Merck Announces FDA Orphan Drug Designation for Bifunctional Immunotherapy M7824 in Biliary Tract Cancer - Seite 2

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FDA Orphan Drug Designation (ODD) is granted to medicines intended
to treat rare diseases or disorders that affect fewer than 200,000
people in the US, or those that affect more than 200,000 people but
are unlikely to recover the costs of developing and marketing the
drug. Medicines that meet the FDA's ODD criteria qualify for a number
of incentives to help support advancement.

M7824 is an investigational bifunctional immunotherapy that
combines a TGF-? trap with the anti-PD-L1 mechanism in one fusion
protein. Designed to combine co-localized blocking of the two
immunosuppressive pathways, M7824 is thought to control tumor growth
by potentially restoring and enhancing anti-tumor responses. M7824 is
an important part of a novel combination approach that seeks to
harness the power of the immune system and address the tremendously
complex nature of difficult-to-treat tumors. To-date, more than 670
patients with various types of solid tumors have been treated across
the program with M7824. In addition to BTC, M7824 is being studied in
solid tumor indications, including non-small cell lung, HPV
associated tumors and gastrointestinal cancers, such as gastric
cancer, esophageal squamous cell carcinoma and esophageal
adenocarcinoma.

About M7824

M7824 is an investigational bifunctional immunotherapy that is
designed to combine a TGF-? trap with the anti-PD-L1 mechanism in one
fusion protein. M7824 is designed to combine co-localized blocking of
the two immunosuppressive pathways - targeting both pathways aims to
control tumor growth by potentially restoring and enhancing
anti-tumor responses. M7824 is currently in Phase I studies for solid
tumors, as well as a trial to investigate M7824 compared with
pembrolizumab as a first-line treatment in patients with PD-L1
expressing advanced NSCLC. The multicenter, randomized, open-label,
controlled study is evaluating the safety and efficacy of M7824
versus pembrolizumab as a monotherapy treatment.

About the FDA Orphan Designation

FDA orphan drug designation is granted to drugs intended to treat
rare diseases or disorders that affect fewer than 200,000 people in
the US, or those that affect more than 200,000 people, but are
unlikely to recover the costs of developing and marketing the drug.
Orphan drug designation by the FDA qualifies the sponsor for
incentives provided for in the Orphan Drug Act, which can include
protocol assistance for clinical trials, prescription drug user fee
waivers, tax incentives and seven years of market exclusivity. The
granting of an orphan drug designation does not alter the standard