, 2011] [Originally Added On: October 18th, 2011]
- Becker Muscular Dystrophy STS Treatment - Video [Last Updated On: October 18th, 2011] [Originally Added On: October 18th, 2011]
- Potential Stem Cell treatment of Duchenne Muscular Dystrophy - Video [Last Updated On: October 21st, 2011] [Originally Added On: October 21st, 2011]
- Muscular Dystrophy treated by Dr Rajesh Shah at Life Force - Video [Last Updated On: October 22nd, 2011] [Originally Added On: October 22nd, 2011]
- Defying Muscular Dystrophy - I Made It - Video [Last Updated On: October 23rd, 2011] [Originally Added On: October 23rd, 2011]
- Becker Muscular Dystrophy AMAZING treatment results - Video [Last Updated On: October 27th, 2011] [Originally Added On: October 27th, 2011]
- First targeted treatment success for Duchenne muscular dystrophy - Video [Last Updated On: November 6th, 2011] [Originally Added On: November 6th, 2011]
- 125 Days of VECTTOR Treatment Progress [Last Updated On: November 18th, 2011] [Originally Added On: November 18th, 2011]
- Becker Muscular Dystrophy WALKING ABILITY IMPROVED - Video [Last Updated On: November 25th, 2011] [Originally Added On: November 25th, 2011]
- PT Muscular Dystrophy Treatment Results - Video [Last Updated On: December 5th, 2011] [Originally Added On: December 5th, 2011]
- Duchenne Muscular Dystrophy Treated by Cellmedicine - Video [Last Updated On: January 8th, 2012] [Originally Added On: January 8th, 2012]
- muscular DYSTROPHY treatment IN HOMEOPATH.mp4 - Video [Last Updated On: January 28th, 2012] [Originally Added On: January 28th, 2012]
- Giulio's strategy is to cure dystrophy with stem cell treatment - Video [Last Updated On: January 31st, 2012] [Originally Added On: January 31st, 2012]
- Parent Project Muscular Dystrophy is a Featured Nonprofit Selected by Webkinz(TM) Foundation [Last Updated On: February 1st, 2012] [Originally Added On: February 1st, 2012]
- MDA Awards More Than $12 Million in Grants to Advance Neuromuscular Disease Research [Last Updated On: February 1st, 2012] [Originally Added On: February 1st, 2012]
- "For treatment we will have in the future" - Video [Last Updated On: February 2nd, 2012] [Originally Added On: February 2nd, 2012]
- Renowned Pediatric Cardiology Physician-Scientist Linda Cripe Joins Nationwide Children's Hospital [Last Updated On: February 3rd, 2012] [Originally Added On: February 3rd, 2012]
- JumpStart Invests $250,000 in Milo Biotechnology [Last Updated On: February 14th, 2012] [Originally Added On: February 14th, 2012]
- When nerve meets muscle, biglycan seals the deal [Last Updated On: February 14th, 2012] [Originally Added On: February 14th, 2012]
- Medical clinics offer help for Big Island children [Last Updated On: February 16th, 2012] [Originally Added On: February 16th, 2012]
- Dateline Long Beach: The Aquatic Center brings swim therapy to disabled [Last Updated On: February 19th, 2012] [Originally Added On: February 19th, 2012]
- Parent Project Muscular Dystrophy Awards $500,000 to Tivorsan Pharmaceuticals [Last Updated On: February 21st, 2012] [Originally Added On: February 21st, 2012]
- Cure Duchenne Announces Three New Funded Research Projects to Help Develop Treatments and Find a Cure for Duchenne ... [Last Updated On: February 21st, 2012] [Originally Added On: February 21st, 2012]
- Ligand Licenses DARA Program to Retrophin [Last Updated On: February 21st, 2012] [Originally Added On: February 21st, 2012]
- AVI BioPharma Regains NASDAQ Compliance [Last Updated On: February 23rd, 2012] [Originally Added On: February 23rd, 2012]
- Scientists create potent molecules aimed at treating muscular dystrophy [Last Updated On: February 23rd, 2012] [Originally Added On: February 23rd, 2012]
- AVI BioPharma Announces Fourth Quarter and Full Year 2011 Financial Results and Corporate Update Conference Call [Last Updated On: February 23rd, 2012] [Originally Added On: February 23rd, 2012]
