La Jolla Pharmaceutical Company Receives Orphan Drug Designation for Two Novel Compounds for Fibrodysplasia Ossificans Progressiva

SAN DIEGO–(BUSINESS WIRE)– La Jolla Pharmaceutical Company(Nasdaq: LJPC) (the Company or La Jolla), a leader in the development of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases, today announced that the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development has granted La Jolla orphan drug designation for two novel compounds for fibrodysplasia ossificans progressiva (FOP).

The compounds that received 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, ALK2. La Jolla recently entered a worldwide, exclusive license agreement with Vanderbilt University covering this technology. The seven members of the BMP type-I receptor family, activin receptor-like kinase (ALK) 1-7, play critical roles in human development and physiology. In turn, the improper activation of these receptor pathways is responsible for a wide range of disease conditions.

FOP is a rare genetic disorder where the body turns muscle into bone. FOP is caused by a genetic mutation in ALK2 that results in excessive signaling of this pathway. 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 40 years, and death often results from complications of thoracic insufficiency syndrome, which is the inability of the thorax to support normal respiration.

“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,” said George F. Tidmarsh, M.D., Ph.D., President and Chief Executive Officer of La Jolla. “We look forward to further collaborating with our colleagues at Vanderbilt University to find a potential treatment for these patients in need.”

About Orphan Drug Designation

Orphan drug designation is granted by the FDA Office of Orphan Products Development to novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. The designation provides the drug developer with a seven-year period of U.S. marketing exclusivity if the drug is the first of its type approved for the specified indication or if it demonstrates superior safety, efficacy, or a major contribution to patient care versus another drug of its type previously granted the designation for the same indication. The designation also provides the drug developer with tax credits for clinical research costs, the ability to apply for annual grant funding, clinical research trial design assistance and a waiver of Prescription Drug User Fee Act (PDUFA) filing fees.

About La Jolla Pharmaceutical Company

La Jolla Pharmaceutical Company is a biopharmaceutical company focused on the discovery, development and commercialization of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases. The Company has several product candidates in development. LJPC-501 is La Jolla’s proprietary formulation of angiotensin II for the potential treatment of catecholamine-resistant hypotension. LJPC-401 is La Jolla’s novel formulation of hepcidin for the potential treatment of iron overload, which occurs as a result of diseases such as hereditary hemochromatosis and beta thalassemia. LJPC-30Sa and LJPC-30Sb are La Jolla’s next-generation gentamicin derivatives for the potential treatment of serious bacterial infections and rare genetic disorders, such as cystic fibrosis and Duchenne muscular dystrophy. For more information on La Jolla, 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 the Company’s 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 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 timing for the filing of an Investigational New Drug Application (IND), commencement of clinical studies and the anticipated timing for completion of such studies; the success of future development activities for the Company’s drug candidates; potential indications for which the Company’s drug candidates may be developed; and the Company’s ability to obtain the potential benefits of orphan drug designation for its drug candidates. 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.


La Jolla Pharmaceutical Company
George F. Tidmarsh, M.D., Ph.D.
President & Chief Executive Officer
Dennis M. Mulroy
Chief Financial Officer


Source: La Jolla Pharmaceutical Company