US FDA approvals tracker: August

From a total of 13 marketing decisions expected from the FDA, August saw eight approvals and three complete response letters. Notable regulatory nods included two Jak inhibitors: Abbvie’s rheumatoid arthritis drug Rinvoq and Celgene’s myelofibrosis therapy Inrebic; the latter was not included in Vantage's August PDUFA analysis as it had been due a decision in September. Both products came with label warnings that could limit their uses, namely thrombosis for Rinvoq and encephalopathy for Inrebic. Meanwhile, Sarepta got a knockback for its follow-on Duchenne therapy Vyondys 53, with the company citing the risk of infection at intravenous infusion ports and kidney toxicity. Important decisions due this month include the oral version of Novo Nordisk's GLP1 agonist semaglutide (Go or no go? Novo awaits call on oral sema, August 30, 2019).

Notable first-time US approval decisions in August
Project Company

Product NPV ($m)*

Outcome Vantage story
Turalio (pexidartinib) Daiichi Sankyo 545 Approved -
Wakix (pitolisant) Harmony Biosciences - Approved -
KPI-121 Kala Pharmaceuticals 1,650 CRL US rejection knocks Kala off its Stride
Rozlytrek Roche 1,017 Approved Bayer could become the latest casualty of Roche’s targeted pricing
Vyondys 53 (golodirsen) Sarepta 711 CRL The Sarepta fairy tale takes a worrying turn
Xenleta (lefamulin) Nabriva Therapeutics 456** Approved Nabriva gets a win, but investors are lukewarm
Rinvoq (upadacitinib)  Abbvie 10,595 Approved Abbvie’s black box could be bad news for Galapagos and Gilead
Nouriast/Nourianz Kyowa Kirin 216 Approved -
Rexista/Oxycodone ER Intellipharmaceutics International 112 No decision yet (adcom delay) -
NKTR-181 Nektar Therapeutics 532 No decision yet (adcom delay) -
Pretomanid Mylan/TB Alliance - Approved -
Inrebic (fedratinib)*** Celgene 1,730 Approved Celgene's myelofibrosis win represents an expensive green light
*NPV data from August 30; **includes both oral and IV versions; ***not noted in August story, PDUFA date September 3, 2019. Source: EvaluatePharma, Go or no go? Abbvie, Roche and Kala await key decisions.


Supplementary and other notable approval decisions in August
Product Company Event type Outcome
Hetlioz Vanda Pharmaceuticals sNDA for jet lag disorder CRL
Sodium thiosulfate injection Hope Pharmaceuticals sNDA for chemotherapy-induced side effects No decision yet
Source: EvaluatePharma.

Share This Article