- Pembroke's Christine McSherry is an 'Inspirational Woman' [Last Updated On: February 25th, 2012] [Originally Added On: February 25th, 2012]
- The Rare Clinical Diseases Research Network [Last Updated On: February 25th, 2012] [Originally Added On: February 25th, 2012]
- A Solution for Duchenne Muscular Dystrophy? - Research Summary [Last Updated On: February 28th, 2012] [Originally Added On: February 28th, 2012]
- A Solution for Duchenne Muscular Dystrophy? -- In Depth Doctor's Interview [Last Updated On: February 28th, 2012] [Originally Added On: February 28th, 2012]
- Antisense oligonucleotides make sense in myotonic dystrophy [Last Updated On: February 28th, 2012] [Originally Added On: February 28th, 2012]
- A life of dependence [Last Updated On: February 29th, 2012] [Originally Added On: February 29th, 2012]
- Nationwide Children's Hospital neuromuscular disorder podcasts now available on iTunes [Last Updated On: March 2nd, 2012] [Originally Added On: March 2nd, 2012]
- Next-generation DNA sequencing to improve diagnosis for muscular dystrophy [Last Updated On: March 6th, 2012] [Originally Added On: March 6th, 2012]
- The Dire Limits of Health Care [Last Updated On: March 7th, 2012] [Originally Added On: March 7th, 2012]
- AVI BioPharma Announces Late-Breaker Oral Presentation of Phase IIb DMD Study at 2012 AAN Annual Meeting in April [Last Updated On: March 12th, 2012] [Originally Added On: March 12th, 2012]
- Cataracts affect millions of people around the globe, and for many of us they will be a normal part of our aging ... [Last Updated On: March 14th, 2012] [Originally Added On: March 14th, 2012]
- Your Health: Duchenne's Muscular Dystrophy [Last Updated On: March 16th, 2012] [Originally Added On: March 16th, 2012]
- Newborn Screening for Duchenne Muscular Dystrophy Shows Promise as an International Model [Last Updated On: March 20th, 2012] [Originally Added On: March 20th, 2012]
- Newborn screening for DMD shows promise as an international model [Last Updated On: March 20th, 2012] [Originally Added On: March 20th, 2012]
- Rhenovia launches drug discovery for Huntington's disease [Last Updated On: March 20th, 2012] [Originally Added On: March 20th, 2012]
- Halo Therapeutics Reports Favorable Independent Review of Lead Drug Candidate HT-100 [Last Updated On: March 21st, 2012] [Originally Added On: March 21st, 2012]
- Invasive treatment strategy may increase survival for patients with certain neuromuscular disorder [Last Updated On: March 28th, 2012] [Originally Added On: March 28th, 2012]
- Parent Project Muscular Dystrophy Endorses FAST Act Legislation to Expedite FDA Review of Life-Saving Therapies [Last Updated On: March 29th, 2012] [Originally Added On: March 29th, 2012]
- AVI BioPharma Announces Conference Call and Webcast on Monday, April 2, 2012, to Discuss Top-Line Data Results From ... [Last Updated On: March 31st, 2012] [Originally Added On: March 31st, 2012]
- Drug for rare disease may lift AVI BioPharma shares: Barron's [Last Updated On: April 2nd, 2012] [Originally Added On: April 2nd, 2012]
- AVI BioPharma Announces Eteplirsen Meets Primary Endpoint, Demonstrating a Significant Increase in Dystrophin at 24 ... [Last Updated On: April 2nd, 2012] [Originally Added On: April 2nd, 2012]
- U.S. Stock Futures Little Changed Before Factory Report [Last Updated On: April 2nd, 2012] [Originally Added On: April 2nd, 2012]
- Leading experts on congenital muscular dystrophy convene at University of Nevada, Reno [Last Updated On: April 20th, 2012] [Originally Added On: April 20th, 2012]
- Nature Publishes Work Utilizing N-Gene's Core Technology to Advance the Treatment of Duchenne Muscular Dystrophy [Last Updated On: April 20th, 2012] [Originally Added On: April 20th, 2012]
- Getting the boots filled [Last Updated On: April 29th, 2012] [Originally Added On: April 29th, 2012]
- Local business, civic leaders 'arrested' for MDA fundraiser [Last Updated On: May 3rd, 2012] [Originally Added On: May 3rd, 2012]