The Orphan Drug Designation program provides orphan status to drugs and biologics which are defined as those intended for the treatment, prevention or diagnosis of a rare disease or condition, which is one that affects less than 200,000 persons in the US or meets cost recovery provisions of the act.

Facts about Rare Disease

•  More than 7,000 conditions are considered rare diseases
• 1 in 100 people is affected by a rare disease
• 350 million people worldwide are living with a rare disease, but only 5% of known rare diseases have an approved treatment
• About 30 million people in the US are affected by a rare disease
• Half of the people with rare diseases are children.
• Rare Disease Day was started in 2008 and is now observed in 65 counties

Company

Drug Name

Indication

ORPHELIA Pharma	Temozolomide	Neuroblastoma
Minoryx Therapeutics S.L.	Leriglitazone	Friedreich´S Ataxia
Idorsia Pharmaceuticals Ltd	ACT-519276	GM2 Gangliosidosis
Stoke Therapeutics	Antisense Oligonucleotide	Dravet Syndrome
Daewoong Pharmaceutical	DWN12088	Idiopathic Pulmonary Fibrosis
Dynacure SAS	Dyn101	Centronuclear Myopathies
Vitaeris (USA) Corp	Clazakizumab	Chronic Antibody-Mediated Rejection in Kidney Transplant Recipients
Partner Therapeutics, Inc.	Sargramostim	Stage IIB-IV Melanoma
Blueprint Medicines	BLU-782	Fibrodysplasia Ossificans Progressiva
Neuropore Therapies, Inc.	NPT520-34	Amyotrophic Lateral Sclerosis
Insys Therapeutics Inc.	Cannabidiol	Childhood Absence Epilepsy
Inmunova SA	Neutralizing equine anti-Stx hyperimmune immunoglobulin F(ab')2 fragment	Shiga-Toxin Producing Bacterial Infection
Eagle Biosciences, Inc.	DNA based therapy	Osteogenesis Imperfecta
Ocugen, Inc.	AAV-hNR2E3	CEP290 Mutation Associated Retinal Disease
Microbion Corporation	MBN-101	Pulmonary Infections in Patients with Cystic Fibrosis
Sanofi	Romilkimab	Systemic Sclerosis
Incyte Corporation	Pemigatinib	Myeloid/Lymphoid Neoplasms with Eosinophilia and Rearrangement Of PDGFRA, PDGFRB, or FGFR1, or With PCM1-JAK2
Astex Pharmaceuticals, Inc.	Cedazuridine and Decitabine	Myelodysplastic Syndromes (Including Chronic Myelomonocytic Leukemia)
BioXcel Therapeutics	Talabostat	Acute Myelogenous Leukemia
Sigilon Therapeutics, Inc.	SIG-001	Hemophilia A
Regeneron Pharmaceuticals	Dupilumab	Bullous Pemphigoid
Amgen Inc.	Small molecule inhibitor of the pro-survival protein MCL1	Multiple Myeloma
Recordati Rare Diseases, SARL	Propranolol	Retinopathy of Prematurity
Allianz Pharmascience Ltd.	ALZ003	Glioblastoma
Adaptimmune, LLC	SPEARHED-1	Soft Tissue Sarcoma
AstraZeneca Pharmaceuticals	Benralizumab	Eosinophilic Esophagitis
AI Therapeutics, Inc.	Sirolimus	Tuberous Sclerosis Complex
CrystalGenomics, Inc.	CG-745	Pancreatic Cancer
Puma Biotechnology Inc.	Neratinib	Breast Cancer Patients With Brain Metastases
Ra Pharmaceuticals, Inc.	Zilucoplan	Myasthenia Gravis
Celgene	B-cell maturation antigen (BCMA) x CD3 T-cell bispecific monoclonal antibody	Multiple Myeloma
Akari Therapeutics Plc	Nomacopan	Hematopoietic Stem Cell Transplantation-Associated Thrombotic Microangiopathy
CARsgen Therapeutics	Fully Human Anti-BCMA Autologous CART Cell	Multiple Myeloma
GeneTx Biotherapeutics, LLC	GTX-102	Angelman Syndrome

AB Science

Masitinib

Acute Myeloid Leukemia

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