Efgartigimod developed by Argenx is a strong competitor to Soliris.
Rare/Orphan Diseases
Posted inNew Drugs, Rare/Orphan Diseases, Regulatory
Trikafta/Kaftrio: World’s First Triple Therapy for Cystic Fibrosis
The combination drug will help almost all cystic fibrosis patients.
Posted inNew Drugs, Rare/Orphan Diseases, Regulatory
Voxzogo: First Drug to Treat Dwarfism
Vosoritide by BioMarin Pharmaceutical may cure achondroplasia.
Posted inNew Drugs, Oncology, Rare/Orphan Diseases, Regulatory
Fyarro: First Drug to Treat Perivascular Epithelioid Cell Tumor
PEComa is a very rare and aggressive subtype of soft tissue sarcoma.
Posted inClinical Trials, Rare/Orphan Diseases
Vyjuvek: Effective Treatment for Dystrophic Epidermolysis Bullosa
Krystal Biotech’s beremagene geperpavec gene therapy will save from imminent death.
Posted inMetabolism, Neuroscience, Oncology, Pharma & Biotech, Rare/Orphan Diseases
Alltrna: World’s First Drugs Based on Transfer RNAs
A completely new therapeutic modality will help treat both rare and common diseases.
Posted inOncology, Pharma & Biotech, Rare/Orphan Diseases
Aura Biosciences: Anticancer Virus-Like Drug Conjugates
A new class of oncology drugs with a dual mechanism of action.