Update for May 17, 2023

Regulatory Update

The FDA approved pegunigalsidase alpha (Elfabrio, Chiesi, Protalix BioTherapeutic), on 5/10/2023, for the treatment of adult patients with Fabry disease. Pegunigalsidasealpha was approved with a boxed waring regarding hypersensitivity reactions, including anaphylaxis.


The FDA approved fezolinetant (Veozah,Astellas Pharma), on 5/12/2023, to treat moderate to severe vasomotor symptoms caused by menopause. Astellas priced fezolinetant a t$6,600 per year, which is higher than the $2,000 to $2,500 range recommended byICER.


The FDA rejected N-803 plus BacillusCalmette-Guerin (BCG) for the treatment of BCG-unresponsive non-muscle invasive bladder carcinoma due to manufacturing deficiencies.


The FDA rejected trastuzumab duocarmazine for the treatment of pretreated HER2-positive unresectable locally advanced or metastatic breast cancer and requested additional unspecified information.


An FDA review of delandistrogene moxeparvovec questioned whether microdystrophin protein expressionis a valid surrogate endpoint that can be used for approval, since microdystrophin has not demonstrated a pharmacologic effect on a biomarker in the pathway of the disease. The FDA failed to find a benefit for ambulatory DMD patients in a review of delandistrogene moxeparvovectrials. The FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) voted 8 to 6 to recommend approval of delandistrogene moxeparvovec.


The FDA designated ampreloxetine an Orphan Drug for the treatment of symptomatic neurogenic orthostatic hypotension in patients with multiple system atrophy.