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. |