Update for September 9, 2022

The FDA approved spesolimab (Spevigo, Boehringer Ingelheim), on 9/1/2022, for the treatment of generalized pustular psoriasis flares.
 
The FDA approved daxibotulinumtoxin (Daxxify, Revance Therapeutics), on 9/8/2022, for the temporary improvement in the appearance of moderate to severe glabellar lines. Daxibotulinumtoxin was approved with a back box warning regarding the potential for the toxin affect to spread beyond the injection site.
 
The FDA’s review of AMX0035 for the March 2022 AdComm meeting and a new review for the September 2022 AdComm meeting both found the NDA did not provide enough evidence to support approval. The second review noted the revised NDA contained the same survival data with a different analysis. The review suggested that the 48-week, 600 patient, Phase III, PHOENIX trial should have data by late 2023 or early 2024 would give a better indication of the affect of AMX0035 on ALS. The FDA’s Peripheral and Central Nervous System Drugs Advisory Committee (PCNSDAC) voted 6-4 to reject AMX0035 in March 2022 and recommended an additional trial to evaluate efficacy. The FDA extended the PDUFA date by three months to 9/29/2022. The PCNSDAC voted 7 to 2 to recommend approval of AMX0035 to treat ALS in September 2022.
 
The FDA accepted the re-submitted BLA for omburtamab for the treatment of pediatric patients with CNS/leptomeningeal metastases from neuroblastoma and set a PDUFA date for 11/30/2022. The FDA’s Oncologic Drugs Advisory Committee will meet on 10/28/2022 to review omburtamab.
 
Ascendis Pharma has submitted an NDA for TransCon PTH to treat hypoparathyroidism.
 
The FDA halted the Phase II MOMENTUM trial, in June 2022, due to a serious occurrence of hypomagnesemia and requested a risk mitigation and safety monitoring plan. The hold was removed in September 2022 after changes to the study protocol.
 
Tarsas submitted an NDA for lotilaner ophthalmic solution for the treatment of Demodex blepharitis.