FDA Grants Orphan Drug Designation to Kadmon’s Tesevatinib for the Treatment of EGFR-Mutated Non-Small Cell Lung Cancer

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.

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business model, strategic plans for our business, product candidates and
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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.
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forward-looking statements as a result of new information, future events
or otherwise.

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