Miplyffa (arimoclomol) was approved for Niemann-Pick disease type C when administered with another high-priced drug used off label as standard of care for about 80% of NPC patients. 

New therapies in the wings include two candidates for Niemman-Pick disease type C, a first-in-class antipsychotic, a targeted oncology therapy under Real-Time Oncology Review, and a new drug for gastroparesis.

After a quiet July, August and September are shaping up to be busy on the approvals front. Already this month, the US FDA has approved Adaptimmune’s Teclera for synovial sarcoma, Phathom’s Voquezna for gastro-esophageal reflux disease and Servier’s Voranigo for gliomas. Here, Scrip takes a look at ten other approvals for novel products in the offing for the third quarter.

Half of the FDA’s upcoming 2024 user fee goals target rare diseases, but highly prevalent conditions like uncomplicated urinary tract infections and atopic dermatitis are also up for agency action in the second half of the year.