treatment of idiopathic pulmonary fibrosis
All entries for: Rare Diseases
August 31, 2026
Telomere Theraperutics SL
Orphan Drug Designation
Madrid
1-50 employees
Disease Area: Rare Diseases
August 30, 2026
AstraZeneca Pharmaceuticals LP
Orphan Drug Designation
treatment of esophageal cancer
Disease Area: Rare Diseases
August 27, 2026
Biomed Valley Discoveries Inc.
Orphan Drug Designation
Kansas City
1-50 employees
treatment of malignant glioma
Disease Area: Rare Diseases
August 27, 2026
Kalevala Therapeutics, Inc.
Orphan Drug Designation
Dover
1-50 employees
treatment of mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes (MELAS)
Disease Area: Rare Diseases
August 27, 2026
Mirkine Pharma, Incorporated
Orphan Drug Designation
New York
1-50 employees
treatment of pancreatic cancer
Disease Area: Rare Diseases
August 26, 2026
RayzeBio, Inc.
Orphan Drug Designation
San Diego
1-50 employees
treatment of gastroenteropancreatic neuroendocrine tumors
Disease Area: Rare Diseases
August 26, 2026
X4 Pharmaceuticals, Inc.
Orphan Drug Designation
Boston
1-50 employees
treatment of congenital neutropenia
Disease Area: Rare Diseases
August 25, 2026
Kedrion S.p.A
Orphan Drug Designation
treatment of stiff person syndrome
Disease Area: Rare Diseases
August 24, 2026
SillaJen Inc.
Orphan Drug Designation
Seoul
1-50 employees
treatment of acute myeloid leukemia
Disease Area: Rare Diseases
August 24, 2026
LILA Holding AG
Orphan Drug Designation
Kusnacht
1-50 employees
treatment of Gaucher disease
Disease Area: Rare Diseases