ORPHAN DRUG DEVELOPMENT PIPELINE-AREAS OF FOCUS
Neuromuscular & Neurodegenerative Disorders:
- Amyotrophic Lateral Sclerosis (ALS)
- Duchenne Muscular Dystrophy (DMD)
- Friedreich's Ataxia (FRDA)
Rare Genetic & Metabolic Disorders:
- Pompe Disease
- MELAS (Mitochondrial Encephalomyopathy)
- Fanconi Anemia
- Beta Thalassemia
Rare Pulmonary & Multisystem Disorders:
- Cystic Fibrosis (CF)
- Systemic Sclerosis (Scleroderma)
Rare Ophthalmologic Disorders:
- Retinitis Pigmentosa (RP)
ORPHAN DRUG PROGRAM LONG TERM VALUE DRIVER
Global Sickle Cell Disease Market - Revenue
- TAM 7.7 million people living with SCD
- globally (WHO) ~100,000 in the U.S.
Large concentrations in
- Sub-Saharan Africа
- India
- Middle East
- Brazil
- EU/UK
The commercially addressable market is concentrated in
- U.S.
- EU5 + UK
- GCC countries
- Select LATAM,
- Limited Asia
Portfolio Allocation Model
Finished Dose Products
- Generics
- Private Label Programs
- Orphan Drugs (Pipeline)
API-Integrated Revenue:
- U.S. Final Processing
- Peptide Strategy
- 503A/503B Partnerships