These drugs have interesting mechanisms, unique use, or may support expanded indications in the future.
This approval makes birch triterpenes the first treatment for wounds associated with junctional epidermolysis bullosa to be approved by the FDA.
If approved, V116 would offer significant improvements in safety and effectiveness for the prevention of pneumococcal disease.
Previously, garadacimab was granted an orphan drug designation as a therapy for hereditary angioedema by both the FDA and EMA.
The approval marks the first for an alternative to platinum-containing chemotherapy.
Belzutifan (Welireg; Merck) demonstrated superior progression-free survival compared to everolimus in advanced renal cell carcinoma.
The study will assess the use of the drug to treat impaired social motivation, or asociality, which is a difficult-to-treat symptom of schizophrenia that can cause significant functional impairment.
Officials from the FDA have granted Geron Corporation’s imetelstat fast track designation for adult patients with intermediate-2 or high-risk myelofibrosis (MF) whose disease has relapsed after or is refractory to janus kinase (JAK) inhibitor treatment.
