Orphan Drug Designation For Novel Compounds - Report on La Jolla Pharmaceutical

NEW YORK, September 16, 2015 /PRNewswire/ --

ACI Association has initiated research coverage on La Jolla Pharmaceutical Co. (NASDAQ: LJPC). Select highlights from the internally released reports are being made available to the general public (included below), with access to the entirety of the research available to new members.

Today, membership is open to readers on a complementary basis at the following URL: http://www.aciassociation.com/reports?keyword=LJPC  

Highlights from our LJPC Report include:

• Orphan Drug Designation Granted to Two Novel Compounds - On August 18, 2015, the biopharmaceutical company focused on the discovery, development and commercialization of innovative therapies, La Jolla Pharmaceutical Co. (La Jolla), announced that US Food and Drug Administration (USFDA) Office of Orphan Products Development has granted the orphan drug designation to its two novel compounds for the treatment of fibrodysplasia ossificans progressiva (FOP).

• A Brief Introduction of the Novel Compounds - The novel compounds which have been granted the orphan drug designation are small-molecule kinase inhibitors designed to selectively block a specific member of the bone morphogenetic protein (BMP) type-I receptor family, activin receptor-like kinase (ALK) 2. According to the Company, seven members of the BMP type-I receptor family, ALK 1-7 play critical roles in human development and physiology and any improper activation of these receptor pathways is responsible for a wide range of disease conditions. Additionally, La Jolla recently entered a worldwide, exclusive license agreement with Vanderbilt University covering this technology.

• More on FOP Disease - While providing information about FOP, La Jolla said that FOP is a rare genetic disorder caused by a genetic mutation in ALK2, where the body turns muscle into bone. In early childhood, the affected individuals develop soft tissue swellings that transform into bone. Development of such lesions is exacerbated by trauma, and surgical intervention leads to dramatic and explosive new bone growth. While the average age of such patients is approximately 40 years, death often results from complications of thoracic insufficiency syndrome, which is the inability of the thorax to support normal respiration.

• Management Commentary - President and Chief Executive Officer of La Jolla, George F. Tidmarsh, M.D., Ph.D., said, "There are no known treatment options available for patients suffering from FOP, and the orphan drug designations recognize the significant unmet need that exists within this disease." He further said, "We look forward to further collaborating with our colleagues at Vanderbilt University to find a potential treatment for these patients in need."

To find out how this influences our rating on La Jolla Pharmaceutical Co., read the full report in its entirety here: http://www.aciassociation.com/reports?keyword=LJPC

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