News Updates
Tracking Rare Disease Drug Development Challenges Amid a Shifting Policy Environment
Rare diseases are defined as those which affect fewer than 200,000 people in the United States. Despite advancements in rare disease research and development due to incentives created in the Orphan Drug Act more than 40 years ago, many rare diseases still lack effective treatments due to the complex science and limited market incentives involved in their development. Of the 10,000+ known rare diseases, just 5% have an FDA-approved treatment. Incubate's Rare Disease Investment Tracker measures the impacts of a challenging policy environment on rare disease R&D and notes where passage of policies like the ORPHAN Cures Act have had a positive impact.
Mahzi Therapeutics
Orphan Drug Designation
treatment of Pitt Hopkins syndrome
OcuNeo Therapeutics Inc.
Orphan Drug Designation
Sioux Falls
1-50 employees
treatment of vernal keratoconjunctivitis
GENFIT
Orphan Drug Designation
treatment of acute-on-chronic liver failure
Aminex Therapeutics, Inc.
Orphan Drug Designation
Kenmore
1-50 employees
prevention of allograft rejection in liver transplantation
Aminex Therapeutics, Inc.
Orphan Drug Designation
Kenmore
1-50 employees
treatment of malignant glioma
Multitude Therapeutics Inc.
Orphan Drug Designation
Shanghai
1-50 employees
treatment of malignant glioma
Multitude Therapeutics Inc.
Orphan Drug Designation
Shanghai
1-50 employees
treatment of soft tissue sarcoma
RNR Biomedical inc
Orphan Drug Designation
Tenafly
1-50 employees
treatment of Behcet disease
Shanghai Allovanta Biotechnology Co., Ltd.
Orphan Drug Designation
Shanghai
1-50 employees
treatment of follicular lymphoma
Takeda Development Center Americas, Inc.
Orphan Drug Designation
Cambridge
1-50 employees
treatment of narcolepsy type 1