Kisho

SHIONOGI INC. — Rare Disease Pipeline | Kisho

SHIONOGI INC.

Other

Orphan Designations

1 approved, 2 designated

FDA Approvals

Latest: FETROJA (2019)

Active Trials

200 recruiting

Rare Diseases

across 19 areas

News (30d)

Quiet

SHIONOGI INC. is a company with 3 orphan drug designations across 10 rare diseases, including 2 FDA-approved therapies. gene therapy candidates in 1 disease. 1 active policy bill affects their portfolio.

Gene Therapy Active

Diversified Portfolio

Disease-by-disease pipeline progression from orphan designation through clinical trials to FDA approval.

Source: FDA Orphan Drug Designations, ClinicalTrials.gov, Drugs@FDA

Approved: 0

In Trials: 0

Designated Only: 10

10 total diseases

Disease	Drug(s)	Stage
Nezelof syndrome	Doripenem	Des.TrialAppr.
aspartylglucosaminuria	Cuvposa	Des.TrialAppr.
blue rubber bleb nevus	Cuvposa	Des.TrialAppr.
cystic fibrosis	Doripenem	Des.TrialAppr.
developmental and epileptic encephalopathy, 39	Cuvposa	Des.TrialAppr.
glycogen storage disease II	selective inhibitor of GYS1	Des.TrialAppr.
intellectual disability, autosomal dominant 5	Cuvposa	Des.TrialAppr.
microcephaly, epilepsy, and diabetes syndrome 1	Cuvposa	Des.TrialAppr.
osteopetrosis, autosomal recessive 9	Cuvposa	Des.TrialAppr.
trichothiodystrophy	Doripenem	Des.TrialAppr.