aTyr Pharma Inc. (NASDAQ: LIFE) watched its shares climb early on Monday after the company received a key U.S. Food and Drug Administration (FDA) approval. The company announced that its product candidate Resolaris was granted Fast Track designation by the FDA for the treatment of facioscapulohumeral muscular dystrophy (FSHD), making it the first known therapeutic candidate for the treatment of FSHD to receive the designation.
Resolaris, a designated Orphan Drug in FSHD, is currently being studied in a Phase 1b/2 clinical program for three types of rare myopathies with an immune component. The company expects to report data from the three clinical trials in December 2016.
The primary objectives of these trials are to establish a safety and tolerability database and to explore and establish activity signals, such as various endpoints and biomarkers, which will best inform aTyr’s clinical development path forward, including endpoints for a Biologic License Application (BLA).
Fast Track is a process designed by the FDA to facilitate the development and expedite the review of new drugs, biologics or treatments. Importantly, the therapy’s sponsors are eligible for more frequent written communication and meetings with the FDA, the benefit of which may be to develop a pivotal study design that more closely meets the FDA’s criteria, thereby creating a more collaborative and efficient pathway to approval.
CEO John Mendlein, PhD, of aTyr Pharma, commented:
This Fast Track designation, which is granted to drug candidates addressing serious conditions and that demonstrate the potential to address unmet medical needs, represents another step forward for our rare muscle disease franchise. This designation highlights the significant need to develop and ultimately approve meaningful new therapeutics to treat patients with rare myopathies, such as FSHD.
Shares of aTyr traded Monday morning at $3.05, with a consensus analyst price target of $6.67 and a 52-week trading range of $2.48 to $13.26.