Update for December 22, 2022

​Regulatory Update
 
The FDA approved nadofaragene firadenovec (Adstiladrin, Ferring Pharmaceuticals), on 12/16/2022, for the treatment of high-risk, Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer with carcinoma in situ with or without papillary tumors.
 
The FDA accepted the NDA for olorofim for the treatment of invasive fungal infections and set a PDUFA date for 6/17/2023.
 
The EU approved tabelecleucel (Ebvallo, Altara Biotherapeutics) for the treatment of relapsed or refractory Epstein‑Barr virus positive post‑transplant lymphoproliferative disease.
 
CHMP recommend the EMA approve:

• Etranacogene dezaparvovec for the treatment of severe and moderately severe Haemophilia B
• Tremelimumab in combination with durvalumab for the treatment of hepatocellular carcinoma and metastatic non-small-cell lung cancer
• Cipaglucosidase alfa for the treatment of Pompe disease

 
In December 2021, the FDA placed a hold on patients under 18 years in trials evaluating lovotibeglogene autotemcel in the treatment of sickle cell disease, after anemia developed in an adolescent patient. Enrollment of patients 18 and older was not affected. The FDA lifted the hold on patients younger than 18 in December 2022 after data suggested that anemia is only seen in patients with alpha-thalassemia trait. These patients are now excluded from studies.
 
The FDA accepted the NDA for etrasimod for for the treatment of moderately-to-severely active ulcerative colitis and set a PDUFA date for 10/15/2023.
 
Idorsia submitted an NDA for aprocitentan for the treatment of difficult-to-control hypertension.