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Pfizer Ireland Pharmaceuticals — Rare Disease Pipeline | Kisho

Pfizer Ireland Pharmaceuticals

Other

Orphan Designations

1 approved

FDA Approvals

Active Trials

Rare Diseases

across 5 areas

News (30d)

Quiet

Pfizer Ireland Pharmaceuticals is a company with 1 orphan drug designation across 6 rare diseases. gene therapy candidates in 1 disease.

Gene Therapy Active

Competitive Landscape

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: 6

6 total diseases

Disease	Drug(s)	Stage
isolated congenital growth hormone deficiency	Ngenla	Des.TrialAppr.
isolated growth hormone deficiency type IA	Ngenla	Des.TrialAppr.
isolated growth hormone deficiency type IB	Ngenla	Des.TrialAppr.
isolated growth hormone deficiency type II	Ngenla	Des.TrialAppr.
pediatric hepatocellular carcinoma	Ngenla	Des.TrialAppr.
pediatric-onset Graves disease	Ngenla	Des.TrialAppr.