NEW YORK--(BUSINESS WIRE)--Kadmon Holdings, Inc. (NYSE:KDMN) today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to tesevatinib, the Company’s oral tyrosine kinase inhibitor, for the treatment of non-small cell lung cancer (NSCLC) with epithelial growth factor receptor (EGFR)-activating mutations. This is the second orphan drug designation for tesevatinib; the first designation was granted in March 2016 for the treatment of autosomal recessive polycystic kidney disease (ARPKD).
The FDA grants orphan designation to promote the development of promising products for rare conditions affecting fewer than 200,000 U.S. patients annually. It qualifies a company for certain financial benefits, including seven years of market exclusivity following marketing approval, tax credits for clinical research costs, eligibility for Orphan Product grants and the waiver of certain administrative fees.
Kadmon is conducting ongoing Phase 2 clinical trials of tesevatinib for the treatment of EGFR-mutation-positive NSCLC that has metastasized to the brain and/or the leptomeninges (membranes lining the brain and spinal cord) and for the treatment of glioblastoma. The Company is also developing tesevatinib for the treatment of autosomal dominant polycystic kidney disease (ADPKD) and ARPKD and expects to initiate clinical trials in these indications in Q3 2017.
Kadmon Holdings, Inc. is a fully integrated biopharmaceutical company developing innovative products for significant unmet medical needs. We have a diversified product pipeline focused on autoimmune and fibrotic diseases.
Forward Looking Statements
This press release contains forward-looking statements. Such statements may be preceded by the words “may,” “will,” “should,” “expects,” “plans,” “anticipates,” “could,” “intends,” “targets,” “projects,” “contemplates,” “believes,” “estimates,” “predicts,” “potential” or “continue” or the negative of these terms or other similar expressions. Forward-looking statements involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. We believe that these factors include, but are not limited to, (i) the initiation, timing, progress and results of our preclinical studies and clinical trials, and our research and development programs; (ii) our ability to advance product candidates into, and successfully complete, clinical trials; (iii) our reliance on the success of our product candidates; (iv) the timing or likelihood of regulatory filings and approvals; (v) our ability to expand our sales and marketing capabilities; (vi) the commercialization of our product candidates, if approved; (vii) the pricing and reimbursement of our product candidates, if approved; (viii) the implementation of our business model, strategic plans for our business, product candidates and technology; (ix) the scope of protection we are able to establish and maintain for intellectual property rights covering our product candidates and technology; (x) our ability to operate our business without infringing the intellectual property rights and proprietary technology of third parties; (xi) costs associated with defending intellectual property infringement, product liability and other claims; (xii) regulatory developments in the United States, Europe and other jurisdictions; (xiii) estimates of our expenses, future revenues, capital requirements and our needs for additional financing; (xiv) the potential benefits of strategic collaboration agreements and our ability to enter into strategic arrangements; (xv) our ability to maintain and establish collaborations or obtain additional grant funding; (xvi) the rate and degree of market acceptance of our product candidates; (xvii) developments relating to our competitors and our industry, including competing therapies; (xviii) our ability to effectively manage our anticipated growth; (xix) our ability to attract and retain qualified employees and key personnel; (xx) our ability to achieve cost savings and other benefits from our efforts to streamline our operations and to not harm our business with such efforts; and (xxi) the use of proceeds from our recent private placement. More detailed information about Kadmon and the risk factors that may affect the realization of forward-looking statements is set forth in the Company's filings with the U.S. Securities and Exchange Commission (SEC), including the Company's Quarterly Report on Form 10-Q filed pursuant to Section 13 of the Securities Exchange Act of 1934, as amended, with the SEC on August 3, 2017. Investors and security holders are urged to read these documents free of charge on the SEC's web site at www.sec.gov. The Company assumes no obligation to publicly update or revise its forward-looking statements as a result of new information, future events or otherwise.