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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