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aTyr Pharma Rises 16% on Orphan Drug Status

aTyr Pharma Rises 16% on Orphan Drug Status

aTyr Pharma Inc. (LIFE) said its Resolaris drug secured Orphan Drug Designation from the U.S. Food And Drug Administration (FDA) for treating limb girdle muscular dystrophy (LGMD).

An orphan drug is defined as a product used to treat or prevent rare diseases that affect fewer than 200,000 people in the United States, or that affect more than 200,000 people but are not expected to recover the costs of developing and marketing a drug to treat the condition.

Orphan drug status announcement is often accompanied by a spike in a company’s stock price, as the status allows the drug maker to enjoy certain privileges, including seven years of exclusive rights to the marketplace even if the drug patent expires, and liberty to set and raise drug prices as it wishes. The exclusivity is intended to offer compensation to rare disease drug makers, as fewer patients won’t lead to high-volume sales. (See also, Many Drug Makers Are Abusing Orphan Drug Rules.)

Following the announcement, aTyr stock spiked by around 28% on Tuesday, to touch an intraday high of $4.45 per share, before closing at $4 a share, up 16% compared to the previous day’s close.

Resolaris Success Run Continues

This is another milestone for aTyr’s Resolaris drug, which secured Fast Track designation by the FDA last month for the treatment of limb girdle muscular dystrophy 2B (LGMD2B) or dysferlinopathy, a sub-type autosomal recessive form of LGMD. The FDA also lifted a partial clinical ban last month on a dosing ceiling for Resolaris. (For more, see Atyr Pharma Spikes 33% on FDA Update.)

The drug also secured Fast Track designation in October 2016 for treating facioscapulohumeral muscular dystrophy. (See also, aTyr Pharma Receives Fast Track Designation.)

Resolaris is being developed as an intravenous protein-based therapy by the San Diego, California-based company for treating rare myopathies with an immune component. The drug is extracted from a naturally occurring protein which is released by human skeletal muscle cells.

Myopathy is a neuromuscular disorder which leads to muscle weakness, and is caused due to dysfunction of muscle fibers. It can either be inherited or acquired. Limb girdle muscular dystrophy (LGMD) refers to a group of rare genetic myopathies which are known to have more than 20 different subtypes, including LGMD2B. None of the LGMD disorders have any approved treatments, and are estimated to impact around 16,000 patients in the U.S.

LGMD2B is a recessive genetic disease caused by a toxic loss of function in the dysferlin gene, which impacts 3,000 U.S. residents.

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