August PDUFA Preview

There are seven investigational drugs with PDUFA Dates in August 2019.

1. ​Pexidartinib from Daiichi Sankyo/Plexxikon with an August 3, 2019 PDUFA Date is a CSF-1R inhibitor administered orally to treat giant cell tumors of the tendon sheath. Pexidartinib has Orphan and Breakthrough Therapy priority designations. 
2. Bioproject Pharma’s Pitolisant has an August 12, 2019 PDUFA Date and is a H3 receptor antagonist administered orally for treating Narcolepsy. Pitolisant has Breakthrough and Fast Track priority designations.
3. Entrectinib from Roche, PDUFA Date of August 18, 2019, is an oral Tyrosine kinase inhibitor for the treatment of advanced or metastatic solid tumors that harbor NTRK1/2/3 or ROS1 gene activating fusions. Roche is seeking approval based on an integrated analysis from four Phase I and II trials that included data from 53 people with ROS1-activating gene fusions and 54 people with locally advanced or metastatic NTRK fusion-positive solid tumors. If approved, entrectinib would compete with larotrectinib to treat NTRK-fusion positive solid tumors and critzotinib to treat ROS1-mutated non-small cell lung cancer. The drug has a Breakthrough Therapy priority designation.
4. Sarepta Therapeutics’ golodirsen’s PDUFA Date is set for August 19, 2019. The drug is an Exon 53 skipping Intravenous infusion targeting Duchenne muscular dystrophy. A review by ICER of treatments for Duchenne’s found limited data that a slight increase in levels of dystrophin with eteplirsen and golodirsen provides a clinically significant effect. ICER concluded that if golodirsen is priced like eteplirsen, it would not meet cost benefit standards. At the current price, if either drug were able to restore perfect health in DMD patients for an additional 40 years of life, the drugs would far exceed cost-effectiveness thresholds of $100,000 and $150,000 per Quality-Adjusted Life Year (QALY) gained or $100,000 and $150,000 per Life Year Gained (LYG).
5. Lefamulin from Nabriva has an August 19, 2019 PDUFA Date. It’s a pleuromutilin antibiotic administered orally or by intravenous bolus to treat community-acquired pneumonia. The drug has Fast Track and Qualified Infectious Disease Product (QIDP) priority designations. 
6. AbbVie’s upadacitinib, with a PDUFA Date of August 19, 2019, is an oral JAK Inhibitor for the treatment of Rheumatoid Arthritis and Crohn's disease. The product has a Breakthrough Therapy priority designation.
7. Istradefylline from Kyowa Hakko Kirin has a PDUFA Date of August 27, 2019 and is an A2A antagonist administered orally for patients on levodopa therapy with Parkinson's Disease wearing-off phenomenon. Istradefylline is approved in Japan as adjunctive therapy for the treatment of Parkinson's Disease. The FDA rejected Istradefylline in February 2008 due to efficacy concerns. The FDA accepted the NDA for istradefylline in April 2019 and set the August PDUFA date at that time. The drug has no priority designations.