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BioPharma Global, a division of Pace Life Sciences — Rare Disease Pipeline | Kisho

BioPharma Global, a division of Pace Life Sciences

Other

Orphan Designations

0 approved, 3 designated

FDA Approvals

Active Trials

200 recruiting

Rare Diseases

across 12 areas

News (30d)

Quiet

BioPharma Global, a division of Pace Life Sciences is a company with 3 orphan drug designations across 5 rare diseases.

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: 5

5 total diseases

Disease	Drug(s)	Stage
alpha thalassemia-X-linked intellectual disability syndrome	Cutamesine dihydrochloride	Des.TrialAppr.
alpha-thalassemia-myelodysplastic syndrome	Cutamesine dihydrochloride	Des.TrialAppr.
amyotrophic lateral sclerosis	Fasudil hydrochloride	Des.TrialAppr.
beta-thalassemia-X-linked thrombocytopenia syndrome	Cutamesine dihydrochloride	Des.TrialAppr.
cerebellar ataxia	Rovatirelin	Des.TrialAppr.