aTyr Pharma Receives Orphan Drug Designation for Resolaris in FSHD

Industry News

aTyr, a San Diego, CA based biotherapeutics company, was granted Orphan Drug Designation for its Resolaris for the treatment of facioscapulohumeral muscular dystrophy (FSHD).  FSHD is a rare hereditary disorder caused by genetic changes involving the long arm of chromosome 4.  FSHD is a type of muscular dystrophy that initially affects the skeletal muscles of the face, scapula and upper arms, and as the disease progress, about 20% of the patients become severely disabled.  There is no FDA-approved treatment for this disease.  Resolaris is recombinant human histidyl tRNA synthetase and is an intravenous protein therapeutic.  Resolaris received ODD from the European Union earlier this year and is currently in Phase I/II clinical trial in FSHD patients for safety, tolerability, pharmacokinetics and efficacy.

 

Related Links:

http://www.atyrpharma.com/atyrpharma-receivesema-orphandrug-designation-for-resolaris-tm-in-fshd/
http://ghr.nlm.nih.gov/condition/facioscapulohumeral-muscular-dystrophy
http://adisinsight.springer.com/drugs/800041283
https://www.clinicaltrials.gov/ct2/show/NCT02239224?term=facioscapulohumeral+muscular+dystrophy&rank=15&submit_fld_opt=

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