San Diego-based biotech company aTyr Pharma announced today its first clinical trial in FSHD patients of its investigational new drug Resolaris™. The Phase 1b/2 study is a double-blind, placebo-controlled, multiple ascending dose trial in up to 44 FSHD patients at multiple sites in the European Union. The exploratory trial is designed to evaluate safety, tolerability, pharmacokinetics and the biological activity of Resolaris™ in adult patients with FSHD.
Resolaris™ is a first-in-class intravenous protein therapeutic derived from a naturally occurring protein released in vitro by human skeletal muscle cells. aTyr believes Resolaris™ will provide therapeutic benefit to patients with rare myopathies with an immune component characterized by excessive immune cell involvement.
Read more about the trial here. UPDATE: aTyr Pharma Receives EMA Orphan Drug Designation for Resolaris™ in FSHD.
The FSH Society collaborated with aTyr on recruiting volunteers for a blood biomarker study. You can learn more about that here.
aTyr pharma say they recieved orphan drug designation for that medicine, but I couldn’t find anything on the EMA site (European Medicines Agency).
Could you give a link to verify this information ?
Thank you in advance !
Sylvie
We’ll check and get back to you with an answer.
Here’s further information from Frederic Chereau, President & COO of aTyr Pharma:
“The European Commission validated the ODD decision on February 12, 2015 and we got notify by a letter that day. The EMA website info has not yet been updated to include Resolaris’ ODD. The EMA’s search area for approved ODD is below, but I also confirmed our product is not yet listed… Our ODD should be on the EMA website by next month.”
http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/landing/orphan_search.jsp&mid=WC0b01ac058001d12b
Thank you for your answer !
I was wondering also about the link between FSHD and immune response.
I didn’t know that the immune sytem was involved in FSHD.
You’re right that there has not been much published about immune response in FSHD, but there are a few intriguing papers (see below). There are also reports (unpublished) from MRI studies indicating edema as a precursor of muscle degeneration.
Geng LN1, Yao Z, Snider L, Fong AP, Cech JN, Young JM, van der Maarel SM, Ruzzo WL, Gentleman RC, Tawil R, Tapscott SJ.. DUX4 activates germline genes, retroelements, and immune mediators: implications for facioscapulohumeraldystrophy. Dev Cell. 2012 Jan 17;22(1):38-51. doi: 10.1016/j.devcel.2011.11.013. Epub 2011 Dec 29.
Spuler S1, Engel AG. Unexpected sarcolemmal complement membrane attack complex deposits on nonnecrotic muscle fibers in muscular dystrophies. Neurology. 1998 Jan;50(1):41-6.
Arahata K1, Ishihara T, Fukunaga H, Orimo S, Lee JH, Goto K, Nonaka I. Inflammatory response in facioscapulohumeral muscular dystrophy (FSHD): immunocytochemical and genetic analyses. Muscle Nerve Suppl. 1995;(2):S56-66.
Thank you very much ! I will occupy my week-end reading all this 😉
Can you tell me how to be included in trial I have a 30 yr old son who has fasioscalpular muscular dystrophy who has had shoulder fusion in his 20 s but now has severe muscle weakness in his elbows thanks
Check clinicaltrials.gov for current information on the aTyr trials:
https://clinicaltrials.gov/ct2/results?term=aTyr&Search=Search