SOURCE: La Jolla Pharmaceutical Company

La Jolla Pharmaceutical Company

April 18, 2013 09:41 ET

La Jolla Pharmaceutical Company Receives Orphan Designation From FDA for LJPC-6417

SAN DIEGO, CA--(Marketwired - Apr 18, 2013) - La Jolla Pharmaceutical Company (OTCQB: LJPC) ("La Jolla"), a leader in the development of therapeutics targeting galectin proteins, announced today that the FDA Office of Orphan Products Development has granted an "Orphan Drug" designation to La Jolla's drug candidate LJPC-6417 for fibrodysplasia ossificans progressiva ("FOP").

"We are pleased that the FDA has granted an Orphan Drug designation to LJPC-6417," said George Tidmarsh, M.D. Ph.D., President and Chief Executive Officer of La Jolla Pharmaceutical Company. "FOP causes progressive debilitation eventually leading to early death through the replacement of skeletal muscle and soft connective tissues with bone. There are no known treatments, and we are excited about the potential of LJPC-6417 to help long suffering FOP patients and treat other forms of heterotopic ossification."

LJPC-6417 is a kinase inhibitor targeting the bone morphogenetic protein (BMP) type-1 receptor, which is mutated in FOP. FOP is a progressive and debilitating genetic disorder characterized by heterotopic ossification. The defining sign present at birth is a malformation of the great toes. In early childhood, afflicted 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. The median survival of FOP patients is approximately 45 years, and death often results from complications of thoracic insufficiency syndrome.

FOP is an extremely rare condition affecting 1 in 2,000,000 people. According to the International Fibrodysplasia Ossificans Progressiva Association, there are 185 individuals with FOP in the United States. In addition to FOP, over activity of the BMP receptors causes heterotopic ossification as a result of trauma. In particular, soldiers who suffer combat injuries are prone to heterotopic ossification, a severely debilitating complication that is increasing significantly in prevalence. It is estimated that 64% of soldiers with blast injuries to the extremities develop heterotypic ossification, compromising functional mobility and prosthesis use. 

About La Jolla Pharmaceutical Company

La Jolla Pharmaceutical Company is a biopharmaceutical company dedicated to the development of medical treatments that significantly improve outcomes in patients with life-threatening diseases. GCS-100, the Company's lead product candidate, is a first-in-class inhibitor of galectin-3, a novel molecular target implicated in chronic organ failure and cancer. For more information on the Company please visit

Forward Looking Statement Safe Harbor

This document contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements relate to future events or our future results of operations. These statements are only predictions and involve known and unknown risks, uncertainties and other factors, which may cause actual results to be materially different from these forward-looking statements. The Company cautions readers not to place undue reliance on any such forward-looking statements, which speak only as of the date they were made. Certain of these risks, uncertainties, and other factors are described in greater detail in the Company's filings from time to time with the U.S. Securities and Exchange Commission (SEC), all of which are available free of charge on the SEC's web site These risks include, but are not limited to, risks relating to the development of GCS-100, the success and timing of future preclinical and clinical studies of this compound, and potential indications for which GCS-100 may be developed. Subsequent written and oral forward-looking statements attributable to the Company or to persons acting on its behalf are expressly qualified in their entirety by the cautionary statements set forth in the Company's reports filed with the SEC. The Company expressly disclaims any intent to update any forward-looking statements.

Contact Information

  • Company Contact

    George F. Tidmarsh, M.D., Ph.D.
    President & Chief Executive Officer
    La Jolla Pharmaceutical Company
    Phone: (858) 207-4264


    Chester S. Zygmont, III
    Director of Finance
    La Jolla Pharmaceutical Company
    Phone: (858) 207-4262