Update for the week ended 23 February 2019

FDA approved one drug and added four new PDUFA Dates

The FDA approved turoctocog alfa pegol (Esperoct from Novo Nordisk) on 2/21/2019 for the treatment of hemophilia A, however due to a contractual agreement, the drug will not launch until 2020. Esperocot is a long acting version of NovoEight (turoctocog alfa).

An FDA review of selinexor found limited efficacy and significant toxicity in patients with relapsed/refractory multiple myeloma treated with a combination of selinexor dexamethasone. Based on data submitted and historical studies with high-dose dexamethasone, the FDA felt it was difficult to isolate the treatment effect of selinexor. The FDA's Oncologic Drugs Advisory Committee will review selinexor on 2/26/2019.

New PDUFA dates

• The FDA has accepted the BLA for entrectinib, which suggests a PDUFA date of August 18, 2019.
• The FDA has accepted the BLA for polatuzumab, which suggests a PDUFA date of August 19, 2019.
• The FDA has accepted the NDA for upadacitinib, which suggests a PDUFA date of August 19, 2019.
• The FDA has accepted the NDA for oral and IV lefamulin, which suggests a PDUFA date of August 19, 2019.
  

New Generic Names

• NKTR-214 has been assigned the generic name bempegaldesleukin.
• OMS721 has been assigned the generic name narsoplimab.
• BHV3241 has been assigned the generic name verdiperstat. The FDA has awarded verdiperstat Orphan Drug status for the treatment of multiple system atrophy.​

Pfizer and Lilly announced that a 16-week, 1,832 patient, Phase III trial, treatment with tanezumab 10 mg reduced the daily average Low Back Pain Intensity score compared to placebo, but the 5 mg doses did not reach a significant difference in patients with moderate-to-severe chronic low back pain. 

In the 1,509 patient, Phase III ARAMIS trial, the most common ADR with darolutamide compared to placebo was fatigue.

Reata announced that in the 12-week, 103 patient, Phase II, open-label, PHOENIX study, patients treated with bardoxolone had an increase in their mean estimated glomerular filtration rate (eGFR) from of 7.8 mL/min/1.73 m2 improvement in patients with patients with autosomal dominant polycystic kidney disease, IgA nephropathy, Focal Segmental Glomerulosclerosis, and type 1 diabetic chronic kidney disease. 

Alder submitted a BLA for eptinezumab in February 2019.

Vanda is suing the FDA to lift the partial hold on tradipitant clinical trials.